Saliogen Therapeutics Inc. has nominated a development candidate, SGT-1001, for the treatment of Stargardt disease. SGT-1001 is being developed as a one-time, nonviral therapy to slow or stop the progressive loss of central vision in people with Stargardt disease, regardless of the type of mutation.
Geneventiv Therapeutics Inc. has been awarded a Direct to Phase II Small Business Innovation Research (SBIR) grant for approximately $2.5 million from the National Heart Lung Blood Institute (NHLBI) at the National Institutes of Health (NIH) to support development of a universal gene therapy for hemophilia A or B with or without inhibitors.
Voyager Therapeutics Inc. has announced the selection of a lead development candidate in the GBA1 gene therapy program for the treatment of Parkinson’s disease and other GBA1-mediated diseases under its collaboration with Neurocrine Biosciences Inc.
Atamyo Therapeutics SAS has received clinical trial application (CTA) authorizations in Italy and France for ATA-200, its gene therapy for the treatment of γ-sarcoglycan related limb-girdle muscular dystrophy type 2C/R5 (LGMD2C/R5).
Samsung Life Science Fund, created jointly between Samsung Biologics Co. Ltd., Samsung Bioepis Co. Ltd. and Samsung C&T, and managed by Samsung Venture Investment Corp., and Brickbio Inc. have announced Samsung’s investment in preclinical-stage biopharmaceutical company Brickbio.
Asgard Therapeutics AB has announced a €30 million (US$32.7 million) series A financing to advance its first-in-class in vivo cell reprogramming platform for immuno-oncology.
Fractyl Health Inc. has reported promising new preclinical findings supporting RJVA-001, the first clinical candidate in its Rejuva pancreatic gene therapy platform. The company’s GLP-1 gene therapy candidate has potential to treat metabolic diseases, including obesity and type 2 diabetes.
The National Institute of Neurological Disorders and Stroke (NINDS) has awarded a 3-year grant totaling approximately $4.1 million to the Research Institute at Nationwide Children’s Hospital to fund completion of preclinical studies, manufacturing and preparation of an IND application for a first-in-human trial to advance adeno-associated virus (AAV)-ATP7A gene therapy for the treatment of Menkes disease.
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