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Business Wire
19-05-2025
- Health
- Business Wire
Genethon Presents Two Year Consolidated Results of Its Gene Therapy Trial for Duchenne Muscular Dystrophy: Maintenance of Motor Functions and Significant, Sustained Reduction in CPK Levels in Patients Treated at the Effective Dose at ASGCT 2025
PARIS--(BUSINESS WIRE)-- Genethon unveiled the 2-year follow-up data from its GNT0004 gene therapy clinical trial for Duchenne Muscular Dystrophy (GNT-016-MDYF) at the annual meeting of the American Society of Gene & Cell Therapy (ASGCT) in New Orleans, May 13 – 17, 2025. Five patients, aged 6 to 10 years, were treated, 2 at the first dose level and 3 at the second dose level (3x10¹³ vg/kg). The initial part of the clinical trial aimed at selecting the optimal dose (dose escalation phase), evaluating the tolerance and preliminary efficacy of the treatment and determined the effective dose for the pivotal phase of the GNT-016-MDYF trial, which is expected to start in mid-2025 (3x10¹³ vg/kg). Genethon CEO Frederic Revah observed, "The results of our gene therapy GNT0004 are very positive in patients treated at the dose of 3x10¹³ vg/kg, both in terms of microdystrophin expression and clinical efficacy criteria. Besides these results, the advantage of our product lies in the selected dose for the pivotal phase, which is lower than those used in other gene therapy trials for Duchenne Muscular Dystrophy. We are currently preparing the pivotal phase that we will conduct in Europe and the US." Safety, efficacy, and pharmacodynamic results show good tolerance of GNT0004 associated with transient immunological prophylactic treatment, as well as efficacy data in terms of microdystrophin expression, CPK reduction, and clinical criteria (NSAA, timed tests). Patients treated at the effective dose show prolonged improvement or stabilization of motor functions and significant persistent reduction in creatine kinase (CPK) levels, a key marker of muscle damage. One-year post-treatment, the comparison of the three patients treated at the effective dose with a group of 34 untreated patients, matched by age and followed in the same centers and by the same practitioners, shows a difference of +4.7 points in the score obtained using the internationally recognized clinical evaluation scale NSAA between treated and untreated patients. At 24 months post-treatment, key observations include: For the 2 patients who reached 2 years post-treatment out of the 3 treated at the effective dose, the trial shows stabilization of motor functions measured by the NSAA scale , while untreated patients from the parallel natural history study showed a continuous and significant average decline in NSAA. For one treated patient, the observed improvement allowed reaching the maximum score of 34 at 12 months, confirmed at 24 months post-treatment. Stabilization of CPK reduction between 50% and 87% on average: >75% at 18 months post-treatment (data from the 3 patients treated at the effective dose), and persistent (up to 24 months follow-up for the first two patients treated at this dose). The reassuring safety profile of the gene therapy drug is confirmed two years after injection, without the occurrence of serious adverse effects at the selected dose, which is notably lower than that used for other gene therapy products under development for Duchenne Muscular Dystrophy. About GNT0004 and the trial The GNT0004 gene therapy is composed of an AAV8 (adeno-associated virus) vector and the optimized hMD1 transgene, a shortened but functional version of the gene encoding dystrophin, the protein deficient in people with DMD. This vector is designed to be expressed in muscle tissue and also in the heart, thanks to a tissue-specific Spc5-12 promoter sequence. GNT0004 is administered by a single intravenous injection. It was developed by Genethon, in partnership with the teams of Prof. Dickson (University of London, Royal Holloway) and the Institut de Myologie (Paris). The trial, sponsored by Genethon, combines Phases 1/2/3, a dose-escalation phase followed by a pivotal phase at the dose finally chosen. The trial is being carried out in France and the UK and includes boys aged 6 to 10 with DMD who have retained their ability to walk. About Duchenne muscular dystrophy DMD is a rare, progressive genetic disease affecting all the body's muscles, and mainly boys (1 in 5000). It is due to abnormalities in the gene responsible for producing dystrophin, a structural protein essential for the stability of muscle fiber membranes and their metabolism. The absence of dystrophin leads to progressive degeneration of skeletal and cardiac muscles, loss of walking and respiratory capacity, cardiomyopathy and death between the ages of 20 and 40. About Genethon A pioneer in the discovery and development of gene therapies for rare diseases, Genethon is a nonprofit organization created by the AFM-Téléthon. The first gene therapy to treat spinal muscular atrophy, incorporating technologies developed at Genethon, is marketed worldwide. With over 240 scientists and professionals, Genethon pursues its goal of developing innovative therapies that change the lives of patients suffering from rare genetic diseases. Thirteen products from Genethon's R&D or collaborations are in clinical trials for diseases of the liver, blood, immune system, muscles and eyes. A further seven products could enter clinical trials in the next five years. To find out more visit:
Business Wire
30-04-2025
- Business
- Business Wire
Capstan Therapeutics to Participate in Upcoming Scientific Conferences and Present New Preclinical Data in Support of Lead Anti-CD19 In Vivo CAR-T Candidate, CPTX2309
SAN DIEGO--(BUSINESS WIRE)--Capstan Therapeutics, Inc. ('Capstan'), a biotechnology company dedicated to advancing in vivo reprogramming of cells through RNA delivery using targeted lipid nanoparticles (tLNP), today announced that the Company will present at upcoming scientific conferences and showcase new preclinical data on CPTX2309, Capstan's lead anti-CD19 in vivo CAR-T candidate, at the American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting in New Orleans, LA. 'Among the diverse set of presentations showcasing Capstan's non-viral CellSeeker™ platform, we are particularly encouraged by new preclinical data in support of our in vivo anti-CD19 CAR-T program, which demonstrate that a compact two-dose cycle was sufficient to induce rapid and deep B cell depletion in blood and tissues of non-human primates,' said Adrian Bot, M.D., Ph.D., Chief Scientific Officer and Executive Vice President of R&D at Capstan. 'These preclinical data highlight the potency of B cell depletion achievable with a transient in vivo CAR mRNA approach, without the need for lymphodepletion, and set the stage for clinical evaluation of CPTX2309.' Featured Presentation: American Society of Gene & Cell Therapy (ASGCT) 28 th Annual Meeting Date: Location: New Orleans, LA Oral presentation title: A Two-Infusion Regimen with a Novel In Vivo Non-Viral Chimeric Antigen Receptor (CAR) Achieves up to 90% CD8+ T Cell Engineering and Tissue Depletion of Target Cells in Non-Human Primates (NHPs) Presenter: Haig Aghajanian, Ph.D., Vice President of Research, Co-founder Session title: CAR T Innovations in Autoimmune and Infectious Disease and Allergy Date and time: May 15, 2025, 5:00 p.m. - 5:15 p.m. CT Location: Room 393-396 Additional Presentations feature progress with our CellSeeker TM platform technology applicable to both mRNA and gene editing payloads: Cellicon Valley '25: The Future of Cell and Gene Therapies Date: Apr 30-May 2, 2025 Location: Philadelphia, PA Oral Presentation Title: Leading the Charge for In Vivo Cell Therapy for The Treatment of Autoimmune Disorders Presenter: Laura Shawver, Ph.D., Chief Executive Officer Session title: Plenary Session 2, Leadership in Cell and Gene Therapies: The XX Factor Date and Time: May 1, 2025, 10:20 a.m. - 10:30 a.m. ET International Society of Cell & Gene Therapy (ISCT) 2025 Date: May 7-10, 2025 Location: New Orleans, LA Poster title: Design and Preclinical Development of a Novel In Vivo Chimeric Antigen Receptor (CAR) Product for B-Cell Involved Diseases Presenter: Haig Aghajanian, Ph.D., Vice President of Research, Co-founder Poster number: 901 Session title: Poster Networking Reception 1; Immunotherapy (CAR-T, T Reg, NK Cells, etc.) Date and time: May 7, 2025, 7:00 p.m. - 8.30 p.m. CT 21 st Annual PEGS Boston: The Essential Protein & Antibody Engineering Summit Date: May 12-16, 2025 Location: Omni Boston Hotel at the Seaport, Boston, MA Title: Chairperson's Remarks Presenter: Adrian Bot, M.D., Ph.D., Chief Scientific Officer Track: In Vivo CAR T Engineering: Moving into the Clinic Date and time: May 15, 2025, 8:25 a.m. ET Oral presentation title: In vivo mRNA-Based CAR T Cell Engineering for Treatment of B Cell Disorders Presenter: John Rossi, Vice President, Translational Medicine Track: In Vivo CAR T Engineering: Moving into the Clinic Date and time: May 15, 2025, 9:00 a.m. ET American Society of Gene & Cell Therapy (ASGCT) 28 th Annual Meeting Date: May 13-17, 2025 Location: New Orleans, LA Science Symposium: Targeted Nanosystems For Gene Transfer And Editing: Beyond Delivery To The Liver Oral presentation title: In vivo immune cell engineering using targeted nanoparticles Presenter: Priya Karmali, Ph.D., Chief Technology Officer Date and time: May 16, 2025, 4:10 p.m. - 4:35 p.m. CT Location: NOLA Theater B Poster: Efficient In Vivo Gene Editing of T Cells Utilizing Novel Targeted Lipid Nanoparticles Presenter: Esther Chen, Ph.D., Principal Scientist, R&D Session title: Tuesday Poster Reception Date and time: May 13, 2025, 6:00 p.m. - 7:30 p.m. CT Location: Poster Hall I2 Poster: Effective Gene Editing in Hematopoietic Stem and Progenitor Cells (HSPCs) through a Novel Targeted Lipid Nanoparticle Presenter: Esther Chen, Ph.D., Principal Scientist, R&D Session title: Thursday Poster Reception Date and time: May 15, 2025, 5:30 p.m. - 7:00 p.m. CT Location: Poster Hall I2 TIDES USA Date: May 19-22, 2025 Location: Manchester Grand Hyatt San Diego, San Diego, CA Oral presentation title: In Vivo Engineering of Cells Using Targeted Lipid Nanoparticles Presenter: Priya Karmali, Ph.D., Chief Technology Officer Track: mRNA Technology and Applications Date and time: May 21, 2025, 2:00 p.m. PT About Capstan Therapeutics, Inc. ( Capstan is a biotechnology company with a mission to multiply the therapeutic possibilities for patients by developing targeted in vivo RNA technologies. Our proprietary CellSeeker™ tLNP platform technology is composed of novel LNPs conjugated with a recombinant protein binder, such as a monoclonal antibody. tLNPs are designed to deliver payloads, including mRNA or gene editing tools, capable of reprogramming specific cell types in vivo. Capstan's CellSeeker™ technology has the potential to generate transformative therapies with possible applications across a broad range of disease areas, including autoimmune disorders, oncology, fibrosis, and monogenic blood disorders. For more information, please visit and follow us on LinkedIn.
Business Wire
28-04-2025
- Business
- Business Wire
Adicet Bio Announces Oral Presentation Highlighting Preclinical ADI-270 Data at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting
REDWOOD CITY, Calif. & BOSTON--(BUSINESS WIRE)--Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing allogeneic gamma delta T cell therapies for autoimmune diseases and cancer, today announced the acceptance of an abstract for an oral presentation at the upcoming American Society of Gene & Cell Therapy (ASGCT) Annual Meeting taking place May 13-17, 2025, in New Orleans, LA. Details of the oral presentation are as follows: Title: ADI-270, an Armored Allogeneic Anti-CD70 CAR γδ T Cell Therapy, Demonstrates Robust CAR-Directed and -Independent Anti-Tumor Activity Against Hematologic and Solid Tumor Models Compared to Conventional CAR αβ T Cells Session Name: Engineered Immune Effector Cells for Solid Tumors Presenting Author: Melinda Au Date and Time About Adicet Bio, Inc. Adicet Bio, Inc. is a clinical stage biotechnology company discovering and developing allogeneic gamma delta T cell therapies for autoimmune diseases and cancer. Adicet is advancing a pipeline of 'off-the-shelf' gamma delta T cells, engineered with chimeric antigen receptors (CARs), to facilitate durable activity in patients. For more information, please visit our website at
