Bloomsbury Genetic Therapies Ltd. has announced it received orphan drug designations from the FDA and the European Commission for BGT-INAD, an investigational gene therapy for the treatment of infantile neuroaxonal dystrophy (INAD).
Activated phosphoinositide 3-kinase δ syndrome type 1 (APDS1) is a rare disease caused by gain-of-function (GOF) mutations in the PIK3CD gene that presents with combined immunodeficiency due to decreases in IgA, IgG, naive CD4 and naive CD8 cells. Nearly all APDS1 patients suffer from recurrent respiratory tract infections with most presenting with bronchiectasis and chronic viral infections.
Bloomsbury Genetic Therapies Ltd. has announced U.S. and E.U. orphan drug designations for BGT-NPC, an investigational gene therapy for the treatment of Niemann-Pick disease type C (NPC). BGT-NPC is an investigational AAV9 gene therapy designed to provide a potentially curative solution to NPC patients following a one-time injection in the cerebrospinal fluid.
Rejuvenate Bio Inc. has released new preclinical efficacy data for its gene therapy, RJB-0402, in a mouse model of arrhythmogenic cardiomyopathy (ACM), an inherited disease caused by mutations in one of several genes encoding desmosomal proteins.
Kriya Therapeutics Inc. has entered into an exclusive license, collaboration and supply agreement with Everads Therapy Ltd. to advance Kriya’s portfolio of gene therapies for retinal diseases using Everads’ suprachoroidal delivery device.
A new gene editing method uses the CRISPR technique to modify the cells of an organ in vivo, creating a mosaic used to identify the effects of each altered gene. Scientists from the Swiss Federal Institute of Technology (ETH) in Zürich developed this technology called AAV-Perturb-seq, based on adeno-associated virus (AAV) to target, edit and analyze single-cell genetic perturbations.
Krystal Biotech Inc. has received IND clearance from the FDA for KB-408 for the treatment of α1-antitrypsin deficiency (AATD). KB-408 is a modified, replication-defective, nonintegrating HSV-1-derived vector carrying two full-length copies of the serpin family A member 1 gene (SERPINA1) to enable expression of α1-antitrypsin (AAT).
Genflow Biosciences plc has received correspondence from Belgium’s Federal Agency for Medicines and Health Products (FAHMP) with a recommendation to initiate a phase I/II trial of GF-1002 in patients with nonalcoholic steatohepatitis (NASH), rather than in healthy volunteers.
Atamyo Therapeutics SAS has filed a clinical trial application (CTA) in Europe for ATA-200, its gene therapy targeting γ-sarcoglycan (SGCG)-related limb-girdle muscular dystrophy type 2C/R5 (LGMD-R5).
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