Introduction to LGMD
Facing the Challenge of LGMD: A Rare and Progressive Disease
“MyoGene’s second-generation vector eliminates
liver toxicity risks, solving the safety issues that
led to past gene therapy failures.”
Each year, children and families worldwide receive devastating diagnoses of Limb-Girdle Muscular Dystrophy (LGMD), types 2C and 2D—rare neuromuscular diseases that are both progressive and currently untreatable. Affecting approximately 20,000 people globally, LGMD2C and LGMD2D lead to progressive muscle loss, respiratory failure, and severe mobility challenges, significantly impacting quality of life and longevity.
Our Pipeline and Timeline
Proprietary Vector Acquisition & Development Network
License promising new vector and leverage MyoGene’s superior stakeholder “network” to bridge final development of a unique proprietary vector already in process.
Second-Generation AAV Technology with Enhanced Tropism
Deploy a novel 2nd generation adeno-associated virus (“AAV”) modified to target muscle and avoid the liver and related toxicities.
Complete Gene Replacement Therapeutic Approach
Focus on curative treatment that replaces entire missing gene.
- Target gene size < 4.2 KB, so not Duchenne.
GRASP-LGMD Patient Consortium Integration
Leverage international Genetic Resolution and
Assessments Solving Phenotypes
(“GRASP”)-LGMD consortium of 500+ patients
that affords MyoGene:
- Unique biomarkers and disease progression measurement techniques.
- “Trial Preparedness” with known patients ready to treat now.
- Globally renowned academic and clinical expertise.
About Us
Meet Our Team
Nicholas E. Johnson
MD, MSCI, FAAS
Founder/CEO
Founder/CTO
- VCU Professor; Vice-Chair of Research
- George Bliley Research Chair in Neurology
- Director, Center for Inherited Myology Research
- Director, GRASP-LMGD Consortium
- MD (Arizona), MSCI (Utah), Residency (Rochester)
Melissa Hale
PhD
Co-Founder/CTO
Co-Founder/CTO
- VCU Assistant Professor
- Investigator, Center for Inherited Myology Research
- PhD (Florida)
Kevin L. Passarello,
JD
Co-Founder/COO
Co-Founder/COO
- Partner, Buchanan Ingersoll & RooneyCU Assistant Professor
- Co-Founder/COO—TRADOS (sold to SDL plc)
- Co-Founder/President—Pong Research (sold to L Catterton)
- Co-Founder/CEO—AMP3D (sold to Nihon Kohden)
- Co-Founder—Harvest Equity Partners–$600m+ in exits
- JD (Georgetown)
Ellie Carrell
PhD
Chief Scientific Officer
Contact us
“MyoGene’s second-generation vector eliminates
liver toxicity risks, solving the safety issues that
led to past gene therapy failures.”