China-based Suzhou GenAssist Therapeutic Co., Ltd has announced that the US Food and Drug Administration (FDA) has accepted a pre-Investigational New Drug (pre-IND) application for the company’s first pipeline candidate, GEN6050. This in vivo base editing drug targets exon 50 skipping in the Duchenne muscular dystrophy (DMD) gene, with the aim of restoring the expression of dystrophin protein through base editing.
Pre-IND Process and Its Significance
Pre-IND meetings, though voluntary, are crucial for sponsors to submit and communicate with the FDA prior to initiating full clinical trials. The primary goal is to ensure that pharmacological and non-clinical studies are properly aligned and to determine if existing research results are sufficient to support clinical research endeavors.
Duchenne Muscular Dystrophy (DMD): A Rare and Fatal Disease
DMD is a rare and fatal neuromuscular genetic disease that primarily affects boys, with no effective treatments available beyond hormone therapy as the primary treatment option. Despite being an orphan condition, DMD has a relatively high incidence rate, with one DMD patient diagnosed per 4,000 newborn boys. Exon 50 skipping has the potential to treat 4% of DMD patients, according to the source.
Suzhou GenAssist’s Background and Funding
Founded in July 2020 with initial backing from Redhill Capital, Suzhou GenAssist aims to leverage its proprietary TAM (Targeted AID-based Mutagenesis) base editing technology. The company completed a Series A funding round in December 2022, with investors including the AstraZeneca CICC medical venture capital fund, Baidu Ventures, Sequoia Capital China, and others.-Fineline Info & Tech
