Therapeutic Candidate or Device

RP-A501, a recombinant Adeno-Associated Virus Serotype 9 containing the LAMP2B transgene

Indication

Danon Disease

Therapeutic Mechanism

The proposed product, RP-A501, is an in vivo gene therapy consisting of a recombinant AAV9 vector engineered with genetic elements designed to ensure consistent delivery and expression of the LAMP2B transgene. The therapy is administered intravenously as a single dose, and due to the tropism of the AAV9 vector (for cardiac tissue), is able to transduce and cause those cells to sustainably produce LAMP2B protein.

Unmet Medical Need

RP-A501, if successful, will address the unmet clinical need by serving as a disease-modifying therapy with curative potential for Danon Disease patients at high risk for disease complications.

Project Objective

Phase 2 Trial Completion

Major Proposed Activities

• Manufacture product to supply the proposed Phase 2 trial at UCSD Medical Center
• Assess clinical safety and preliminary efficacy of the therapeutic candidate
• Coordinate various CROs to support the clinical trial the entire patient journey, including follow-up visits

Rocket Pharmaceuticals is developing RP-A501 under collaboration with the University of California San Diego School of Medicine. UC San Diego Health will be the initial and lead center for the planned Phase 2 trial under the direction of Dr. Barry Greenberg, M.D., Director, Advanced Heart Failure Treatment Program at UC San Diego Health. UCSD Health will be a leader in gene therapy for heart failure, employing dozens of Californians to support research and clinical trials in this new fi eld.