Form Bio, a leader in AI-powered genetic medicine development and genome engineering, announced a strategic partnership with Cure Rare Disease (CRD), a nonprofit biotechnology company developing genetic therapies for the treatment of rare and ultra-rare neuromuscular and neurodegenerative diseases. Together, the organizations will accelerate programs to identify and optimize multiple lead indications, beginning with Duchenne muscular dystrophy (DMD).
Through this collaboration, Form Bio will conduct AI research using its proprietary in silico platform to support CRD in developing gene therapy drug candidates optimized for packaging, expression, safety, and manufacturability, critical factors in ensuring clinical success and cost reduction. The program is designed to streamline lead candidate selection, accelerating development timelines and advancing outcomes for patients with devastating neuromuscular diseases that currently lack treatment options.
CRD initially partnered with Form Bio to improve a DMD therapeutic candidate that was not yet optimized for manufacturing or reduced immunogenicity. Using long-read sequencing, genome integrity analysis, rational design, and AI/ML models, Form Bio identified packaging inefficiencies that could have inhibited clinical success. By modifying the promoter and optimizing the nuclease, Form Bio improved the percentage of AAV full genome from 13% to 59%, a 4.4-fold increase in therapeutic yield achieved within just one 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 DMD program, advancing it to in vivo preclinical studies with significantly greater manufacturability and efficiency. This partnership represents a powerful example of how nonprofits and technology leaders can join forces to accelerate the development of life-changing therapies for overlooked populations.”
“Our mission at Form Bio is to empower partners like Cure Rare Disease with our AI-driven insights that transform biology into engineered, programmable systems,” said Michelle Chen, Ph.D., President and CEO of Form Bio. “By optimizing AAV vector design upfront, we can reduce time, cost, and risk across the entire gene therapy development pipeline. We are proud to enable CRD in bringing much-needed therapies closer to patients who currently have no options.”
Read More: Clinical Trials for Duchenne (List of All Researches)
Together, Form Bio and CRD are demonstrating how advanced computational tools and patient-focused biotech innovation can accelerate breakthroughs, reduce costs, and bring safer, more effective therapies to patients with urgent unmet needs.
