Century Therapeutics Announces Acquisition of Empirica Therapeutics | DNA RNA and Cells | News Channels – PipelineReview.com

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Century Therapeutics Canada will develop induced pluripotent Stem Cell (iPSC)-derived allogeneic immune cell therapies against glioblastoma (GBM)

PHILADELPHIA, PA, USA I June 23, 2020 ICentury Therapeutics today announced its acquisition of Empirica Therapeutics to leverage its iPSC-derived allogeneic cell therapies against glioblastoma (GBM).

We are pleased to welcome the Empirica team to the Century family. Their deep expertise and unique capabilities will allow us to accelerate efforts to develop iPSC derived immune effector cell products designed to treat and potentially cure brain cancer, said Lalo Flores, PhD, Chief Executive Officer of Century Therapeutics. GBM is a particularly aggressive, often treatment-resistant form of adult brain cancer with an average survival time of under two years. Together, we are in a stronger position to develop potentially curative cell therapies for this devastating disease.

Empirica Therapeutics was founded by Dr. Sheila Singh, MD, PhD, Professor of Surgery and Biochemistry and chief pediatric neurosurgeon at McMaster Childrens Hospital, and Dr. Jason Moffat, PhD, Professor of Molecular Genetics at the University of Toronto and an expert in functional genomics and gene-editing platforms. The companys science is based on a powerful integrative multi-omics platform, combined with its unique patient-derived, therapy-adapted models of recurrent GBM, that has led to the discovery and validation of novel brain tumor targets. Empiricas cutting edge preclinical models of recurrent GBM, have demonstrated the potential of CAR-T cell therapy in GBM, as published in a May 2020 Cell Stem Cell paper.

Our team is excited to become part of Century Therapeutics, whose iPSC-derived allogeneic cell therapies show immense potential for treating solid as well as hematologic malignancies, said Dr. Singh. Dr. Singh served as Empiricas CEO after co-founding the company with Chief Scientific Officer Dr. Moffat. We look forward to combining our unique patient-based cancer models with Centurys platform to create promising treatments for the patients who need them most, Singh said.

Janelle Anderson, PhD, Chief Strategy Officer at Century Therapeutics, shepherded the deal forming the subsidiary, which will be known as Century Therapeutics Canada and based in Hamilton, Ontario. Financial terms of the deal have not been disclosed.

About Century Therapeutics

Century Therapeutics is harnessing the power of stem cells to develop curative cell therapy products for cancer that overcome the limitations of first-generation cell therapies. Our genetically engineered, universal iPSC-derived immune effector cell products (iNK, iT) are designed to specifically target hematologic and solid tumor cancers. Our commitment to developing off-the-shelf cell therapies will expand patient access and provides an unparalleled opportunity to advance the course of cancer care. Century was launched in 2019 by founding investor Versant Ventures in partnership with Fujifilm and Leaps by Bayer. For more information, please visit http://www.centurytx.com.

About Glioblastoma (GBM)

Glioblastoma (GBM) is one of the most common types of primary brain tumor in adults and is almost uniformly lethal, with less than 5% of patients living beyond five years. GBM has an incidence rate of 3 per 100,000 people annually in the United States of America. The standard of care for GBM consists of tumor resection following by chemotherapy and radiation. Despite aggressive multimodal treatment, almost all patients experience relapse 7-9 months post-diagnosis and median survival has not extended beyond 16-20 months over the past decade. Recent studies suggest that the primary GBM tumor evolves significantly during the course of therapy and presents itself as a much more aggressive tumor at the time of recurrence. The treatment-resistant nature of GBM to standard therapies provides compelling motivation for developing novel treatment approaches.

SOURCE: Century Therapeutics

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BrainStorm Cell Therapeutics to Join the Russell 2000 Index and Russell 3000 Index – PRNewswire

NEW YORK, June 23, 2020 /PRNewswire/ --BrainStorm Cell Therapeutics Inc. (NasdaqCM: BCLI), a leading developer of adult stem cell therapies for neurodegenerative diseases, today announced that its shares will join the Russell 2000 Index and the broad-market Russell 3000 Index at the conclusion of the 2020 Russell indexes annual reconstitution, effective after the US stock market opens on June 29, 2020.

Annual Russell indexes reconstitution captures the 4,000 largest US stocks as of May 8, ranking them by total market capitalization. Membership in the US all-cap Russell 3000 Index, which remains in place for one year, means automatic inclusion in the large-cap Russell 1000 Index or small-cap Russell 2000 Index as well as the appropriate growth and value style indexes. FTSE Russell determines membership for its Russell indexes primarily by objective, market-capitalization rankings and style attributes.

Russell indexes are widely used by investment managers and institutional investors for index funds and as benchmarks for active investment strategies. Approximately $9 trillion in assets are benchmarked against Russell's US indexes. Russell indexes are part of FTSE Russell, a leading global index provider.

For more information on the Russell 3000 Index and the Russell indexes reconstitution, go to the "Russell Reconstitution" section on the FTSE Russell website.

About FTSE Russell

FTSE Russell is a leading global index provider creating and managing a wide range of indexes, data and analytic solutions to meet client needs across asset classes, style and strategies. Covering 98% of the investable market, FTSE Russell indexes offer a true picture of global markets, combined with the specialist knowledge gained from developing local benchmarks around the world.

FTSE Russell index expertise and products are used extensively by institutional and retail investors globally. Approximately $16 trillion is currently benchmarked to FTSE Russell indexes. For over 30 years, leading asset owners, asset managers, ETF providers and investment banks have chosen FTSE Russell indexes to benchmark their investment performance and create investment funds, ETFs, structured products and index-based derivatives. FTSE Russell indexes also provide clients with tools for asset allocation, investment strategy analysis and risk management.

A core set of universal principles guides FTSE Russell index design and management: a transparent rules-based methodology is informed by independent committees of leading market participants. FTSE Russell is focused on index innovation and customer partnership applying the highest industry standards and embracing the IOSCO Principles. FTSE Russell is wholly owned by London Stock Exchange Group. For more information, visit http://www.ftserussell.com/.

About BrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm has fully enrolled a Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six U.S. sites supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. BrainStorm also recently received U.S. FDA clearance to initiate a Phase 2 open-label multicenter trial in progressive Multiple Sclerosis. The Phase 2 study of autologous MSC-NTF cells in patients with progressive MS (NCT03799718) started enrollment in March 2019.

Safe-Harbor Statement

Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorm's need to raise additional capital, BrainStorm's ability to continue as a going concern, regulatory approval of BrainStorm's NurOwn treatment candidate, the success of BrainStorm's product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorm's NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorm's ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorm's ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at http://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

CONTACTS

Investor Relations:Michael RiceLifeSci Advisors, LLCPhone: +1 646 889 1200[emailprotected]

Public Relations:Paul TyhalaSmithSolve973.442.1555[emailprotected]

SOURCE Brainstorm Cell Therapeutics Inc

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Acute Myeloid Leukemia (AML) Therapeutics Market Promising Growth Opportunities over 2017 2025 – 3rd Watch News

Leukemia are a heterogeneous group of cancers affecting the bone marrow and White Blood Cells (WBC). Leukemia is characterized by the rapid increase of abnormal blood cells growth or blasts, resulting in a decrease in the numbers of healthy, normal fully modified blood cells, leading to the typical symptoms of bleeding, anemia, and high risk of infection. Leukemia can grow along either the myeloid or lymphoid stem cell lines, it depends on the effect of genetic and epigenetic mutations on the progression of pluripotent stem cells to the various lines of mature cells which then pass into the blood. The effected line, combined with the rate of action and growth of disease reflects the four types of leukemias- Acute Myeloid Leukemia (AML), chronic lymphoblastic leukemia, chronic myeloid leukemia, acute lymphoblastic leukemia. AML: Acute Myeloid Leukemia, is a serious condition, its the most common leukemia suffered by adult people. According to a report from American Cancer Society, the average age for first diagnostic for AML is 64. With few days without treatment, AML develops fast, in duration of few weeks, the patient becomes severely ill. Due to its fast onset and acuteness in nature, there is no staging system for Acute Myeloid Leukemia (AML).The treatment for Acute Myeloid Leukemia (AML) has changed in last 4 decades.

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The drug approval process is difficult in AML, (many drugs have not been approved by USFDA, for instance Laromustine, Dacogen and Tipitarnib) efforts have been made to introduce new therapies in the AML market.

Primary drivers boosting the growth of acute myeloid leukemia (AML) therapeutics market are minimal but increased prevalence of acute myeloid leukemia (AML), increased drug approval rate for AML, classification of acute myeloid leukemia (AML) as an orphan disease. Over the forecast period, population of people over 65 year is anticipated to increase, which is another key driver for acute myeloid leukemia (AML) therapeutics market.

However, lack of targeted therapies in current acute myeloid leukemia (AML) therapeutics landscape, the drug difficult approval process in AML can hinder the growth of acute myeloid leukemia (AML) therapeutics market, but this restraint has opened an opportunity for key players to innovate acute myeloid leukemia (AML) therapeutics market.

The global acute myeloid leukemia (AML) therapeutics market is segmented on the basic of disease subtype, treatment type, end user and region.

Based on the disease subtype, the acute myeloid leukemia (AML) therapeutics market is segmented into the following:

Based on treatment type, the acute myeloid leukemia (AML) therapeutics market is segmented into the following:

Based on end user, the acute myeloid leukemia (AML) therapeutics market is segmented into the following:

The global acute myeloid leukemia (AML) therapeutics market is anticipated to show lucrative growth owing to increased investment in innovative technologies by key players. Players in this market using various strategies to fuel their global footprint and to gain a competitive edge. Product pipelines, new product launches, agreements and collaborations, acquisitions, mergers and clinical trials are some key strategies applied from global players in recent years are anticipated to give a robust hike to the market in the forecast period.

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Geographically, acute myeloid leukemia (AML) therapeutics market is segmented into regions viz. North America, Latin America, Europe, Asia Pacific and Japan, Middle East and Africa. North America is anticipated to be major contributor to this market accounting maximum percent of share in AML therapeutics market followed by Europe. Slow but constant growth in prevalence for AML in North America is anticipated to fuel the growth in acute myeloid leukemia (AML) therapeutics market. In Asia pacific region, China and India are anticipated to show high growth in acute myeloid leukemia (AML) therapeutics market due to new developments in healthcare infrastructure in the region.

The players in acute myeloid leukemia (AML) therapeutics market include Ambit Biosciences Corporation, Celgene Corporation, Cephalon Inc., Clavis Pharma ASA, Eisai Co. Ltd, Genzyme Corporation, and Sunesis Pharmaceuticals Inc., Abbvie Inc., Astellas Pharma Inc, CTI Biopharma Corp etc.

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UK Startup Bags 37M to Remove Cell Therapy Manufacturing… – Labiotech.eu

UK biotech startup Bit Bio has received 36.9M in Series A funding to boost the manufacturing efficiency of human cells for use in cell therapy and drug discovery.

The impressive deal brings Bit Bios total funding to 44.4M since it was spun off from the University of Cambridge in 2016. Its the second-largest Series A round raised by a European biotech startup so far this year, just after a 55M round raised by the Dutch neurology company Prilenia Therapeutics earlier this month.

Richard Klausner, an individual investor who previously founded US companies Lyell Immunopharma, Juno, and Grail, led the round. Other contributors included the Chicago-based ARCH Venture Partners; the San Francisco healthcare investment firm Foresite Capital; and the German early-stage investor Blueyard Capital.

Human cell lines are essential for the development of drugs and cell therapies. But specialized human cells such as neurons and muscle cells are difficult to source from tissue samples in bulk. One way around this problem is to source more easily obtainable types of human cells from tissue samples, reprogram them into stem cells, and then transform them into the desired cell type. However, existing methods tend to use viral vectors to engineer the stem cells, an approach that produces a low yield of the desired cell type.

Today, the limitations of traditional stem cell biology have become obvious. The protocols for deriving cells using classical methods too often lack consistency and scalability, Bit Bio founder and CEO, Mark Kotter, told me.

Bit Bios approach is to screen large cell biology datasets for cocktails of proteins called transcription factors that are needed to turn stem cells into the desired cell type. The company then genetically engineers the stem cells so that they switch on this cocktail when given the antibiotic doxycycline. This way, the stem cells can be transformed more precisely and efficiently than with viral vectors.

If one considers the transcription factor combinations that determine a cellular identity as a program, then [our technology] is the enter button to the operating system of life that enables faithful execution of any genetic program, said Kotter.

So far, the team has successfully reprogrammed human stem cells into a range of specialized cells such as neurons and muscle cells. If developed at a commercial scale, the technology could reduce the limits on human cell manufacture that is currently holding back the production of cell therapies and drug discovery tools.

Bio Bit will use the latest Series A funding to take its technology to the industrial scale. Once this is established, the company expects to apply its technology to the development of cell therapies.

Another Cambridge-based biotech company working to optimize the production of human cells is Mogrify, which turns cells from adult tissue samples into other mature cells, except without the step of turning them into stem cells first. This could make the process even cheaper and more scalable than technology using stem cell stages.

Manuela Callari is a freelance science and medical writer from Sydney, Australia. She has a Ph.D. in Medical Science, a Bachelors, and a Masters degree in Material Science. She used to wear a lab coat, now she writes about science, technology, environmental science, health, and medicine.

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EHA25Virtual: Adult Patients With Sickle Cell Disease May Be at Increased Risk of Adverse Outcomes From COVID-19 – Yahoo Finance

THE HAGUE, Netherlands, June 13, 2020 /PRNewswire/ -- Sickle cell disease (SCD) and thalassemia are severe inherited blood disorders, often referred to as "hemoglobinopathies." They predominantly affect the Black and Asian ethnic minority populations in England. To ensure good standards and equitable access to care, the National Health Service in England has recently commissioned a model of regional care networks overseen by a new body, the National Haemoglobinopathy Panel. This organizational structure has enabled a rapid response to the COVID-19 epidemic and enabled collection of national data on new cases and outcomes to determine if hemoglobinopathy patients are at risk of adverse COVID-19 outcomes.

EHA Logo (PRNewsfoto/European Hematology Association)

We present an analysis on data collected up to June 5th indicating that the majority of cases have been mild, and in particular children do not appear to be at increased risk. However, the data suggests that adults with SCD may be more vulnerable to adverse outcomes. Therefore, we recommend that isolation precautions should be lifted cautiously, and that new therapies and vaccination for COVID-19, when available, should be prioritized for this patient group.

Presenter: Dr Paul Telfer Affiliation:Queen Mary University of London, Barts Health NHS Trust, London, UK Abstract:#LB2606 REAL-TIME NATIONAL SURVEY OF COVID-19 IN HEMOGLOBINOPATHY AND RARE INHERITED ANEMIA PATIENTS

About the EHA Annual Congress: Every year in June, EHA organizes its Annual Congress in a major European city. This year due to the COVID19 pandemic, EHA transformed its physical meeting into a Virtual Congress. The Congress is aimed at health professionals working in or interested in the field of hematology. The scientific program topics range from stem cell physiology and development to leukemia; lymphoma; diagnosis and treatment; red blood cells; white blood cells and platelet disorders; hemophilia and myeloma; thrombosis and bleeding disorders; as well as transfusion and stem cell transplantation. Embargo: Please note that our embargo policy applies to all selected abstracts in the Press Briefings. For more information, see our EHA Media and Embargo policy here.

Website: ehaweb.org

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SOURCE European Hematology Association (EHA)

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CRISPR Therapeutics and Vertex Announce New Clinical Data for Investigational Gene-Editing Therapy CTX001 in Severe Hemoglobinopathies at the 25th…

-Beta thalassemia: Two patients are transfusion independent at 5 and 15 months after CTX001 infusion; data demonstrate clinical proof-of-concept for CTX001 in transfusion-dependent beta thalassemia-

-Sickle cell disease: Patient is free of vaso-occlusive crises at 9 months after CTX001 infusion-

-Five patients with beta thalassemia and two patients with sickle cell disease have been treated to date with CTX001 and all have successfully engrafted-

ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, June 12, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced new clinical data for CTX001, an investigational CRISPR/Cas9 gene-editing therapy, from the CLIMB-111 and CLIMB-121 Phase 1/2 trials in transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD), and highlighted recent progress in the CTX001 development program. These data were presented during an oral presentation at the European Hematology Association (EHA) virtual congress by Dr. Selim Corbacioglu, Professor of Pediatrics and the Chair of Pediatric Hematology, Oncology, and Stem Cell Transplantation, Regensburg University Hospital, Regensburg, Germany.

CLIMB-111 Trial in Transfusion-Dependent Beta Thalassemia Updated ResultsData presented today at EHA demonstrate clinical proof-of-concept for CTX001 in TDT. Data include longer-duration follow-up data for the first patient with TDT treated with CTX001 and new data for the second TDT patient treated. CRISPR Therapeutics and Vertex announced initial data for the first TDT patient in November of 2019.

Patient 1 with TDT has the 0/IVS-I-110 genotype, which is associated with a severe phenotype similar to 0/0, and had a transfusion requirement of 34 units of packed red blood cells per year (annualized rate during the two years prior to consenting for the trial) before enrolling in the clinical trial. As previously reported, the patient achieved neutrophil engraftment 33 days after CTX001 infusion and platelet engraftment 37 days after infusion. After CTX001 infusion, two serious adverse events (SAEs) occurred, neither of which the principal investigator (PI) considered related to CTX001: pneumonia in the presence of neutropenia, and veno-occlusive liver disease attributed to busulfan conditioning; both subsequently resolved. New data presented today show that at 15 months after CTX001 infusion, the patient was transfusion independent and had total hemoglobin levels of 14.2 g/dL, fetal hemoglobin of 13.5 g/dL, and F-cells (erythrocytes expressing fetal hemoglobin) of 100.0%. Bone marrow allelic editing was 78.1% at 6 months and 76.1% at one year.

Patient 2 with TDT has the 0/IVS-II-745 genotype and had a transfusion requirement of 61 units of packed red blood cells per year (annualized rate during the two years prior to consenting for the trial) before enrolling in the clinical trial. The patient achieved neutrophil engraftment 36 days after CTX001 infusion and platelet engraftment 34 days after infusion. After CTX001 infusion, two SAEs occurred, neither of which the PI considered related to CTX001: pneumonia and an upper respiratory tract infection; both subsequently resolved. At 5 months after CTX001 infusion, the patient was transfusion independent and had total hemoglobin levels of 12.5 g/dL, fetal hemoglobin of 12.2 g/dL, and F-cells (erythrocytes expressing fetal hemoglobin) of 99.4%.

Hemoglobin data over time are presented for Patient 1 and Patient 2 below.

Figure 1accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/35581299-d683-44b0-a75e-7a1a9b9fe9eb

CLIMB-121 Trial in Severe Sickle Cell Disease Updated Results Data presented today at EHA reflect longer-duration follow-up data for the first patient with SCD treated with CTX001. CRISPR Therapeutics and Vertex announced initial data for this first SCD patient in November of 2019.

Patient 1 with SCD experienced seven vaso-occlusive crises (VOCs) and five packed red blood cell transfusions per year (annualized rate during the two years prior to consenting for the trial) before enrolling in the clinical trial. As previously reported, the patient achieved neutrophil and platelet engraftment 30 days after CTX001 infusion. After CTX001 infusion, three SAEs occurred, none of which the PI considered related to CTX001: sepsis in the presence of neutropenia, cholelithiasis and abdominal pain; all subsequently resolved. New data presented today show that at 9 months after CTX001 infusion, the patient was free of VOCs, was transfusion independent and had total hemoglobin levels of 11.8 g/dL, 46.1% fetal hemoglobin, and F-cells (erythrocytes expressing fetal hemoglobin) of 99.7%. Bone marrow allelic editing was 81.4% at 6 months. Figure 2 presents the hemoglobin data over time for this patient.

Figure 2 accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/7610c5bd-25c8-4f5b-be86-8bc16ed57eb1

With these new data, we are beginning to see early evidence of the potential durability of benefit from treatment with CTX001, as well as consistency of the therapeutic effect across patients, said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. These highly encouraging early data represent one more step toward delivering on the promise and potential of CRISPR/Cas9 therapies as a new class of potentially transformative medicines to treat serious diseases.

The data announced today are remarkable, including the demonstration of clinical proof-of-concept in TDT, said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. While these are still early days, these data mark another important milestone for this program and for the field of gene editing. The results presented at this medical conference add to results previously shared demonstrating that CRISPR/Cas9 gene editing has the potential to be a curative therapy for severe genetic diseases like sickle cell and beta thalassemia.

In my 25 years of caring for children and young adults facing both sickle cell disease and beta thalassemia, I have seen how these diseases can adversely affect patients lives in very significant ways, said Dr. Haydar Frangoul, Medical Director of Pediatric Hematology and Oncology at Sarah Cannon Research Institute, HCA Healthcares TriStar Centennial Medical Center and senior author of the abstract presented at the EHA virtual congress. I am encouraged by the preliminary results, which demonstrate, in essence, a functional cure for patients with beta thalassemia and sickle cell disease.

Recent Progress in the Phase 1/2 Clinical TrialsCLIMB-111 for TDT has dosed a total of 5 patients, and all patients have successfully engrafted. The trial is also now open for concurrent dosing after successful dosing and engraftment of the first two patients. Additionally, CLIMB-111 has been expanded to allow enrollment of 0/0 patients and is in the process of being expanded to allow enrollment of pediatric patients ages 12 years or older.

CLIMB-121 for SCD has dosed a total of 2 patients and both patients have successfully engrafted. The trial is also now open for concurrent dosing after successful dosing and engraftment of these first two patients.

The initial safety profile in these trials appears to be consistent with myeloablative busulfan conditioning and an autologous hematopoietic stem cell transplant.

In March 2020, clinical trial sites in the U.S. and Europe temporarily paused their elective hematopoietic stem cell transplant programs due to the COVID-19 pandemic, and as a result, CRISPR and Vertex temporarily paused conditioning and dosing in these trials. Enrollment, mobilization and drug product manufacturing in each trial remains ongoing. The companies are now in the process of re-initiating dosing with CTX001 at certain clinical trial sites. The CLIMB-111 and CLIMB-121 clinical trials are ongoing, and patients will be followed for 2 years following CTX001 infusion. The companies expect to provide additional data in the second half of 2020.

About CTX001CTX001 is an investigational ex vivo CRISPR gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD in which a patients hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth, which then switches to the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for TDT patients and reduce painful and debilitating sickle crises for SCD patients.

Based on progress in this program to date, CTX001 has been granted Regenerative Medicine Advanced Therapy (RMAT) from the U.S. FDA, Orphan Drug Designation from both the FDA and the European Medicines Agency (EMA), and Fast Track Designation from the FDA for both SCD and TDT.

CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex. CTX001 is the most advanced gene-editing approach in development for TDT and SCD.

About CLIMB-111The ongoing Phase 1/2 open-label trial, CLIMB-Thal-111, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 18 to 35 with TDT. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About CLIMB-121The ongoing Phase 1/2 open-label trial, CLIMB-SCD-121, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 18 to 35 with severe SCD. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About the Gene-Editing Process in These TrialsPatients who enroll in these trials will have their own hematopoietic stem and progenitor cells collected from peripheral blood. The patients cells will be edited using the CRISPR/Cas9 technology. The edited cells, CTX001, will then be infused back into the patient as part of a stem cell transplant, a process which involves, among other things, a patient being treated with myeloablative busulfan conditioning. Patients undergoing stem cell transplants may also encounter side effects (ranging from mild to severe) that are unrelated to the administration of CTX001. Patients will initially be monitored to determine when the edited cells begin to produce mature blood cells, a process known as engraftment. After engraftment, patients will continue to be monitored to track the impact of CTX001 on multiple measures of disease and for safety.

About the CRISPR-Vertex Collaboration CRISPR Therapeutics and Vertex entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 represents the first treatment to emerge from the joint research program. CRISPR Therapeutics and Vertex will jointly develop and commercialize CTX001 and equally share all research and development costs and profits worldwide.

About CRISPR TherapeuticsCRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit http://www.crisprtx.com.

CRISPR Therapeutics Forward-Looking StatementThis press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements made by Dr. Kulkarni, Dr. Kewalramani and Dr. Frangoul in this press release, as well as statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) the status of clinical trials (including, without limitation, the expected timing of data releases and activities at clinical trial sites) related to product candidates under development by CRISPR Therapeutics and its collaborators, including expectations regarding the data that is being presented at the European Hematology Associations virtual congress; (ii) the expected benefits of CRISPR Therapeutics collaborations; and (iii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: potential impacts due to the coronavirus pandemic, such as the timing and progress of clinical trials; the potential for initial and preliminary data from any clinical trial and initial data from a limited number of patients (as is the case with CTX001 at this time) not to be indicative of final trial results; the potential that CTX001 clinical trial results may not be favorable; that future competitive or other market factors may adversely affect the commercial potential for CTX001; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics most recent annual report on Form 10-K, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

About VertexVertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes. For company updates and to learn more about Vertex's history of innovation, visit http://www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

Vertex Special Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Dr. Kulkarni, Dr. Kewalramani and Dr. Frangoul in this press release, and statements regarding our plans and expectations for our clinical trials and clinical trial sites, and our expectations regarding future data announcements. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under Risk Factors in Vertex's annual report and subsequent quarterly reports filed with the Securities and Exchange Commission and available through the company's website at http://www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

CRISPR Therapeutics Investor Contact:Susan Kim, +1 617-307-7503susan.kim@crisprtx.com

CRISPR Therapeutics Media Contact:Rachel EidesWCG on behalf of CRISPR+1 617-337-4167reides@wcgworld.com

Vertex Pharmaceuticals IncorporatedInvestors:Michael Partridge, +1 617-341-6108orZach Barber, +1 617-341-6470orBrenda Eustace, +1 617-341-6187

Media:mediainfo@vrtx.comorU.S.: +1 617-341-6992orHeather Nichols: +1 617-839-3607orInternational: +44 20 3204 5275

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CRISPR Therapeutics and Vertex Announce New Clinical Data for Investigational Gene-Editing Therapy CTX001 in Severe Hemoglobinopathies at the 25th...

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BrainStorm to Present at the Raymond James Human Health Innovations Conference – PRNewswire

NEW YORK, June 11, 2020 /PRNewswire/ --BrainStorm Cell Therapeutics Inc.(NASDAQ: BCLI), a leading developer of adult stem cell therapies for neurodegenerative diseases, today announced Chaim Lebovits, CEO and Ralph Kern, MD, MHSc, President and Chief Medical Officer, will present a corporate overview on Thursday, June 18 at 9:00 am EST, during theRaymond James Human Health Innovations Conference, a virtual event connecting institutional investors with company management teams that will be held June 15-18, 2020.

Mr. Lebovits and Dr. Kern will update conference participants on the Company's investigational therapeutic, NurOwn, that is currently in a fully enrolled phase 3 study for the treatment of ALS and a phase 2 study for the treatment of progressive multiple sclerosis. Additionally, they will present an overview of the Company's financial position and pipeline. After the presentation, the management team will participate in a question and answer session with institutional investors.

Mr. Lebovits and Dr. Kern will be joined by David Setboun, PhD, MBA, Chief Operating Officer, Stacy Lindborg, PhD, Head of Global Clinical Research, and Preetam Shah, PhD, MBA, Chief Financial Officer, for a series of one-on-one meetings, with select institutional investors arranged by Raymond James.

Participants can view the presentation via the event link and those unable to join will have access to an archived link on the Company's Events and Presentation webpage after the conclusion of the conference.

EVENT: Raymond James Human Health Innovations Conference

PRESENTATION: Thursday, June 18th at 9:00 am EST

LINK: https://bit.ly/2YmZf8u

About NurOwn

NurOwn (autologous MSC-NTF) cells represent a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. BrainStorm has fully enrolled a Phase 3 pivotal trial of autologous MSC-NTF cells for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm also recently receivedU.S.FDA acceptance to initiate a Phase 2 open-label multicenter trial in progressive MS and enrollment began inMarch 2019.

AboutBrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc.is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from theU.S. Food and Drug Administration(U.S.FDA) and theEuropean Medicines Agency(EMA) in ALS. BrainStorm has fully enrolled a Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at sixU.S.sites supported by a grant from theCalifornia Institute for Regenerative Medicine(CIRM CLIN2-0989). The pivotal study is intended to support a filing forU.S.FDA approval of autologous MSC-NTF cells in ALS. BrainStorm also recently receivedU.S.FDA clearance to initiate a Phase 2 open-label multicenter trial in progressive Multiple Sclerosis. The Phase 2 study of autologous MSC-NTF cells in patients with progressive MS (NCT03799718) started enrollment inMarch 2019.

Safe-Harbor Statement

Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could causeBrainStorm Cell Therapeutics Inc.'sactual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorm's need to raise additional capital, BrainStorm's ability to continue as a going concern, regulatory approval of BrainStorm's NurOwn treatment candidate, the success of BrainStorm's product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorm's NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorm's ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorm's ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

CONTACTS

Investor Relations:Preetam Shah, MBA, PhDChief Financial OfficerBrainStorm Cell Therapeutics Inc.Phone: +1-862-397-1860[emailprotected]

Media:

Sean LeousWestwicke/ICR PRPhone: +1-646-677-1839[emailprotected]

SOURCE Brainstorm Cell Therapeutics Inc

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BrainStorm to Present at the Raymond James Human Health Innovations Conference - PRNewswire

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Mesenchymal Stem Cells Market – Advances & Applications scrutinized in the new analysis – WhaTech Technology and Markets News

Mesenchymal Stem Cells Market- Advances & Applications (2018) report provides 73 company profiles in each market niche, Identify the market segments that compose the MSC industry, Advanced Therapy Medicinal Products, Stem Cell Research Publications, Funding And Patents, Clinical Trials Involving MSCS. This 200+ page global strategic report presents trend rate data for Mesenchymal Stem Cells market, including rates of MSC patents, grants, scientific publications, and clinical trials, as well associal analytics that identify online behavior related to MSCs.

Mesenchymal stem cells (MSCs) are multipotent cells that can differentiate into a variety of cell types, including osteoblasts, chondrocytes, myocytes, adipocytes, and potentially other cell types.

In addition to secreting factors that can stimulate tissue repair, MSCs can substantially alter their microenvironment, exerting effects that are both anti-nflammatory and anti-fibrotic. MSCs are advantageous over other stem cells types for a variety of reasons, including that they are immuno-privileged, making them an advantageous cell type for allogenic transplantation.

MSCs appear to be an exceptionally promising tool for cell therapy, because of their unusual advantages, which include availability, expand ability, transplant ability, and ethical implications.

Complete Report Mesenchymal Stem Cells Market - Advances & Applications (2018) including 216 pages and 73 Company Profiles with List of Figures are available at http://www.reportsnreports.com/reportstions.html

Company Profiles Discussed: AdiSave, AdiCyte, AlloSource Inc., American CryoStem Corporation, Anterogen Co., Ltd., Apceth Biopharma GmbH, Athersys Inc., Astarte Biologics Inc., BioCardia Inc., BioRestorative Therapies Inc., Bone Therapeutics SA, Brainstorm Cell Therapeutics, Caladrius Biosciences Inc., Capricor Therapeutics Inc., Cell Applications Inc., Cell Cure Neurosciences Ltd., CellGenix Technologie Transfer GmbH, CellProthera SAS, Celltex Therapeutics Corporation, Cellular Dynamics International Inc., Celprogen Inc., Cell Therapies Pty Ltd.

, Celvive Inc., Cesca Therapeurics Inc., Celyad SA, Cyagen Biosciences Inc., Cynata Therapeucics Ltd., Cytori Therapeutics Inc., Escape Therapeutics Inc., Future Health BioBank, Gamida Cell Ltd., Zen-Bio Inc.

and more company profiles.

Interest in therapeutic applications of human MSCs arises from their diverse ability to differentiate into a range of cell types, as well as their ability to migrate to sites of tissue injury/inflammation or tumor growth. Additionally, MSCs well-suited for use in the exponential growth area of 3D printing, because of their capacity to form structural tissues.

Download Free Sample at http://www.reportsnreports.com/contactname=10598

Growing attention is now being given to manufacturing technologies to support commercial-scale production of MSCs. Numerous market competitors are also exploring commercialization strategies for MSC-derived exosomes, because exosomes represent a novel strategy for accessing the therapeutic effects of stem cells without the risks and difficulties of administering the cells to patients.

As the most common stem cell type being used in regenerative medicine, there is substantial potential for growth within the MSC market. Today, there are more than 40,000 scientific publications published about the cell type , more than 800 clinical trials underway worldwide , and Google Trend data reveals that MSC searches are more than twice as common as the next most common adult stem cell type .

The Burgeoning Market for Mesenchymal Stem Cells (MSCs)

Mesenchymal stem cells (MSCs) are the most utilized cell type within the regenerative medicine industry. To support this rapidly expanding marketplace

The report describes the current status of mesenchymal stem cell (MSC) research, ongoing clinical trials involving MSCs, late stage MSC clinical trials, and uses of MSCs in cell therapy. It explores recent advances in MSC products and technologies, identifies research priorities by market segment, and assesses 73 leading competitors within the MSC marketplace.

Direct Accessthis Mesenchymal Stem Cells Market (MSCs) report at http://www.reportsnreports.com/purchasname=10598

Because MSC therapy is an integrated component of Advanced Therapy Medicinal Products (ATMPs), this market report also provides an introduction to ATMPs.

For pharmaceutical companies, the report reveals how advances in MSC research can reveal potential new drug targets, improve methods of drug delivery, and provide personalized treatment strategies. With Big Pharma diversifying their product development pipelines by investing in cell therapy companies, MSC companies now represent promising candidates for collaboration, investment, and acquisition.

Based in Washington, DC, Bio Informant is first and only market research firm to specialize in the stem cell industry. Unlike other publishers that hire analysts from foreign countries, Bio Informant is an American-based company with more than a decade of experience with tracking the stem cell market (2006 to present).

Bio Informant compiled this global strategic report using interviews with more than 90 individuals from across the stem cell industry.

Bio Informant conducted interviews with representatives from Cynata Therapeutics (first company to bring a iPSC-derived MSC therapeutic product into a clinical trial), Rooster Bio (global leader in MSC manufacturing technology that can produce to tens of billions of cells in suspension bioreactors), Pluristem Therapeutics (commercializing placenta-derived mesenchymal-like adherent stromal cells in late-stage clinical trials), Bio Eden (leading company preserving MSCs derived from dental tissues), Regenexx (worlds leading provider of MSC therapies for orthopedic applications), and many more.

The report also incorporates secondary findings from SEC filings, company websites, press releases, investor presentations, government policy documents, Google Trends, and Google Adwords.

Browse Complete Report at http://www.reportsnreports.com/contactname=10598

Claim this report to:

Table of Contents

List of Figures.

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Autolus Therapeutics presents AUTO1 and AUTO3 data at the 2020 EHA25 Virtual Congress – BioSpace

Pivotal AUTO1 study in adult ALL patients enrolling

Conference Call and Webcast to be held Friday, June 12, 2020 at 7:30 am EDT / 12:30 pm BST

LONDON, June 12, 2020 (GLOBE NEWSWIRE) -- Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, today announced new data highlighting progress on its AUTO1 program, the companys CAR T cell therapy being investigated in the ongoing ALLCAR Phase 1 study of relapsed / refractory adult B-Acute Lymphocytic Leukemia (ALL), at the European Hematology Association EHA25 Virtual Congress beginning June 11.

AUTO1 in ALLAs of the data cut-off date of May 13, 2020, 19 patients had received AUTO1. AUTO1 was well tolerated, with no patients experiencing Grade 3 CRS. Three patients (16%) with high leukemia burden (>50% blasts) experienced Grade 3 neurotoxicity that resolved swiftly with the application of steroids. Of the 19 patients, 16 (84%) achieved MRD-negative CR. Two out of 16 patients received a transplant while in remission and CD19-negative relapse occurred in 3 (16%) patients. Durability of remissions is encouraging. Event Free Survival (EFS) and Overall Survival (OS) at 6 months are 62% and 72% respectively in all patients, and 76% and 92% respectively in the 13 patients treated with the closed (commercial) process. Median EFS and OS has not been reached, at a median follow up of 12.2 months (range up to 24.4 months).

I am very encouraged by the tolerable safety profile and high level of sustained CRs we have observed with AUTO1 in the ALLCAR19 study that was achieved without subsequent stem cell transplant, said Dr. Claire Roddie, Consultant Hematologist, UCL Cancer Institute and University College London Hospital.

Approximately 60% of adult ALL patients relapse or are refractory to first line therapy and there continues to exist a high unmet need, said Dr. Michael Bishop, MD, Professor of Medicine and Director of the Cellular Therapy Program at University of Chicago Medicine. AUTO1 is a novel CD19 CAR T candidate with a compelling activity and safety profile and has the potential to change standard of care as a curative therapy for r/r ALL.

The data update on AUTO1 presented at this years EHA meeting show an encouraging durability of response without subsequent stem cell transplant and confirm the positive safety profile, said Dr. Christian Itin, chairman and chief executive officer of Autolus. We have started enrolment of patients with r/r aALL in our pivotal Phase 1b/2 AUTO1-AL1 study.

AUTO3 in DLBCLDr. Wendy Osborne presented ALEXANDER Phase 1/2 clinical trial data for AUTO3. This data is consistent with our update on May 29, 2020, with a data cut-off date of April 27, 2020.

These data are very encouraging, in terms of safety and tolerability, with a high level of clinical activity, said Dr. Wendy Osborne, Consultant Hematologist, Freeman Hospital, Newcastle upon Tyne Hospitals NHS Foundation Trust. We are looking forward to enrolling additional patients in the outpatient cohort.

Dr. Wendy Osborne of Newcastle upon Tyne Hospitals NHS Foundation Trust also discusses AUTO3 data during the American Society of Clinical Oncology (ASCO) Annual Meeting in this video courtesy of the Lymphoma Hub.

Investor call on Friday June 12, 2020Management will host a conference call and webcast at 7:30 am EDT/12:30 pm BST to discuss the EHA data. To listen to the webcast and view the accompanying slide presentation, please go to: https://www.autolus.com/investor-relations/news-and-events/events.

The call may also be accessed by dialing (866) 679-5407 for U.S. and Canada callers or (409) 217-8320 for international callers. Please reference conference ID 4838626. After the conference call, a replay will be available for one week. To access the replay, please dial (855) 859-2056 for U.S. and Canada callers or (404) 537-3406 for international callers. Please reference conference ID 4838626.

About Autolus Therapeutics plcAutolus is a clinical-stage biopharmaceutical company developing next-generation, programmed T cell therapies for the treatment of cancer. Using a broad suite of proprietary and modular T cell programming technologies, the company is engineering precisely targeted, controlled and highly active T cell therapies that are designed to better recognize cancer cells, break down their defense mechanisms and eliminate these cells. Autolus has a pipeline of product candidates in development for the treatment of hematological malignancies and solid tumors. For more information please visit http://www.autolus.com.

About AUTO1 AUTO1 is a CD19 CAR T cell investigational therapy designed to overcome the limitations in safety - while maintaining similar levels of efficacy - compared to current CD19 CAR T cell therapies. Designed to have a fast target binding off-rate to minimize excessive activation of the programmed T cells, AUTO1 may reduce toxicity and be less prone to T cell exhaustion, which could enhance persistence and improve the ability of the programmed T cells to engage in serial killing of target cancer cells. AUTO1 is currently being evaluated in two Phase 1 studies, one in pediatric ALL and one in adult ALL. The company has also now progressed the program to a potential pivotal study, AUTO1-AL1.

About AUTO1-AL1 pivotal studyThe AUTO1-AL1 study will enroll patients with relapsed / refractory ALL. The study will have a short Phase1b component prior to proceeding to a single arm Phase 2 study. The primary end point is overall response rate and the key secondary end points include duration of response MRD negative CR rate and safety. The study will enroll approximately 100 patients across 30 of the leading academic and non-academic centers in the US, UK and Europe.

About AUTO3AUTO3 is a programmed T cell therapy containing two independent chimeric antigen receptors targeting CD19 and CD22 that have each been independently optimized for single target activity. By simultaneously targeting two B cell antigens, AUTO3 is designed to minimize relapse due to single antigen loss in patients with B cell malignancies. AUTO3 is currently being tested in diffuse large B cell lymphoma in the ALEXANDER clinical trial, with a 20-patient cohort that was initiated in Q2 2020 to assess feasibility of treatment in an outpatient setting.

Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are statements that are not historical facts, and in some cases can be identified by terms such as "may," "will," "could," "expects," "plans," "anticipates," and "believes." These statements include, but are not limited to, statements regarding Autolus financial condition and results of operations, including its expected cash runway; the development of Autolus product candidates, including statements regarding the timing of initiation, completion and the outcome of pre-clinical studies or clinical trials and related preparatory work, and the periods during which the results of the studies and trials will become available; Autolus plans to research, develop, manufacture and commercialize its product candidates; the potential for Autolus product candidates to be alternatives in the therapeutic areas investigated; and Autolus manufacturing capabilities and strategy. Any forward-looking statements are based on management's current views and assumptions and involve risks and uncertainties that could cause actual results, performance or events to differ materially from those expressed or implied in such statements. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section titled "Risk Factors" in Autolus' Annual Report on Form 20-F filed with the Securities and Exchange Commission on March 3, 2020 as well as discussions of potential risks, uncertainties, and other important factors in Autolus' future filings with the Securities and Exchange Commission from time to time. All information in this press release is as of the date of the release, and the company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events, or otherwise, except as required by law.

Contact:

Lucinda Crabtree, PhDVice President, Investor Relations and Corporate Communications+44 (0) 7587 372 619l.crabtree@autolus.com

Julia Wilson+44 (0) 7818 430877j.wilson@autolus.com

Susan A. NoonanS.A. Noonan Communications+1-212-966-3650susan@sanoonan.com

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Autolus Therapeutics presents AUTO1 and AUTO3 data at the 2020 EHA25 Virtual Congress - BioSpace

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Genmab Announces European Marketing Authorization for the Subcutaneous Formulation of DARZALEX (daratumumab) for the Treatment of Patients with Multi…

Copenhagen, Denmark; June 4, 2020 Genmab A/S (Nasdaq: GMAB) announced today that the European Commission (EC) has granted marketing authorization for the subcutaneous (SC) formulation of DARZALEX (daratumumab), for the treatment of adult patients with multiple myeloma in all currently approved daratumumab intravenous (IV) formulation indications in frontline and relapsed / refractory settings. The approval follows a Positive Opinion by the CHMP of the European Medicines Agency (EMA) in April 2020. The SC formulation is administered as a fixed-dose over approximately three to five minutes, significantly less time than IV daratumumab, which is given over several hours. Patients currently on daratumumab IV will have the choice to switch to the SC formulation. In August 2012, Genmab granted Janssen Biotech, Inc. (Janssen) an exclusive worldwide license to develop, manufacture and commercialize daratumumab.

We are extremely pleased that patients in Europe with multiple myeloma will now, like patients in the U.S., have the opportunity for treatment with the subcutaneous formulation of daratumumab. With consistent efficacy, and greater convenience for patients and health care providers with dosing time reduced from hours to just minutes and fewer infusion-related reactions, this formulation provides significant benefits for patients, said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab

The approval was based on data from two studies: the Phase III non-inferiority COLUMBA (MMY3012) study, which compared the SC formulation of daratumumab to the IV formulation in patients with relapsed or refractory multiple myeloma, and data from the Phase II PLEIADES (MMY2040) study, which is evaluating SC daratumumab in combination with certain standard multiple myeloma regimens. The topline results from the COLUMBA study were announced in February 2019 and subsequently presented in oral sessions at the 2019 American Society of Clinical Oncology (ASCO) Annual Meeting and the 24th European Hematology Association (EHA) Annual Congress. Updated data of the COLUMBA and the PLEIADES studies were presented during poster sessions at the 61st American Society of Hematology (ASH) Annual Meeting in December 2019.

About the COLUMBA (MMY3012) studyThe Phase III trial (NCT03277105) is a randomized, open-label, parallel assignment study that included 522 adults diagnosed with relapsed and refractory multiple myeloma. Patients were randomized to receive either: SC daratumumab, as 1800 mg daratumumab with rHuPH20 2000 U/mL once weekly in Cycle 1 and 2, every two weeks in Cycles 3 to 6, every 4 weeks in Cycle 7 and thereafter until disease progression, unacceptable toxicity or the end of study; or 16 mg/kg IV daratumumab once weekly in Cycle 1 and 2, every two weeks in Cycles 3 to 6, every 4 weeks in Cycle 7 and thereafter until disease progression, unacceptable toxicity or the end of study. The co-primary endpoints of the study are overall response rate and Maximum trough concentration of daratumumab (Ctrough; defined as the serum pre-dose concentration of daratumumab on Cycle 3 Day 1).

About the PLEIADES (MMY2040) studyThe Phase II trial (NCT03412565) is a non-randomized, open-label, parallel assignment study that includes 265 adults either newly diagnosed or with relapsed or refractory multiple myeloma. Patients with newly diagnosed multiple myeloma are being treated with 1,800 mg SC daratumumab in combination with either bortezomib, lenalidomide and dexamethasone (D-VRd) or bortezomib, melphalan and prednisone (D-VMP). Patients with relapsed or refractory multiple myeloma are being treated with 1,800 mg SC daratumumab plus lenalidomide and dexamethasone (D-Rd). An additional cohort of patients with relapsed and refractory multiple myeloma treated with daratumumab plus carfilzomib and dexamethasone (D-Kd) was subsequently added to the study. The primary endpoint for the D-VMP, D-Kd and D-Rd cohorts is overall response rate. The primary endpoint for the D-VRd cohort is very good partial response or better rate.

About DARZALEX(daratumumab)DARZALEX (daratumumab) intravenous infusion is indicated for the treatment of adult patients in the United States: in combination with bortezomib, thalidomide and dexamethasone as treatment for patients newly diagnosed with multiple myeloma who are eligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with bortezomib, melphalan and prednisone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of patients with multiple myeloma who have received at least one prior therapy; in combination with pomalidomide and dexamethasone for the treatment of patients with multiple myeloma who have received at least two prior therapies, including lenalidomide and a proteasome inhibitor (PI); and as a monotherapy for the treatment of patients with multiple myeloma who have received at least three prior lines of therapy, including a PI and an immunomodulatory agent, or who are double-refractory to a PI and an immunomodulatory agent.1 DARZALEX is the first monoclonal antibody (mAb) to receive U.S. Food and Drug Administration (U.S. FDA) approval to treat multiple myeloma.

DARZALEX is indicated for the treatment of adult patients in Europe via intravenous infusion or subcutaneous administration: in combination with bortezomib, thalidomide and dexamethasone as treatment for patients newly diagnosed with multiple myeloma who are eligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with bortezomib, melphalan and prednisone for the treatment of adult patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; for use in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least one prior therapy; and as monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma, whose prior therapy included a PI and an immunomodulatory agent and who have demonstrated disease progression on the last therapy2. Daratumumab is the first subcutaneous CD38-directed antibody approved in Europe for the treatment of multiple myeloma. The option to split the first infusion of DARZALEX over two consecutive days has been approved in both Europe and the U.S.

In Japan, DARZALEX intravenous infusion is approved for the treatment of adult patients: in combination with lenalidomide and dexamethasone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with bortezomib, melphalan and prednisone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone for the treatment of relapsed or refractory multiple myeloma. DARZALEX is the first human CD38 monoclonal antibody to reach the market in the United States, Europe and Japan. For more information, visit http://www.DARZALEX.com.

DARZALEX FASPRO (daratumumab and hyaluronidase-fihj), a subcutaneous formulation of daratumumab, is approved in the United States for the treatment of adult patients with multiple myeloma: in combination with bortezomib, melphalan and prednisone in newly diagnosed patients who are ineligible for ASCT; in combination with lenalidomide and dexamethasone in newly diagnosed patients who are ineligible for ASCT and in patients with relapsed or refractory multiple myeloma who have received at least one prior therapy; in combination with bortezomib and dexamethasone in patients who have received at least one prior therapy; and as monotherapy, in patients who have received at least three prior lines of therapy including a PI and an immunomodulatory agent or who are double-refractory to a PI and an immunomodulatory agent.3 DARZALEX FASPRO is the first subcutaneous CD38-directed antibody approved in the U.S. for the treatment of multiple myeloma.

Daratumumab is a human IgG1k monoclonal antibody (mAb) that binds with high affinity to the CD38 molecule, which is highly expressed on the surface of multiple myeloma cells. Daratumumab triggers a persons own immune system to attack the cancer cells, resulting in rapid tumor cell death through multiple immune-mediated mechanisms of action and through immunomodulatory effects, in addition to direct tumor cell death, via apoptosis (programmed cell death).1,4,5,6,7

Daratumumab is being developed by Janssen Biotech, Inc. under an exclusive worldwide license to develop, manufacture and commercialize daratumumab from Genmab. A comprehensive clinical development program for daratumumab is ongoing, including multiple Phase III studies in smoldering, relapsed and refractory and frontline multiple myeloma settings. Additional studies are ongoing or planned to assess the potential of daratumumab in other malignant and pre-malignant diseases in which CD38 is expressed, such as amyloidosis and T-cell acute lymphocytic leukemia (ALL). Daratumumab has received two Breakthrough Therapy Designations from the U.S. FDA for certain indications of multiple myeloma, including as a monotherapy for heavily pretreated multiple myeloma and in combination with certain other therapies for second-line treatment of multiple myeloma.

About Genmab Genmab is a publicly traded, international biotechnology company specializing in the creation and development of differentiated antibody therapeutics for the treatment of cancer. Founded in 1999, the company is the creator of three approved antibodies: DARZALEX (daratumumab, under agreement with Janssen Biotech, Inc.) for the treatment of certain multiple myeloma indications in territories including the U.S., Europe and Japan, Arzerra (ofatumumab, under agreement with Novartis AG), for the treatment of certain chronic lymphocytic leukemia indications in the U.S., Japan and certain other territories and TEPEZZA (teprotumumab, under agreement with Roche granting sublicense to Horizon Therapeutics plc) for the treatment of thyroid eye disease in the U.S. A subcutaneous formulation of daratumumab, DARZALEX FASPRO (daratumumab and hyaluronidase-fihj), has been approved in the U.S. for the treatment of adult patients with certain multiple myeloma indications. Daratumumab is in clinical development by Janssen for the treatment of additional multiple myeloma indications, other blood cancers and amyloidosis. A subcutaneous formulation of ofatumumab is in development by Novartis for the treatment of relapsing multiple sclerosis. Genmab also has a broad clinical and pre-clinical product pipeline. Genmab's technology base consists of validated and proprietary next generation antibody technologies - the DuoBody platform for generation of bispecific antibodies, the HexaBody platform, which creates effector function enhanced antibodies, the HexElect platform, which combines two co-dependently acting HexaBody molecules to introduce selectivity while maximizing therapeutic potency and the DuoHexaBody platform, which enhances the potential potency of bispecific antibodies through hexamerization. The company intends to leverage these technologies to create opportunities for full or co-ownership of future products. Genmab has alliances with top tier pharmaceutical and biotechnology companies. Genmab is headquartered in Copenhagen, Denmark with sites in Utrecht, the Netherlands, Princeton, New Jersey, U.S. and Tokyo, Japan.

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Genmab Announces European Marketing Authorization for the Subcutaneous Formulation of DARZALEX (daratumumab) for the Treatment of Patients with Multi...

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