Cure Rare Disease Secures $7.4 Million CIRM Grant for Novel Gene Therapy Targeting LGMD2i/R9
核心洞察
Cure Rare Disease (搜索) received a $7.4 million grant from the California Institute for Regenerative Medicine (搜索) to advance CRD-003 (搜索), a gene therapy for Limb-Girdle Muscular Dystrophy Type 2i/R9 (搜索).
The investigational therapy features a novel liver-detargeting, muscle-tropic capsid called AAVMYO2 (搜索) designed to improve safety and efficacy by directing treatment specifically to muscle tissue.
The company has already secured FDA Orphan Drug Designation and completed a pre-IND meeting, establishing a clear regulatory pathway for clinical development.
Cure Rare Disease (搜索) (CRD) has been awarded a $7.4 million grant from the California Institute for Regenerative Medicine (搜索) (CIRM (搜索)) to advance development of CRD-003 (搜索), a novel gene therapy for Limb-Girdle Muscular Dystrophy Type 2i/R9 (搜索) (LGMD2i/R9 (搜索)), a progressive neuromuscular disorder with limited treatment options.
The investigational gene therapy is designed with a novel, liver-detargeting, muscle-tropic capsid called AAVMYO2 (搜索), intended to improve safety and efficacy by directing the therapy specifically to muscle tissue. This approach represents a second-generation AAV capsid technology that addresses safety concerns associated with liver targeting in gene therapies.
Regulatory Progress and Clinical Pathway
The Cure Rare Disease (搜索) team and its collaborators have generated promising preclinical data in earlier developmental stages. 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 (搜索) funding support, Cure Rare Disease (搜索) 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.
Addressing Critical Unmet Medical Need
Limb-Girdle Muscular Dystrophy Type 2i/R9 (搜索) is a rare form of muscular dystrophy (搜索) caused by mutations in the FKRP (搜索) 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 (搜索), making the development of effective genetic medicines a critical unmet need.
"This program is a powerful example of what can be achieved when patients, organizations, researchers, and governing bodies come together with a shared mission," said Cure Rare Disease (搜索) CEO and founder Richard Horgan. "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 (搜索) has been a truly unified effort. We are incredibly appreciative of CIRM (搜索)'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."
Community Impact and Broader Implications
The funding announcement comes after a challenging period for rare disease innovation. Kelly Brazzo, Co-Founder and CEO of CureLGMD2i (搜索), and mother to a child living with LGMD2I/R9 (搜索), emphasized the significance of the investment: "This CIRM (搜索) funding represents a critical moment of hope for the LGMD2I/R9 community. 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."
Brazzo also highlighted the broader therapeutic potential of the technology: "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."
The California Institute for Regenerative Medicine (搜索) 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 (搜索) supports stem cell and gene therapy discoveries from inception through clinical trials.