Genethon's GNT0004 gene therapy shows long-term efficacy in patients with Duchenne muscular dystrophy, maintaining clinical efficacy and safety at two years. The trial included 72 boys aged 6-10 with retained walking ability, treated with GNT0004 at a therapeutic dose.
The company's ATA-200 gene therapy has shown safety, pharmacodynamics, and efficacy results in the first patients treated, offering hope for children with LGMD-R5. The therapy delivers a normal copy of the γ-sarcoglycan gene and has been awarded Orphan Drug Designation in the US and Europe.
A new Immunology Center will accelerate discoveries in muscle immunology and immune responses to gene therapies. Klaudia Kuranda brings expertise in immunology, onco-immunology, and leadership experience to the center.
Pompe disease is a rare genetic disorder caused by a deficiency in the GAA enzyme, leading to glycogen accumulation in cells. Genethon's gene therapy approach has shown preclinical efficacy in animal studies, correcting glycogen accumulation and improving cardiac hypertrophy and muscular dysfunction.
www.mitogether.com centralizes knowledge on genetic mitochondrial diseases affecting 1 in 4,300 people in Europe, with innovative therapies emerging. The platform facilitates information searches for monitoring, care, research, and clinical trials.
Genethon is launching a Phase 3 clinical trial in Europe for its low-dose microdystrophin gene therapy GNT0004, targeting boys aged 6 to 10 with retained walking ability. The trial aims to demonstrate efficacy and tolerance of the treatment.
A Phase 2 clinical trial has been initiated by Genethon and Hansa Biopharma to evaluate the efficacy and safety of imlifidase, a gene therapy for Crigler-Najjar syndrome. The trial aims to address anti-AAV antibodies, which prevent up to 1 in 3 people from benefiting from gene therapies.
Positive initial results from Genethon's gene therapy GNT0004 show stabilization of motor functions and improved dystrophin expression in patients with Duchenne Muscular Dystrophy. The therapy is expected to be launched in pivotal trial phases in Europe and the US in Q2/2025.
Genethon has developed an innovative gene therapy vector that effectively targets muscle tissue while reducing the risk of liver penetration. The new capsid design uses AI predictive methodology to improve efficacy and safety, paving the way for more effective treatments for neuromuscular diseases.
A European gene therapy clinical trial has demonstrated the possibility of restoring expression of the UGT1A1 enzyme in patients with severe Crigler-Najjar syndrome. The treatment resulted in significant reduction in bilirubin levels and sustained efficacy in patients, offering a new hope for treating this rare metabolic disease.
Researchers from Genethon and Université de Paris have developed an AAV-based gene therapy that corrects skeletal damage caused by XLH in mouse models. The treatment targets liver hepatocytes to express therapeutic proteins, potentially offering a lifelong solution for this rare disease.
A European gene therapy trial for Crigler-Najjar syndrome has demonstrated the first therapeutic effects of the treatment, allowing patients to stop phototherapy. The trial's preliminary results are encouraging, suggesting that gene therapy could become an alternative treatment for this severe liver disease.
Two important breakthroughs will be presented by Institute of Myology experts: a new genetic diagnosis for myopathy triggered by specific mutations, and an MRI study identifying spinal cord degeneration in ALS and FTD patients. The Muscle Atlas database will accelerate research, diagnosis, and therapeutic approaches.
A research team from Genethon has successfully inhibited the immune response induced by AAV antibodies, paving the way for repeated administration of gene therapy treatments. This breakthrough could enable treatment of rare genetic diseases and improve patient outcomes.
A gene therapy trial conducted in US and UK patients with X-linked Chronic Septic Granulomatosis, a rare genetic disease causing severe infections, has shown success. Six of nine patients are free of treatment complications, offering hope for sustainable treatment.
Researchers identify protein CaVbeta1E that promotes muscle mass maintenance via GDF5 signaling, counteracting sarcopenia. The study opens a new field of activity in developing therapeutic strategies against muscular decline associated with aging.
Researchers have successfully developed and tested a gene therapy approach using CRISPR-Cas9 technology to treat Steinert's myotonic dystrophy, a devastating neuromuscular disease. The study showed that the expanded CTG triplet repeat in the DMPK gene was 'cut' and removed from the gene, reducing toxic RNA aggregates in muscle cells.
Researchers developed a gene therapy using microdystrophin to treat Duchenne muscular dystrophy in dogs. The treatment restored muscle function and stabilized clinical symptoms for over 2 years. This breakthrough could lead to a new treatment for children with the disease.
A new gene therapy approach has restored muscle strength and prolonged life in dogs affected by myotubular myopathy, a rare X-linked genetic disorder. The treatment, administered via a single intravenous injection, was developed by Genethon and demonstrated efficacy in a large animal model.
Dr. Ana Buj Bello, a leading expert in gene therapy, has received the Outstanding New Investigator Award for her groundbreaking research on treating myotubular myopathy through gene therapy. Her work has shown promising results in animal models and paves the way for a potential clinical trial to treat children with this rare disease.
Researchers successfully restored bilirubin levels to those found in healthy animals using gene therapy, offering new hope for the treatment of Crigler-Najjar syndrome. The therapy involved transferring a copy of the UGT1A1 gene into liver cells using an AAV vector, resulting in long-term correction without immunosuppression.
A new gene therapy treatment has shown significant clinical improvement in six patients with Wiskott-Aldrich Syndrome, a rare and complex immune disorder. The treatment involves correcting genetic anomalies in blood stem cells using a lentiviral vector, resulting in restored immune function and reduced symptoms.
A new gene therapy treatment has demonstrated effectiveness in a canine model of DMD, skipping exons to produce functional dystrophin protein. The treatment was well-tolerated and resulted in high levels of dystrophin expression, with muscle strength increasing at higher doses.
Genethon, a non-profit organization, has partnered with Esteve to manufacture the gene therapy AAV9-hsulfamidase for treating Sanfilippo syndrome. This agreement supports clinical development and advances the treatment towards trials.