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 …
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Niemann-Pick Type C News
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 …
Azafaros Receives FDA’s IND Clearance and Fast Track Designation
Azafaros B.V. announced in January 2023 that it Investigational New Drug (IND) clearance from the United States Food and Drug Administration (FDA) to conduct a clinical Phase 2 trial for its lead asset, AZ-3102, for the treatment of GM2 gangliosidosis (GM2) and Niemann-Pick disease type C (NP-C). In addition to the IND clearance, the FDA …