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).
Alveogene Ltd. has launched with a focus on inhaled gene therapies for respiratory disorders. The company has been created by Oxford Science Enterprises, Harrington Discovery Institute at University Hospitals, and Old College Capital in partnership with six scientists from the UK Respiratory Gene Therapy Consortium (GTC).
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