21 August 2023 – RPDD granted in the United States – London, UK, 21 August 2023 – Bloomsbury Genetic Therapies Limited, a clinical-stage biotechnology company developing potentially curative treatments for patients suffering from rare neurological and metabolic diseases based on clinically proven gene therapy technologies, announced today that the U.S. Food and Drug Administration (FDA) …
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Bloomsbury Genetic Therapies Outlines Pre-Clinical Data on CNS Programs including NPC
London, UK, 18 May 2023 – Bloomsbury Genetic Therapies Limited, a clinical-stage biotechnology company developing potentially curative treatments for patients suffering from rare neurological and metabolic diseases based on clinically proven gene therapy technologies, announced today that pre-clinical data from two of its brain-targeted programs, BGT-INAD and BGT-NPC for the treatment of Infantile Neuroaxonal Dystrophy …
Azafaros Announces IRB approval of 1st Site in U.S. for Phase 2 RAINBOW Study NPC and GM2
RAINBOW study design enables rapid advancement into the company’s planned Phase III efficacy trial Leiden, The Netherlands, May 22- 2023 – Azafaros B.V. today announced that the first US site for the Phase II RAINBOW study, led by Dr Marc Patterson of the Mayo Clinic in Minnesota, has received Institutional Review Board approval. The clinical …