Latest news with #FredericRevah
Yahoo
28-07-2025
- Business
- Yahoo
Genethon to Launch Pivotal Trial in Europe of GNT0004 a Low-Dose Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy
Phase 3 trial clearance is based on the Phase 1/2 results demonstrating safety and efficacy of the 3x10¹³ vg/kg dose of microdystrophin, which is lower than doses used in other gene therapies for Duchenne muscular dystrophy (DMD). The double blind trial will be initiated in the UK and France beginning in August and September, and will enroll 64 boys aged 6 to 10 with DMD who have retained their walking ability. PARIS, July 28, 2025--(BUSINESS WIRE)--Genethon, a worldwide pioneer and leader in research and development in gene therapy for rare genetic diseases, has received approvals from regulatory authorities, MHRA and EMA*, to begin pivotal Phase 3 clinical trials in France and the UK of its gene therapy, GNT0004, for Duchenne muscular dystrophy (DMD). Genethon CEO Frederic Revah observed, "We are delighted to be able to continue these trials and are determined to bring GNT0004 to market for young patients and their families who are waiting for a therapeutic solution. This marks a decisive step forward for our gene therapy program for DMD, which began in 2021 and has demonstrated extremely promising results in the first children treated in the Phase1/2 portion of our Phase1/2/3 study." Dr. Revah added, "In addition to the very positive results in patients treated in the early phases, one of the strengths of our product is the dose selected for the pivotal phase, which is lower than those used in other gene therapy trials for DMD. Approvals of our Phase 3 trials reflect the regulatory authorities' confidence in GNT0004 as well as the work accomplished by our teams." The Phase 3 authorizations in Europe are based on the results of Phase 1/2 studies showing good tolerance of GNT0004 as well as efficacy in terms of microdystrophin expression, creatine phosphokinase (CPK) reduction, and motor function. Patients showed prolonged improvement or stabilization of motor functions and significant persistent reduction in CPK, a key marker of muscle damage. The Phase 3 double blind trials will begin in August and September in the UK and France using a single intravenous injection of GNT0004, which contains an optimized hMD1 transgene, a shortened (3x10¹³ vg/kg microdystrophin) but functional version of the gene encoding dystrophin in an AAV8 vector associated with transient immunological prophylactic treatment. The vector is designed to express itself in muscle tissue and the heart thanks to a Spc5-12 promoter sequence specific to these tissues. A total of 64 boys aged 6 to 10 with DMD who have retained their walking ability will be enrolled. * French ANSM as reporting member state for the EMA About Duchenne muscular dystrophy Duchenne muscular dystrophy is a rare progressive genetic disease that affects all the muscles in the body and mainly boys (1 in 5,000). It is caused by abnormalities in the gene responsible for the production of dystrophin, a structural protein essential for the stability of muscle fiber membranes and their metabolism. The absence of dystrophin leads to progressive degeneration of the skeletal and cardiac muscles, loss of walking and respiratory capacity, progressive heart failure, 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 non-profit laboratory created by the AFM-Telethon. A first gene therapy drug, to which Genethon contributed, has been approved for marketing for spinal muscular atrophy. With more than 240 scientists and experts, Genethon's goal is to develop innovative therapies that change the lives of patients suffering from rare genetic diseases. Thirteen gene therapy products developed by Genethon or to which Genethon has contributed are currently undergoing clinical trials for diseases of the liver, blood, immune system, muscles, and eyes. Seven other products are in preparation for clinical trials over the next five years. View source version on Contacts Press contact:Marion Delbouis/Stéphanie Bardon – communication@ / +33 (0)6 45 15 95 87 Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Business Wire
28-07-2025
- Business
- Business Wire
Genethon to Launch Pivotal Trial in Europe of GNT0004 a Low-Dose Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy
PARIS--(BUSINESS WIRE)--Genethon, a worldwide pioneer and leader in research and development in gene therapy for rare genetic diseases, has received approvals from regulatory authorities, MHRA and EMA*, to begin pivotal Phase 3 clinical trials in France and the UK of its gene therapy, GNT0004, for Duchenne muscular dystrophy (DMD). Genethon CEO Frederic Revah observed, 'We are delighted to be able to continue these trials and are determined to bring GNT0004 to market for young patients and their families who are waiting for a therapeutic solution. This marks a decisive step forward for our gene therapy program for DMD, which began in 2021 and has demonstrated extremely promising results in the first children treated in the Phase1/2 portion of our Phase1/2/3 study.' Dr. Revah added, 'In addition to the very positive results in patients treated in the early phases, one of the strengths of our product is the dose selected for the pivotal phase, which is lower than those used in other gene therapy trials for DMD. Approvals of our Phase 3 trials reflect the regulatory authorities' confidence in GNT0004 as well as the work accomplished by our teams.' The Phase 3 authorizations in Europe are based on the results of Phase 1/2 studies showing good tolerance of GNT0004 as well as efficacy in terms of microdystrophin expression, creatine phosphokinase (CPK) reduction, and motor function. Patients showed prolonged improvement or stabilization of motor functions and significant persistent reduction in CPK, a key marker of muscle damage. The Phase 3 double blind trials will begin in August and September in the UK and France using a single intravenous injection of GNT0004, which contains an optimized hMD1 transgene, a shortened (3x10¹³ vg/kg microdystrophin) but functional version of the gene encoding dystrophin in an AAV8 vector associated with transient immunological prophylactic treatment. The vector is designed to express itself in muscle tissue and the heart thanks to a Spc5-12 promoter sequence specific to these tissues. A total of 64 boys aged 6 to 10 with DMD who have retained their walking ability will be enrolled. * French ANSM as reporting member state for the EMA About Duchenne muscular dystrophy Duchenne muscular dystrophy is a rare progressive genetic disease that affects all the muscles in the body and mainly boys (1 in 5,000). It is caused by abnormalities in the gene responsible for the production of dystrophin, a structural protein essential for the stability of muscle fiber membranes and their metabolism. The absence of dystrophin leads to progressive degeneration of the skeletal and cardiac muscles, loss of walking and respiratory capacity, progressive heart failure, 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 non-profit laboratory created by the AFM-Telethon. A first gene therapy drug, to which Genethon contributed, has been approved for marketing for spinal muscular atrophy. With more than 240 scientists and experts, Genethon's goal is to develop innovative therapies that change the lives of patients suffering from rare genetic diseases. Thirteen gene therapy products developed by Genethon or to which Genethon has contributed are currently undergoing clinical trials for diseases of the liver, blood, immune system, muscles, and eyes. Seven other products are in preparation for clinical trials over the next five years.
Business Wire
19-06-2025
- Health
- Business Wire
Clinical Milestones, Vector Innovation Key to Gene Therapy, GenoTher Biocluster's First International Summit,
PARIS--(BUSINESS WIRE)--Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), and its spin-off Atamyo Therapeutics made significant advances in their gene therapies for multiple rare diseases as highlighted in Genethon's most recent Newsletter. Chief among them are key clinical milestones reached, including those reflected in Genethon's Phase 3 ready Duchene muscular dystrophy gene therapy (GNT0004); and Atamyo's significant milestones in clinical trials of gene therapies (ATA-100 and ATA-200) for treatment of two subtypes of limb-girdle muscular dystrophy (LGMD2I/R9 and LGMD2C/R5). In addition, the GenoTher Biocluster, a public-private partnership serving as a leading ecosystem for generation of innovative genetic medicines' approaches, held its first international summit attended by 350 researchers, investors, biotech executives and public decision-makers representing more than 100 organizations. In his CEO Commentary, Genethon's Frederic Revah discusses new research involving the organization's AAV platform to increase safety, efficacy and disease applications of gene therapies. Dr. Revah reports Genethon also is exploring non-viral based delivery systems, such as Lipid Nano Particles (LNPs), for their potential in gene editing. Atamyo in April completed the dose escalation phase of a Phase 1b/2b trial in Europe of ATA-100 for LGMD2I/R9 and announced in June dosing in the US of the first two patients in a Phase 1b/2 study of ATA-200 for LGMD2C/R5. Both subtypes lead to loss of ambulation and other complications in children and there are no cures. Read the full Newsletter to learn about these gene therapy advances and others. About Genethon As a pioneer in the discovery and development of gene therapies for rare diseases, Genethon is a non-profit laboratory that was established by AFM-Telethon. A first gene therapy for spinal muscular atrophy to which Genethon contributed has obtained a product license. With more than 200 scientists and professional staff, Genethon is pursuing its aim to develop therapies which change the lives of patients suffering from rare genetic diseases. Thirteen products stemming from Genethon's R&D or from collaborations are in clinical trial for diseases of the liver, blood, immune system, muscles and eyes. Seven other products could enter clinical trials over the next five years. More information at
Associated Press
19-05-2025
- Health
- Associated Press
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)--May 19, 2025-- 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: 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: source version on CONTACT: Press contact: Stephanie Bardon [email protected] 06.45.15.95.87 KEYWORD: EUROPE UNITED STATES NORTH AMERICA FRANCE WASHINGTON INDUSTRY KEYWORD: BIOTECHNOLOGY HEALTH GENETICS PHARMACEUTICAL CLINICAL TRIALS SOURCE: Genethon Copyright Business Wire 2025. PUB: 05/19/2025 05:30 AM/DISC: 05/19/2025 05:31 AM
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:
