ASC Therapeutics Joins U S Consortium of Gene Therapy Partners Including the NIH and FDA to Accelerate Treatments for Rare Diseases streetinsider.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from streetinsider.com Daily Mail and Mail on Sunday newspapers.
Guangping Gao and Dan Wang partner with ASC Therapeutics to develop novel gene therapy for maple syrup urine disease umassmed.edu - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from umassmed.edu Daily Mail and Mail on Sunday newspapers.
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ASC Therapeutics Receives IND Clearance From the U.S. Food and Drug Administration for ASC618 Second-Generation Gene Therapy for Hemophilia A
July 8, 2021 GMT
MILPITAS, Calif. (BUSINESS WIRE) Jul 7, 2021
ASC Therapeutics, a privately-held biopharmaceutical company pioneering the development of transformative in-vivo gene replacement, gene editing and allogeneic cell therapies for hematologic and other rare disorders today announced that the U.S. Food & Drug Administration (FDA) has cleared an Investigational New Drug (IND) application for ASC618, an investigational second-generation gene therapy for patients with severe and moderately severe hemophilia A. The transformational Adeno-Associated Virus (AAV) construct contains a proprietary B-domain deleted codon-optimized bioengineered chimeric Factor VIII (FVIII) gene and a minimal-length liver-specific promoter, shown in pre-clinical stu