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NIH – The Foundation for the National Institutes of Health (FNIH) Announces Selection of Eight Rare Diseases for the Bespoke Gene Therapy Consortium Clinical Trial Portfolio (Catapult’s regulatory round-up May)

The Foundation for the National Institutes of Health (FNIH) has announced its plans to prioritise eight rare diseases to provide industry standards for manufacturing, preclinical testing and product analytical testing for gene therapy development.

The eight diseases that will make up the clinical trial portfolio are:

  • Charcot-Marie-Tooth disease type 4J
  • Congenital Hereditary Endothelial Dystrophy
  • Morquio A Syndrome
  • Multiple Sulfatase Deficiency
  • NPHP5 Retinal Degeneration
  • Propionic Acidemia (PCCB)
  • Retinitis pigmentosa 45
  • Spastic paraplegia 50

The Foundation’s Accelerating Medicines Partnership (AMP)Bespoke Gene Therapy Consortium (BGTC) will study these eight conditions by focusing on gene therapies using adeno-associated vectors (AAV).

Published on the 2nd of June 2023. Read the entire regulatory round-up for the month of May published and curated by Catapult here.


OpenEdition vous propose de citer ce billet de la manière suivante :
adelage (21 février 2024). NIH – The Foundation for the National Institutes of Health (FNIH) Announces Selection of Eight Rare Diseases for the Bespoke Gene Therapy Consortium Clinical Trial Portfolio (Catapult’s regulatory round-up May). ELSIBI. Consulté le 18 février 2025 à l’adresse https://doi.org/10.58079/vvhs


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