CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces clinical data presentations at the upcoming 24thAmerican Society of Gene and Cell Therapy (ASGCT) Annual Meeting taking place May 11-14, 2021. Investigators will review new data from Rockets Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), and Fanconi Anemia (FA) gene therapy programs in oral and poster presentations.
Details for oral presentations are as follows:
Title: A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim ResultsSession: Genetic Blood and Immune DisordersPresenter: Donald Kohn, M.D., Professor of Microbiology, Immunology and Molecular Genetics, Pediatrics (Hematology/Oncology), Molecular and Medical Pharmacology, and member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at the University of California, Los AngelesDate: Tuesday May 11, 2021Time: 6:15-6:30 p.m. EDTLocation: Room 7Abstract number: 39
Title: Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: Updated Results of a Global Phase 1 Study for Adult and Pediatric PatientsSession: Gene Therapies for HemoglobinopathiesPresenter: Jos Luis Lpez Lorenzo, M.D., Hospital Universitario Fundacin Jimnez Daz, Madrid, SpainDate: Wednesday May 12, 2021Time: 6:45-7:00 p.m. EDTLocation: Room 7Abstract number: 83
Title: Gene Therapy in Fanconi Anemia: Current Strategies to Enable the Correction of HSCsSession: International Focus on Stem Cell Gene Therapy Presenter: Juan A. Bueren, Ph.D., Head of the Hematopoietic Innovative Therapies Division at the Centro de Investigaciones Energticas, Medioambientales y Tecnolgicas (CIEMAT) in Spain / CIBER-Rare Diseases / IIS-Fundacin Jimnez DazDate: Thursday, May 13, 2021Time: 10:00-10:45 a.m. EDTLocation: Room 7Abstract number: 36
Select results from Dr. Buerens presentation will also be highlighted by Paula Rio, Ph.D. Details for this Invited Presentation are as follows:
Title: Gene Therapy in Fanconi Anemia: Current Strategies to Enable the Correction of HSCsSession: International Focus on Stem Cell Gene TherapyPresenter: Paula Ro, Ph.D., Senior Researcher, Hematopoietic Innovative Therapies Division at CIEMAT in Spain / CIBER-Rare Diseases / IIS-Fundacin Jimnez DazDate: Thursday May 13, 2021Time: 10:00-11:45 a.m. EDT
Details for poster presentation are as follows:
Title: Gene Therapy for Fanconi Anemia [Group A]: Preliminary Results of Ongoing RP-L102 Clinical TrialsSession: Hematologic and Immunologic DiseasesPresenter: Agnieszka Czechowicz, M.D., Ph.D., Assistant Professor of Pediatrics, Division of Stem Cell Transplantation, Stanford University School of MedicineDate: Tuesday, May 11, 2021Time: 8:00-10:00 a.m. EDTLocation: Digital GalleryAbstract number: 697
Abstracts for the presentations can be found online at: https://annualmeeting.asgct.org/
About Leukocyte Adhesion Deficiency-I
Severe Leukocyte Adhesion Deficiency-I (LAD-I) is a rare, autosomal recessive pediatric disease caused by mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18. CD18 is a key protein that facilitates leukocyte adhesion and extravasation from blood vessels to combat infections. As a result, children with severe LAD-I are often affected immediately after birth. During infancy, they suffer from recurrent life-threatening bacterial and fungal infections that respond poorly to antibiotics and require frequent hospitalizations. Children who survive infancy experience recurrent severe infections including pneumonia, gingival ulcers, necrotic skin ulcers, and septicemia. Without a successful bone marrow transplant, mortality in patients with severe LAD-I is 60-75% prior to the age of 2 and survival beyond the age of 5 is uncommon. There is a high unmet medical need for patients with severe LAD-I.
Rockets LAD-I research is made possible by a grant from the California Institute for Regenerative Medicine (Grant Number CLIN2-11480). The contents of this press release are solely the responsibility of Rocket and do not necessarily represent the official views of CIRM or any other agency of the State of California.
About Pyruvate Kinase Deficiency
Pyruvate kinase deficiency (PKD) is a rare, monogenic red blood cell disorder resulting from a mutation in the PKLR gene encoding for the pyruvate kinase enzyme, a key component of the red blood cell glycolytic pathway. Mutations in the PKLR gene result in increased red cell destruction and the disorder ranges from mild to life-threatening anemia. PKD has an estimated prevalence of 3,000 to 8,000 patients in the United States and the European Union. Children are the most commonly and severely affected subgroup of patients. Currently available treatments include splenectomy and red blood cell transfusions, which are associated with immune defects and chronic iron overload.
RP-L301 was in-licensed from the Centro de Investigaciones Energeticas, Medioambientales y Tecnologicas (CIEMAT), Centro de Investigacion Biomedica en Red de Enfermedades Raras (CIBERER) and Instituto de Investigacion Sanitaria Fundacion Jimenez Diaz (IIS-FJD).
About Fanconi Anemia
Fanconi Anemia (FA) is a rare pediatric disease characterized by bone marrow failure, malformations and cancer predisposition. The primary cause of death among patients with FA is bone marrow failure, which typically occurs during the first decade of life. Allogeneic hematopoietic stem cell transplantation (HSCT), when available, corrects the hematologic component of FA, but requires myeloablative conditioning. Graft-versus-host disease, a known complication of allogeneic HSCT, is associated with an increased risk of solid tumors, mainly squamous cell carcinomas of the head and neck region. Approximately 60-70% of patients with FA have a Fanconi Anemia complementation group A (FANCA) gene mutation, which encodes for a protein essential for DNA repair. Mutation in the FANCA gene leads to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Increased sensitivity to DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is a gold standard test for FA diagnosis. Somatic mosaicism occurs when there is a spontaneous correction of the mutated gene that can lead to stabilization or correction of a FA patients blood counts in the absence of any administered therapy. Somatic mosaicism, often referred to as natural gene therapy provides a strong rationale for the development of FA gene therapy because of the selective growth advantage of gene-corrected hematopoietic stem cells over FA cells.
About Rocket Pharmaceuticals, Inc.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders. The Companys platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, Pyruvate Kinase Deficiency (PKD), a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia, and Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. For more information about Rocket, please visit http://www.rocketpharma.com.
Rocket Cautionary Statement Regarding Forward-Looking Statements
Various statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding its guidance for 2021 in light of COVID-19, the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon Disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rockets ongoing trials, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Annual Report on Form 10-K for the year ended December 31, 2020, filed March 1, 2021 with the SEC. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.
Read more from the original source:
- 100 plus years of stem cell research20 years of ISSCR - PMC - March 26th, 2024
- Stem Cell Science and Human Research Studies Ahead of Cargo Arrival - NASA Blogs - February 18th, 2024
- Stem cell research project to launch into space - Fox Weather - January 24th, 2024
- Breakthrough in cancer research opening up stem cell therapy to more people. How you can get involved - 69News WFMZ-TV - January 20th, 2024
- Stem Cell Research Heading to the ISS on Axiom Mission 3 - ISS National Lab - January 18th, 2024
- No, Rep. Steve Scalise Didn't Vote Against Stem Cell Research From Which He Is Now Benefiting - The Dispatch - January 12th, 2024
- Applications are open for the Maryland Stem Cell Research Fund - Technical.ly - January 4th, 2024
- Global Stem Cell Therapy Market to Reach USD 928.6 Million by 2031: Says Allied Market Research - Yahoo Finance - November 19th, 2023
- Current state of stem cell-based therapies: an overview - PMC - November 3rd, 2023
- Dynamic Stem Cell Therapy Uncovers Research in Advance Regenrative Medicine - Yahoo Finance - November 3rd, 2023
- Research Fellow (Aging and Cancer Stem Cell Laboratory ... - Times Higher Education - October 15th, 2023
- Qkine Collaborates with the Cambridge Stem Cell Institute to Facilitate Same-Day Access to Key Research Products for Researchers at the Cambridge... - September 27th, 2023
- Stem cells: a comprehensive review of origins and emerging clinical ... - September 25th, 2023
- Stem Cell Research and Communicating Science | GBH - GBH News - September 20th, 2023
- Stem cell research reveals the earliest stages of a human life - SBS News - September 10th, 2023
- Stem Cell Therapy Market Size 2023 | Innovative Research Methodologies with Emerging Trends and Opportuni - Benzinga - September 10th, 2023
- Autologous Stem Cell and Non-Stem Cell Based Therapies Market Research, Current Trends, Key Industry Play - Benzinga - September 8th, 2023
- Stem Cell Therapy Market 2023 Business Statistics and Research ... - The Knox Student - August 28th, 2023
- Autologous Stem Cell Based Therapies Market Analysis, Research ... - Chatfield News-Record - July 19th, 2023
- Global Stem Cell Market Projected to Reach $14 Bn by 2028: Ken Research - Yahoo Finance - July 11th, 2023
- Theradaptive Awarded Manufacturing Assistance Grant by the Maryland Stem Cell Research Fund - Benzinga - July 10th, 2023
- Bionano Announces Presentation of OGM Utility Across Stem Cell Therapy Applications at the International Society for Stem Cell Research (ISSCR) Annual... - June 19th, 2023
- Sana Biotechnology Highlights Preclinical Data from Hypoimmune and Fusogen Platforms at the International Society for Stem Cell Research (ISSCR) 2023... - June 17th, 2023
- Induced Pluripotent Stem Cell (iPSC) Global Market Report 2023: Effective Research Programs Hold Key in Roll Out of Advanced iPSC Treatments - Yahoo... - June 17th, 2023
- Lung and heart stem cell research paves way for new COVID-19 treatments - Medical Xpress - June 14th, 2023
- Toxicology PhD student cultivating giant leaps in stem cell research ... - June 4th, 2023
- Harvard Stem Cell Institute (HSCI) - May 26th, 2023
- Findings may lead to improved insulin-secreting cells derived from stem ... - May 26th, 2023
- Cell Press: Stem Cell Reports - May 26th, 2023
- Stem cell research could enable blood to be made in other parts of the body - Medical Xpress - May 26th, 2023
- Construction of myocardial patch with mesenchymal stem cells and poly ... - May 22nd, 2023
- Cedars-Sinai to Send Stem Cells to the Space Station to Aid in the ... - May 22nd, 2023
- researchers expand human blood stem cells | Institute for Stem Cell ... - May 22nd, 2023
- A Look Inside Stem Cells Helps Create Personalized Regenerative ... - May 17th, 2023
- Exclusive Research Report on Msenchymal Stem Cell and Exosome Diagnostics and Therapies Market to Witness Comp - openPR - May 17th, 2023
- The Future of Stem Cell Research: Master of Science in ... - The Daily | Case Western Reserve University - May 10th, 2023
- Exclusive Research Report on Stem Cell Therapy for Diabetes and ... - Digital Journal - May 9th, 2023
- Aging melanocyte stem cells and gray hair | National Institutes of ... - May 5th, 2023
- Mouse hair turns gray when certain stem cells get stuck - May 5th, 2023
- Science-First Skincare Company Michal Morrison Secures Exclusive World-Wide License of Proprietary STEM6 Molecule, Supported by Over 25 Years of... - May 5th, 2023
- BioCentriq and panCELLa execute research agreement to study stem cell-derived Natural Killer cell expansi - Benzinga - May 3rd, 2023
- Hair turning gray? Study finds a stem cell 'glitch' may be the cause - May 1st, 2023
- Elevai Labs Announces Research Grant Award and Partnership to Better Characterize the 'Payload' of ELEVAI's Stem Cell-derived Exosomes - Yahoo Finance - April 27th, 2023
- Why does hair turn gray? A new study says 'stuck' stem cells may ... - NPR - April 27th, 2023
- Study advances understanding of how melanocyte stem cells work to color ... - April 21st, 2023
- Stem cell research and therapy legislation to be replaced, says ... - Bahamas Tribune - April 21st, 2023
- Stem Cell Research (journal) - Wikipedia - April 21st, 2023
- Scientists Are About to Try to Create Stem Cells in Space - April 21st, 2023
- Stem Cell Research & Therapy | Articles - BioMed Central - April 16th, 2023
- Stem Cell Junk Yards Reveal a New Clue About Aging | WIRED - April 16th, 2023
- Global Stem Cells Market Research Report 2023: Implications - April 16th, 2023
- Stem cell research can help people with hard- | EurekAlert! - April 16th, 2023
- University Of Edinburgh's stem cell research gets funding boost - India Education Diary - April 14th, 2023
- Two major stem cell research projects supported with more than ... - University of California, Santa Cruz - April 8th, 2023
- Cancer detection predicts tumors before they form: discovery - March 29th, 2023
- Stem cell therapy: a potential cure for hearing loss? - March 29th, 2023
- Turkey's Only Stem Cell Production Application and Research Center - Expat Guide Turkey - March 24th, 2023
- Stem Cell Research Article, Embryonic Cells Information, Cell Therapy ... - March 24th, 2023
- Scientists create mice with cells from 2 males for 1st time - March 19th, 2023
- QBRI concludes stem cells symposium - Gulf Times - March 17th, 2023
- 2020 California Proposition 14 - Wikipedia - March 17th, 2023
- Stem Cell Reports | Journal | ScienceDirect.com by Elsevier - March 17th, 2023
- Stem cell therapy MSC-NP found to ease inflammatory activity of brain ... - March 12th, 2023
- Stem Cell Research | Office of Research Compliance | SIU - March 12th, 2023
- Heart disease study shows hope for stem cell treatment - March 4th, 2023
- Stem cells drive antler regeneration | Science - March 4th, 2023
- Stem Cell Research: Argumentative Essay - Free Essay Example - Edubirdie - March 4th, 2023
- Stem Cell Research | Thermo Fisher Scientific - US - March 4th, 2023
- Stem cell controversy - Wikipedia - February 28th, 2023
- National Institutes of Health grant funds interdisciplinary ... - February 27th, 2023
- Research Associate in Cancer Stem Cell Research job with ... - Times Higher Education - February 25th, 2023
- Pioneering Stem Cell Research Conference at The Aga Khan ... - The Aga Khan University - February 25th, 2023
- Scientists grapple with ethics of stem cell research - STAT - February 17th, 2023
- Research Associate in Stem Cell Neurobiology job with KINGS ... - Times Higher Education - January 30th, 2023
- Stem cells: past, present, and future | Stem Cell Research & Therapy ... - January 12th, 2023
- Edu Thesis & Essay: Stem cell research outline top writers online! - January 12th, 2023
- Stem Cell Biology Research Program | Johns Hopkins Institute for Cell ... - January 5th, 2023
- Stem cell population identified is vital for bone regeneration: Research - ThePrint - January 5th, 2023
- Leading scientist 'blown away' by UAE advances in stem cell research - The National - December 15th, 2022
- The Stem Cell Assay Market Is Set To Grow At A 20% Annual Rate, Due To The High Prevalence Of Chronic Diseases As Per The Business Research Company's... - December 13th, 2022
Recent Comments