Form Bio and Cure Rare Disease have entered a strategic partnership aimed at speeding development of genetic therapies for rare neuromuscular diseases, beginning with Duchenne muscular dystrophy (DMD).
Under the collaboration, Form Bio will leverage its proprietary in silico and AI/ML platform to assist Cure Rare Disease in optimizing adeno-associated virus (AAV) construct designs. The goal is to enhance manufacturability, packaging efficiency, gene expression, and safety, thereby reducing development time and costs. According to the company, initial efforts improved the fraction of full-genome AAV particles from 13% to 59%, a 4.4-fold increase, within one month.
Cure Rare Disease had previously been working on a DMD candidate lacking optimal manufacturability and reduced immunogenicity. Through combining long-read sequencing, genome integrity analysis, promoter modification, and nuclease optimization, the partnership seeks to generate improved lead candidates.
The collaboration is intended to streamline lead candidate selection and advance in vivo preclinical work for DMD and potentially other ultra-rare neuromuscular and neurodegenerative diseases.
Form Bio spun out from Colossal Biosciences in 2022 and positions itself as a provider of AI-powered tools for genome engineering. Cure Rare Disease is a nonprofit biotech developing therapies for ultra-rare diseases.
