Genethon

32 articles about Genethon

• Genethon Pursues Different Strategies for Ensuring Patient Access to Gene Therapies for Rare Diseases

  2/29/2024

  Genethon, a non-profit research organization focused on developing gene therapies for rare diseases, marks International Rare Disease Day by highlighting its efforts to bring gene therapies to patients suffering from rare diseases such as limb girdle muscular dystrophies, Crigler Najjar syndrome and Duchenne muscular dystrophy.

• Genethon Using Artificial Intelligence to Improve Effectiveness of Gene Therapies and Patient Access

  11/27/2023

  Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association, provided an update on its efforts to employ Artificial Intelligence in revolutionizing the development and bioproduction of gene therapies.

• Positive Phase 1/2 Clinical Trial Results of Genethon’s Gene Therapy for Crigler-Najjar Syndrome, a Rare Liver Disease, Published in The New England Journal of Medicine

  8/22/2023

  Genethon, a pioneer and leader in gene therapy research and development for rare genetic diseases, announced positive Phase 1/2 clinical trial results of its gene therapy, GNT-0003, for Crigler-Najjar syndrome were published in The New England Journal of Medicine.

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  Genethon’s Gene Therapy for Rare Liver Disease Shows Promising Early-Stage Results

  8/18/2023

  In a European Phase I/II clinical trial, the experimental drug for treating Crigler-Najjar syndrome reduced bilirubin below toxic threshold in the liver with a single intravenous injection.

• GenoTher Biocluster Designation Bolsters French Leadership in Gene Therapy

  7/10/2023

  Genethon, a pioneer in gene therapy, said the French government’s support of the GenoTher Biocluster validates the efforts of its founding organizations in creating a global center of excellence in gene therapy and establishes their work and dedication as a national priority for advancing gene therapies to treat both rare and common diseases.

• Genethon Given PRIME Status by EMA for Gene Therapy To Treat Crigler-Najjar Syndrome, a Rare Liver Disease

  3/7/2023

  Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association, announced that the European Medicines Agency has granted PRIME status to the gene therapy, GNT-0003, currently in clinical trials for Crigler-Najjar syndrome, a rare liver disease.

• Genethon Launches Pivotal Clinical Trial of Gene Therapy for Crigler-Najjar Syndrome, a Rare Liver Disease

  1/10/2023

  Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association, has launched a pivotal clinical trial in Europe for treatment of Crigler-Najjar syndrome, a life threatening liver disease.

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  Sarepta, Roche Tout Promising Data for Muscular Dystrophy Gene Therapy

  7/6/2022

  Sarepta Therapeutics and its partner, Roche, presented promising functional data from several trials of its gene therapy for Duchenne muscular dystrophy.
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  Dyne's Exon-51 Skipping DMD Therapy Moves Forward on FDA Go Sign

  7/5/2022

  The FDA lifted the clinical hold on Dyne Therapeutics’ clinical study of DYNE-251 in Duchenne muscular dystrophy (DMD) patients amenable to skipping exon 51. A Phase I/II trial is expected by mid-year.
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  Collaborative Analysis Reveals "Class Effect" in DMD Safety Issues

  5/19/2022

  Pfizer, Sarepta Therapeutics, Genethon and Solid Biosciences are working on gene therapies for Duchenne Muscular Dystrophy. Now, the four companies have teamed up.

• Gene Therapies Continue to Emerge as Effective Treatments for Rare Diseases, But Complex Challenges Remain

  2/28/2022

  Gene Therapies Continue to Emerge as Effective Treatments for Rare Diseases, But Complex Challenges Remain.

• Genethon and WhiteLab Genomics Join Forces to Enhance Gene Therapy Through Artificial Intelligence

  2/10/2021

  WhiteLab Genomics, a specialist in artificial intelligence applied to gene and cell therapies, has signed a partnership agreement with Genethon, a pioneering research center in the field of gene therapy. The alliance will harness the power of artificial intelligence to accelerate development of innovative gene therapies.

• Yposkesi selected to produce clinical and large-scale commercial material of micro-dystrophin for Genethon and Sarepta Therapeutics program

  1/9/2020

  Its capacity to produce AAV (Adeno Associated Virus) materials at large-scale will support Genethon and Sarepta’s project to take micro-dystrophin to clinical trials this year

• Orchard Therapeutics and Généthon Announce Gene Therapy Alliance in X-Linked Chronic Granulomatous Disease

  12/15/2017

  X-linked CGD is a primary immune deficiency resulting from a mutation on the X-chromosome and affecting mainly boys.

• Spark Therapeutics Enters Into Licensing Agreement With Genethon

  9/20/2017

• Sarepta Announces Its Partner, Genethon, Published New Micro-Dystrophin Gene Therapy Data In Nature Communications

  7/27/2017

• Sarepta And Genethon Announce A Gene Therapy Research Collaboration For The Treatment Of Duchenne Muscular Dystrophy

  6/21/2017

• CEVEC Pharma And Genethonn Sign A Collaboration Agreement To Develop Lentiviral Vector Packaging Cell Lines Based On CEVEC's Proprietary CAP(R)GT Technology

  9/17/2015

• Audentes Therapeutics And Genethon Announce Agreement To Develop Treatment For Severe Genetic Disease X-Linked Myotubular Myopathy

  2/5/2014

• Genethon, the French AFM-Telethon Laboratory, Becomes the First Not-for-Profit to Obtain Authorization From ANSM to be a Pharmaceutical Manufacturer

  7/8/2013