Chris Christo/MediaNews Group/Boston Herald via Getty Images
Vertex Pharmaceuticals has expanded its collaborativepartnership with CRISPR Therapeutics to develop and commercialize a possible cure for sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). The focus is on the development of CTX001, an autologous, ex vivo CRISPR-CAS9 gene-edited therapy.
Under the terms of the expanded deal, Vertex will be responsible for60% of the costs of developing, manufacturing and commercializing CTX001 with support from CRISPR Therapeutics, and will receive 60% of profits from global sales. This is a 10% increase over the previous agreement. CRISPR will handle the remaining 40% and receive 40% of profits. Vertex is paying CRISPR $900 million up front with another $200 million possible after the first regulatory approval for the therapy.
Working with Vertex, we have made tremendous progress with CTX001, the first CRISPR-Cas9-based therapy to demonstrate proof of concept in the clinic and together we have broken new ground in the treatment of genetic diseases, said Samarth Kulkarni, chief executive officer of CRISPR Therapeutics. We have now dosed more than 30 patients with CTX001, with longest follow-up beyond two years, and we are on track to complete enrollment in both clinical trials this year.
Kulkarni went on to say that with the results and momentum, we are adopting a new operating model to enable a globally coordinated launch of CTX001, leveraging Vertexs best-in-class global capabilities and leadership in development, manufacturing, and commercialization to enable this medicine to reach all patients that can benefit from it as quickly as possible. We remain deeply committed to the Sickle Cell and Thalassemia patient communities and look forward to continued success in our partnership with Vertex.
Based on the CTX001s data generated so far, it has received Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Diseases designations from the U.S. Food and Drug Administration (FDA) for both TDT and SCD. It has also received Orphan Drug Designation for both indications from the European Commission, and Priority Medicines (PRIME) designation from the European Medicines Agency (EMA) for SCD.
In December 2020, the two companies announced new data on a total of 10 patients receiving CTX001, and it demonstrated a consistent and sustained response. All seven patients with TDT, including three with either a severe or bo/bo genotype, were free of transfusions at their last follow-up and all three patients with SCD were free of vaso-occlusive crises (VOCs) from CTX001 infusion through their last follow-up.
With this therapy, patients hematopoietic stem and progenitor cells are collected from peripheral blood. They are then edited using CRISPR-Cas9 gene editing. The edited cells are CTX001, and are infused back into the patient as part of a stem cell transplant. The editing is designed to produce high levels of fetal hemoglobin (HbF) in red blood cells. HbF is a type of oxygen-carrying hemoglobin naturally present at birth, but then switches to an adult form of hemoglobin. The increased levels of HbF by CTX001 can decrease the need for blood transfusions for patients with TDT and decrease the painful and debilitating sickle crises that patients with SCD experience.
Two months later, their primary competitor in this space, bluebird bio, suspended its Phase I/II and Phase III trials of LentiGlobin gene therapy for SCD after Suspected Unexpected Serious Adverse Reactions (SUSARs). This came after a patient from Cohort A of the Phase I/II study was diagnosed with acute myelogenous leukemia (AML), who had received the therapy five years earlier. Also, a patient from Cohort C of the same study was diagnosed with myelodysplastic syndrome (MDS).
On March 10, the company indicated its investigations found that the side effects were unlikely to be linked to their gene therapy. As of April 20, the program was still on clinical hold in the U.S.
So Vertex and CRISPR found themselves in a position of not only having what appeared to be a very effective therapy, but a good shot at being the first approved cure for sickle cell disease.
Vertex and CRISPR are on schedule to fully enroll both trials for beta thalassemia and sickle cell disease later this year and if the data continues to be positive, should be able to file with regulators in 18 to 24 months.
Jeffrey Leiden, executive chairman of Vertex, stated, Cell and genetic therapies are key to our strategy of developing transformative therapies for serious diseases, and this agreement is an important next step in cementing our leadership in these modalities as we bring forward our broad gene and cell-based therapeutics portfolio. As we take the lead on CTX001, we want to acknowledge the foundational contributions by the team at CRISPR Therapeutics. Our increased investment in our partnership with CRISPR is based on the compelling clinical profile of CTX001, which shows its potential to be a durable cure for patients with SCD and TDT, and the rapid progress that we and our partners at CRISPR have made toward registration and commercialization.
- FDA Responds to Creative Medical Technology Holdings Regarding Its ImmCelz IND for Stroke Treatment - KPVI News 6 - May 14th, 2021
- Duluth health center specializes in regenerative medicine, hormone replacement therapy - Duluth News Tribune - May 14th, 2021
- Dr. Krger on Toxicities Associated With Allogeneic Stem Cell Transplant in Myeloma - OncLive - May 14th, 2021
- Magenta Therapeutics Announces Positive Preliminary Results from Phase 2 Clinical Trial of MGTA-145 in Multiple Myeloma and Provides an Update on its... - May 14th, 2021
- Genenta Phase I/II Glioblastoma Data at ASGCT Show Temferon Delivered Tumor-Focused Interferon ExpressionData presented at the 2021 American Society... - May 14th, 2021
- ViaCyte To Participate In World Medical Innovation Forum Panel On Future Of Cell Therapy For Type 1 Diabetes - PRNewswire - May 14th, 2021
- Omeros' Narsoplimab Pivotal Trial Data to Be Shared as an Oral Presentation at the European Hematology Association Congress - Business Wire - May 14th, 2021
- Magenta Therapeutics to Participate in Upcoming Healthcare Investor Conferences in May - Business Wire - May 14th, 2021
- Outlook for multiple myeloma: Figures and factors that affect it - Medical News Today - May 14th, 2021
- Global Regenerative Medicine for Cartilage Market Report 2021: Companies are Continuously Focusing on Introducing New Technologies to Survive in the... - May 14th, 2021
- UB celebrates 175th anniversary of its origins in study of medicine - UB Now: News and views for UB faculty and staff - University at Buffalo Reporter - May 14th, 2021
- Stem cell therapy for COVID-19 and ARDS - News-Medical.Net - May 7th, 2021
- Experimental COVID-19 lung treatment being tested at Ontario hospitals - CityNews Toronto - May 7th, 2021
- EP. 4: Optimizing Patient Selection for CAR T-Cell Therapy - OncLive - May 7th, 2021
- Could stem cells improve the outcome of ARDS in severe COVID-19? - News-Medical.Net - May 7th, 2021
- Motixafortide Reaches Primary End Point of Improved Stem Cell Mobilization in Multiple Myeloma Trial - Cancer Network - May 7th, 2021
- Restricting the Growth and Spread of Head and Neck Cancers - Technology Networks - May 7th, 2021
- The global stem cell therapy market is projected to reach USD 401 million by 2026 from - GlobeNewswire - May 7th, 2021
- UCLA team discovers how to restrict growth, spread of head and neck cancers | UCLA - UCLA Newsroom - May 7th, 2021
- NUS scientists found a key element that affects how genes are expressed in blood stem cells - Newswise - May 7th, 2021
- Citius Pharmaceuticals to Present Mino-Lok and COVID-related Respiratory Treatment Update at Benzinga Global Small Cap Conference on Thursday, May 13... - May 7th, 2021
- Mesoblast says its stem cell treatment saved lives of severely ill COVID patients - Stockhead - May 2nd, 2021
- Antiviral T cells safe, effective as off-the-shelf therapy for painful complication after stem cell transplants - News-Medical.Net - May 2nd, 2021
- Antiviral T cells protected, viable as off-the-shelf treatment for painful complication stem cells - Microbioz India - May 2nd, 2021
- Desperate family of boy, 9, with leukaemia have 10 days to save his life... - The Sun - May 2nd, 2021
- Orchard Therapeutics Announces Multiple Presentations at the 24th Annual Meeting of the American Society of Gene & Cell Therapy - GlobeNewswire - May 2nd, 2021
- Investigating CAR T-Cell Therapy for Use in Different Disease Types - Targeted Oncology - May 2nd, 2021
- Enhanced inhibition of tumor growth using TRAIL-overexpressing adipose-derived stem cells in combination with the chemotherapeutic agent CPT-11 in... - May 2nd, 2021
- Thomas Smeenk on Hemostemix's autologous stem cell therapy technology and why some call it 'the fountain of youth' - InvestorIntel - April 28th, 2021
- Monitoring levels of vimentin-positive circulating cancer stem cells and tumor cells in patients with advanced EGFR-mutated non-small cell lung cancer... - April 28th, 2021
- Fate Therapeutics Announces Four Presentations at the 2021 ASGCT Annual Meeting - GlobeNewswire - April 28th, 2021
- Therapeutic Solutions International Reports Regression of Established Tumors by Combining its Cancer Blood Vessel Targeting Immunotherapy with Low... - April 28th, 2021
- A review of baculovirus vectors in gene therapy | BTT - Dove Medical Press - April 28th, 2021
- Stem Cell Therapy for COPD: Hope and Exploitation - DocWire News - April 26th, 2021
- Axion BioSystems Announces Formation of Scientific Advisory Board - Business Wire - April 26th, 2021
- Global Cell Therapy Market By Therapy Type, By Therapeutic Area, By End User, By Cell Type, By Regional Outlook, Industry Analysis Report and... - April 24th, 2021
- Pure Red Cell Aplasia following ABO-Mismatched Allogeneic Hematopoietic Stem Cell Transplantation: Resolution with Daratumumab Treatment - DocWire... - April 24th, 2021
- Advancing the Development of Safe and Effective Regenerative Medicine Products | FDA - FDA.gov - April 24th, 2021
- New method helps map the specialized diversity and spatial location of cells within a tissue or tumor - News-Medical.Net - April 24th, 2021
- Chimeric embryo may have medical implications The Campus - The Campus - April 24th, 2021
- Survival and treatment patterns of patients with relapsed or refractory multiple myeloma in France - a cohort study using the French National... - April 24th, 2021
- Guenther Koehne, MD, Highlights Recent Advancements in the Treatment of Blood Cancers - Oncology Learning Network - April 24th, 2021
- Rinri Therapeutics raises 10 million to advance stem cell therapy for hearing loss - The Star - April 24th, 2021
- Cancer Diagnosis in Patient Given LentiGlobin Revised to Severe Anemia - Sickle Cell Anemia News - April 24th, 2021
- Hodgkin lymphoma: Symptoms, outlook, treatment, and more - Medical News Today - April 24th, 2021
- Tissue Repair, Mitochondrial Function and Wound Healing Explored in 2021 APS Presidents Symposium - Newswise - April 24th, 2021
- Axion BioSystems Announces Formation of Scientific Advisory Board - BioSpace - April 20th, 2021
- Avalon GloboCare is seeking to tap the power of cells to fight cancer and coronavirus - Proactive Investors USA - April 20th, 2021
- Rinri Therapeutics Raises 10 million from Existing Investors and UK Future Fund to Advance its Novel Stem Cell Therapy to Restore Hearing Loss -... - April 20th, 2021
- Global Regenerative Medicines Market 2020-2030: Opportunities in Drug Approvals and Strong Pipeline of Cell and Gene Therapies & Multiple... - April 20th, 2021
- FDA Grants Orphan Drug Status to Novel IRAK4 Inhibitor in AML and MDS - Targeted Oncology - April 20th, 2021
- Kaleido Biosciences Announces Collaboration with Robert Jenq, MD, to Explore Potential of its Microbiome Metabolic Therapies (MMT) in Preventing... - April 20th, 2021
- Partner Content | Being bionic: the future of regenerative medicine - TheSpec.com - April 14th, 2021
- Ohio State Recruits Top Oncologist to Expand Cell Therapy Program, Lead Bone Marrow Transplant Services - Wexner Medical Center - The Ohio State... - April 14th, 2021
- Leukemia Cutis: Symptoms and Treatment - Healthline - April 14th, 2021
- Green: Variants and outbreaks continue to put stress on hospitals - Vermont Biz - April 14th, 2021
- CRISPR Therapies Pipeline Insights 2021: Analysis of Key Companies, Emerging Therapies, Recent Happenings and Futuristic Trends - GlobeNewswire - April 14th, 2021
- Consortium to investigate role of neurofilament light chain in neurodegenerative diseases Washington University School of Medicine in St. Louis -... - April 14th, 2021
- Man's heart healed by stem cell therapy and love of an old flame - Leeds Live - April 12th, 2021
- New pediatric cancer treatment method being used in Iran - Tehran Times - April 12th, 2021
- Personalized Cancer Vaccine Shows Early Efficacy in Patients With Diverse Tumor Types - Technology Networks - April 12th, 2021
- 1000th Stem Cell Transplant Given to Girl Who Is One in a Million - Stanford Children's Health Blog - Stanford Children's Health - April 10th, 2021
- Home : Stem Cell Treatments - April 10th, 2021
- Researchers discover unique regenerative property of cells in the early human embryo - News-Medical.net - April 10th, 2021
- Study reveals sex-differences in aging of the brain stem cell niche - News-Medical.net - April 10th, 2021
- Antibiotics need further study in cancer treatments - Chicago Daily Herald - April 10th, 2021
- Prolonged infection and inflammation drain immune responses as we age - Baylor College of Medicine News - April 10th, 2021
- Toward a transformative therapy for sickle cell patients - News-Medical.Net - April 10th, 2021
- Mass. Residents 55+ Or With 1 Medical Condition Can Now Get COVID Vaccine | WBZ NewsRadio 1030 - iHeartRadio - April 6th, 2021
- Mortality from Multiple Myeloma Within One Year Following Autologous Stem Cell Transplantation: Defining an Ultra-high Risk Population - DocWire News - April 6th, 2021
- BioRestorative Therapies Announces Appointment of Nickolay V. Kukekov, Ph.D to its Board of Directors - BioSpace - April 6th, 2021
- LGL Leukemia: Overview, Symptoms, and Treatment - Healthline - April 3rd, 2021
- Novel T-Cell Therapy MT-401 Under Evaluation in Post-Transplant AML - OncLive - April 3rd, 2021
- Funding the Next Generation of Cancer Therapies - Genetic Engineering & Biotechnology News - April 3rd, 2021
- Do Therapies for Alzheimer's & Parkinson's that Clear Abnormal Brain Proteins Make the Diseases Worse? - BioSpace - April 3rd, 2021
- Allogeneic hematopoietic stem cell transplantation for therapy-related myeloid neoplasms following treatment of a lymphoid malignancy - DocWire News - April 1st, 2021
- Insulin 100: How the road to a diabetes cure is yielding better treatments - News@UofT - April 1st, 2021
- The Government's Watchful Eye on Fraud Stemming from Stem Cell Therapy - Lexology - April 1st, 2021
- Introducing a neuro regenerative gene into mice with spinal cord injury improves motor function - News-Medical.net - April 1st, 2021
- Gracell Biotechnologies Announces Enrollment of First Patient in Registrational Phase 1/2 Clinical Study for GC007g, an Allogeneic CAR-T Cell Therapy... - April 1st, 2021