Cure Rare Disease (CRD), a non-profit biotechnology organization, announced a landmark partnership with the LGMD2L Foundation. This collaboration is backed by a generous commitment of $7.65 million from the LGMD2L Foundation to develop a novel gene replacement therapy for Anoctamin 5 (ANO5)-related disease, a rare genetic disorder.

The transformational partnership will fund a comprehensive, multi-year program dedicated to advancing a potential treatment from initial design to clinical trial readiness. The funding will support a series of critical milestones, including therapeutic design, preclinical studies, manufacturing scale-up, and ultimately a first-in-human clinical trial. This strategic alliance represents a significant step forward in addressing the unmet needs of patients with ANO5-related diseases.

In addition to the gene replacement therapy for Anoctamin 5 (ANO5)-related disease, Cure Rare Disease's pipeline includes the development of two gene replacement therapeutics for LGMD2i/R9 and LGMD2g/R7, among therapies for other neuromuscular and neurodegenerative diseases. These therapeutic candidates utilize a common next-generation AAV capsid, which has the potential to be safer than first-generation capsids.

“This partnership with the LGMD2L Foundation is a testament to the power of collaboration between drug development organizations and advocacy groups in the fight against rare diseases,” said Rich Horgan, CEO of Cure Rare Disease. “This significant funding allows us to deploy our drug development engine to accelerate a promising gene therapy for the LGMD2L community. We are deeply grateful for the LGMD2L Foundation's trust and commitment as we work together to bring a treatment to patients living with LGMD2L and their families.”

“Gene therapy is the most promising therapeutic avenue for this devastating disease, yet no private-sector programs are currently advancing this approach,” said Hal Tily, VP Research for the LGMD2L Foundation. “This partnership marks a pivotal moment for patient-led organizations in shaping their own futures. CRD’s track record navigating the complexities of therapeutic development gives us strong confidence that this investment can change the lives of individuals and families affected by LGMD2L.”

This effort underscores a shared mission to streamline the development of treatments for rare diseases often overlooked by traditional pharmaceutical pipelines, and highlights how committed organizations can come together to make meaningful progress for their communities.