Form Bio and Cure Rare Disease Partner to Accelerate Genetic Medicine Development

Rare Daily Staff

Form Bio has entered a strategic partnership with the nonprofit biotech Cure Rare Disease to accelerate the development of gene therapies for Duchenne muscular dystrophy and other rare diseases.

Under the collaboration, Form Bio will use its proprietary in silico artificial intelligence platform to help Cure Rare Disease design and optimize gene therapy candidates for packaging, expression, safety, and manufacturability—key factors in boosting clinical success and lowering costs. The program aims to streamline lead candidate selection and shorten development timelines.

Cure Rare Disease first partnered with Form Bio to improve its Duchenne therapeutic candidate, which had not yet been optimized for manufacturing or reduced immunogenicity. By combining long-read sequencing, genome integrity analysis, rational design, and AI modeling, Form Bio identified packaging inefficiencies that could have hindered clinical performance. Adjusting the promoter and optimizing the nuclease increased the percentage of AAV full genome to 59 percent from 13 percent—a 4.4-fold jump in therapeutic yield achieved within a month.

“For patients and families facing rare neuromuscular and neurodegenerative diseases, time is critical,” said Richard Horgan, founder and CEO of Cure Rare Disease. “Form Bio’s expertise and technology have already delivered a measurable improvement in our Duchenne program, advancing it to in vivo preclinical studies with significantly greater manufacturability and efficiency. This partnership shows how nonprofit and technology leaders can join forces to speed the development of life-changing therapies for underserved patients.”

Photo: Richard Horgan, founder and CEO of Cure Rare Disease