Clinical Trials

Inclusion Criteria: 

• Signed informed consent.
• Ages ≥18 to ≤65 years.
• Diagnosed with HCM per American College of Cardiology (ACC)/American Heart Association (AHA) Guidelines (maximal left ventricular wall thickness >13 mm).
• Symptomatic HCM (New York Heart Association [NYHA] Class II-IV).
• Confirmed Pathogenic/Likely Pathogenic MYBPC3 truncating mutation.

Exclusion Criteria:

• Currently receiving systemic immunosuppressive therapy, cytotoxic chemotherapy, immunoglobulin therapy, or monoclonal antibody therapy.
• History of clinically significant liver disease, HBV, HCV, HIV, or latent tuberculosis infection.
• Previously dosed with any investigational or approved gene therapy product at any time.

Eligibility last updated 4/17/22. Questions regarding updates should be directed to the study team contact.