March 03, 2026-
Cure Rare Disease (CRD) has announced a landmark partnership with the LGMD2L Foundation, backed by a $7.65 million funding commitment to develop a novel gene replacement therapy for Anoctamin 5 (ANO5)-related disease, also known as LGMD2L.
The multi-year collaboration will support a comprehensive development program designed to advance the therapy from initial construct design through clinical trial readiness. Funding will cover therapeutic design, preclinical studies, manufacturing scale-up, and progression into a first-in-human clinical trial.
ANO5-related disease is a rare genetic form of limb-girdle muscular dystrophy caused by mutations in the ANO5 gene. Currently, no approved disease-modifying therapies exist, and no private-sector gene therapy programs are actively advancing treatments for this indication.
CRD’s broader pipeline includes gene replacement programs targeting LGMD2i/R9 and LGMD2g/R7, as well as additional neuromuscular and neurodegenerative disorders. These programs utilize a next-generation AAV capsid platform designed to improve safety and performance compared to earlier-generation vectors.
Company and foundation leaders emphasized the importance of collaboration between nonprofit drug developers and patient advocacy organizations, particularly in rare diseases that have historically lacked commercial investment. The partnership reflects a growing model in which patient-led foundations directly fund and shape therapeutic development efforts.
