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# Fast Track for Hemophilia A Gene Therapy
- URL: https://www.fdaweb.com/fast-track-for-hemophilia-a-gene-therapy/
- Published: 2022-04-01T12:00:00.000Z
- Updated: 2026-09-14T17:39:06.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5151503

FDA has granted ASC Therapeutics a fast track designation for ASC618, a second-generation gene therapy for treating hemophilia A patients. The company received an IND approval in 2021 and an Orphan Drug Designation in 2020 for the therapy.

The company describes ASC618 as an AAV8-based gene that “incorporates a novel liver-specific promoter and a bioengineered, codon-optimized B domain-deleted FVIII \[Factor VIII\] variant.” It says that in preclinical studies, ASC618 exhibited at least a 10-fold increase in the biosynthesis and secretion of FVIII compared with native human FVIII bioengineered gene constructs. “ASC618 has the potential to increase durability of clotting factor biosynthesis and secretion by minimizing cellular stress and induction of the unfolded protein response, which may lead to diminished FVIII production from liver cells,” the company says.