The second-generation gene therapy for hemophilia A called ASC618 has received Fast Track Designation from the US Food and Drug Administration (FDA), as published in a press release. It also received a positive opinion for Orphan Medical Product Designation from the European Medical Agency’s (EMA) Committee for Orphan Medical Products.

“This brings us one step closer for providing a truly novel therapeutic approach for hemophilia A, providing potentially a functional cure for patients who currently require life-long care,” said Ruhong Jiang, PhD, chief executive officer at ASC Therapeutics, the developer of ASC618.

Prior regulatory achievements for ASC618 include US FDA Orphan Drug designation in 2020 and Investigational New Drug clearance by the FDA in 2021.


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“Pre-clinical studies have shown that ASC618 has the potential to reduce therapeutic dosing and increase durability of hemophilia A gene therapy with a novel bioengineered construct that can improve biosynthesis, protein folding, and secretion of factor VIII,” said Oscar Segurado, MD, PhD, chief medical officer at ASC Therapeutics.

ASC618 is a gene therapy utilizing an adeno-associated virus 8 vector to deliver a novel liver-specific promoter combined with a bioengineered, codon-optimized B domain-deleted variant of FVIII. Preclinical studies showed that ASC618 yielded a 10-fold increase in the synthesis and fusion of FVII compared to bioengineered gene constructs utilizing native human FVIII.

“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,” according to the press release.

ASC Therapeutics plans to begin recruitment soon for a phase 1/2 clinical trial (NCT04676048) to investigate the safety, tolerability, and preliminary efficacy of ASC618. The study aims to recruit 12 patients with hemophilia A who will receive a single intravenous infusion of ASC618 and then be monitored for 12 months.

Reference

ASC Therapeutics receives key regulatory designations in US and Europe to advance its second-generation gene therapy for hemophilia A. News release. ASC Therapeutics; March 29, 2022.