Cure Rare Disease and LGMD2L Foundation Announce Multi-Year Partnership to Develop Gene Therapy for Anoctamin 5-Related Disease

WOODBRIDGE, Conn.--(BUSINESS WIRE)--Cure Rare Disease (CRD), a non-profit biotechnology organization, today 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.

About Cure Rare Disease

Cure Rare Disease (CRD) is a nonprofit biotechnology company focused on developing genetic medicines for rare and ultra-rare diseases. CRD is committed to accelerating the development of treatments for individuals with rare diseases through a patient-centric approach to research and development. The company's programs include gene therapy and antisense oligonucleotide (ASO) treatments for a range of rare diseases. For more information, visit www.cureraredisease.org.

About LGMD2L Foundation

The LGMD2L Foundation was established in 2018 to unite people afflicted by this rare disease, raise awareness, and provide a forum to connect, discuss, and build community. In 2024, the organization expanded its scope to begin actively funding research towards treatments. Its mission is to work together as a community to improve the lives of those affected by LGMD2L, accelerate the development of effective treatments, and ultimately find a cure. For more information, visit www.lgmd2l-foundation.org.

About LGMD2L

Limb Girdle Muscular Dystrophy type 2L or R12 is a rare genetic disorder that causes progressive muscle weakness. It primarily affects the muscles around the shoulders, upper arms, and thighs, known as the limb girdle muscles. These muscles play a key role in basic activities like walking, lifting, and standing. As the disease progresses, healthy muscle tissue degenerates and is replaced by non-functional tissue, such as fat and scar tissue. This degeneration impairs mobility and muscle strength. The condition is caused by mutations in the ANO5 gene, which is responsible for producing a protein that is vital to muscle cell function and repair.