C3 Scientific Meeting Recap

Coalition to Cure Calpain 3 (C3) recognizes that collaboration and data sharing are integral to drug discovery and therapy development. To foster these interactions, we hosted an in-person meeting focused on advances in clinical trial readiness and treatment development for Calpainopathy (limb-girdle muscular dystrophy types 2A/R1 and 1I/D4). This small, focused meeting was held September 14-15, 2024, in Arlington, Virginia.

Attendance was by invitation only, and brought together 34 prominent neuromuscular clinicians, world renowned research scientists from academia and industry, and representatives from funding agencies. Acknowledging the importance of bringing new researchers into this field, early career investigators were also included. Attendees with a connection to C3 included active Scientific Advisory Board members, research grant recipients, and members of our leadership team who could speak to the patient experience.

The meeting was structured around active discussion, with participation encouraged by all in attendance. Ample time was reserved for questions/answers after each presentation, and the final session was dedicated to a robust and productive whole group discussion. C3 Co-founder Michele Wrubel noted, “The feedback we heard across the board was appreciation for the collaborative and interactive nature of the meeting. This was particularly gratifying as our mission has always been to engage a ‘coalition’ of researchers, clinicians, drug developers, and patients to work together to tackle the challenge of finding a treatment or cure for Calpainopathy.”

Session topics focused on:

• Genetic Testing and the LGMD2A/Calpainopathy Registry
• Outcomes Assessments and Biomarkers
• Preclinical Models
• Novel Targets
• Cell and Gene Therapies

A primary deliverable of the meeting was to set forth concrete recommendations that are actionable by C3 to enable the evaluation of potential therapeutic approaches and to generate interest in Calpainopathy research with drug developers. Topics identified for further exploration include:

• Development of a Calpain 3 activity assay
• Identification of small molecule strategies
• Investigation into the genetics and pathophysiology of autosomal dominant Calpainopathy
• Development of serum and imaging biomarkers
• Support of trainees to foster a future generation of Calpainopathy experts

“I’m coming away from this meeting feeling optimistic and energized about the progress being made. There is a real feeling of momentum moving us closer towards a cure”

C3 President Jordan Boslego

Learnings from the meeting will guide our strategies as we prepare to fund new research projects beginning in 2025, with a goal to accelerate the development of transformative treatments to improve the quality of life for individuals living with Calpainopathy.