Summary of Rocket Pharmaceuticals FY Conference Call Company Overview - **Company**: Rocket Pharmaceuticals (NasdaqGM:RCKT) - **Industry**: Gene Therapy - **Focus**: Two platforms - AAV-based therapies for cardiovascular diseases and ex vivo Lenti-based therapies for hematology [2][3] Core Points and Arguments Clinical Programs - **Clinical Programs**: Three hematology programs (LAD-I, Fanconi anemia, pyruvate kinase deficiency) and three cardiovascular programs (Danon disease, PKP2, BAG3) [2] - **AAV Portfolio Focus**: Increased focus on AAV programs due to high unmet need and potential for value creation [2][3] Danon Disease Program - **Trial Resumption**: The hold on the Danon program was lifted rapidly; trial will restart in the first half of 2026 with a staggered dosing approach [5][6] - **Patient Safety**: Previous safety events led to the removal of complement inhibitors from the trial design [5][6] - **Regulatory Alignment**: Ongoing positive interactions with the FDA regarding trial design and patient count [12][13] PKP2 Program - **Trial Design Discussions**: Ongoing discussions with the FDA about trial design; emphasis on a robust design that demonstrates clear benefit-risk [19][20] - **Endpoints Consideration**: Potential for composite endpoints due to the heterogeneity of the disease [21][22] BAG3 Program - **Clinical Entry**: The BAG3 program is set to enter the clinic next year, focusing on dilated cardiomyopathy (DCM) with traditional endpoints [25][26] - **Predictability**: DCM is better characterized, allowing for more predictable outcomes [25] Lenti-based Therapies - **Focus Shift**: A strategic decision to focus on AAV programs while seeking partnerships for Lenti-based therapies like Fanconi anemia [30][31] - **PRV Eligibility**: All Lenti programs (LAD, Fanconi, PKD, Danon) are eligible for Priority Review Vouchers (PRV), providing potential non-dilutive cash sources [34] CMC and Regulatory Updates - **CMC Findings**: Previous CMC findings have been addressed, and a resubmission to the FDA has been made, with hopes for approval by the PDUFA date of March 28 [35] Other Important Insights - **Community Engagement**: Strong commitment to patient advocacy and community engagement, especially following safety events [16][17] - **Market Opportunity**: The company is preparing for a comprehensive update on trial progress and market opportunity once clarity is achieved [10][15] This summary encapsulates the key points discussed during the conference call, highlighting Rocket Pharmaceuticals' strategic focus, clinical program updates, regulatory interactions, and community engagement efforts.