Cure Rare Disease Awarded $7.4 Million CIRM Grant to Advance Gene Therapy for Limb-Girdle Muscular Dystrophy Type 2i/R9

The program is designed with a novel, liver-detargeting, muscle-tropic capsid intended to improve safety and efficacy

WOODBRIDGE, Conn.--(BUSINESS WIRE)--Cure Rare Disease (CRD) announced it has been awarded a $7.4 million grant from the California Institute for Regenerative Medicine (CIRM) to advance the development of a novel gene therapy for Limb-Girdle Muscular Dystrophy Type 2i/R9 (LGMD2i/R9), a progressive neuromuscular disorder with limited treatment options.

Cure Rare Disease launched its LGMD2i/R9 program to develop a therapeutic solution for patients suffering from this rare condition. The program, CRD-003, is an investigational gene therapy designed with a novel, liver-detargeting, muscle-tropic capsid, AAVMYO2, intended to improve safety and efficacy by directing the therapy specifically to muscle tissue.

In earlier developmental stages, the Cure Rare Disease team and its collaborators have generated promising preclinical data. The organization has successfully completed a pre-IND meeting with the U.S. Food and Drug Administration (FDA) and secured Orphan Drug Designation (ODD) for the therapy, establishing a clear regulatory pathway for future clinical development.

With CIRM funding support, Cure Rare Disease will advance the program through late-stage preclinical development, GMP manufacturing, IND submission and Phase 1/2 clinical trial initiation. The project will be led by Principal Investigator Dr. Tahseen Mozaffar.

"This program is a powerful example of what can be achieved when patients, organizations, researchers, and governing bodies come together with a shared mission. From the initial support of our community to our collaborations with leading scientists and regulatory guidance from the FDA, the development of a therapy for LGMD2i/R9 has been a truly unified effort,” said Cure Rare Disease CEO and founder Richard Horgan. “We are incredibly appreciative of CIRM's support in helping us take this program closer to the clinic, and incredibly excited at the opportunity of bringing a second-generation AAV capsid into the clinic."

“This CIRM funding represents a critical moment of hope for the LGMD2I/R9 community,” said Kelly Brazzo, Co-Founder and CEO of CureLGMD2i, and mother to a child living with LGMD2I/R9. “After a year marked by funding uncertainty and broader innovation challenges in the rare disease space, this investment reaffirms that our community is not forgotten. The technology also demonstrates how advances in one subtype can inform safer, more effective approaches across multiple forms of LGMD—particularly as patients continue to need safer gene therapies that de-target the liver.”

About Limb-Girdle Muscular Dystrophy Type 2i/R9 (LGMD2i/R9)

Limb-Girdle Muscular Dystrophy Type 2i/R9 is a rare form of muscular dystrophy caused by mutations in the FKRP gene. The disease is characterized by progressive muscle weakness and wasting. Over time, it can lead to significant mobility issues and respiratory complications. There are currently no approved treatments for LGMD2i/R9, making the development of effective genetic medicines a critical unmet need.

About Cure Rare Disease (CRD)

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 the California Institute for Regenerative Medicine (CIRM)

CIRM was created by the people of California to fund stem cell and gene therapy research with the goal of accelerating treatments for patients with unmet medical needs. With $5.5 billion in funding allocated through both Proposition 71 in 2004 and Proposition 14 in 2020, CIRM supports stem cell and gene therapy discoveries from inception through clinical trials, trains a workforce in California to fill jobs in the state’s thriving biotech and biomedical research industry, and creates infrastructure to make clinical trials accessible for people throughout California. All of CIRM’s research, workforce development, and infrastructure programs are designed to benefit the people of California, whose vision created the agency. For more information, visit www.cirm.ca.gov.