Rocket Pharmaceuticals, Inc. reports developments in rare-disease genetic medicines, including its commercial-stage therapy KRESLADI for pediatric patients with severe leukocyte adhesion deficiency-I and its broader pipeline of investigational gene therapies. Company updates commonly address FDA actions, clinical-program progress, regulatory submissions, and financial results tied to research, development, and commercial readiness.
Recurring news themes include the AAV cardiovascular portfolio, such as RP-A501 for Danon disease, RP-A601 for PKP2-arrhythmogenic cardiomyopathy, and RP-A701 for BAG3-related dilated cardiomyopathy. Rocket also reports capital and balance-sheet actions, including monetization of its Rare Pediatric Disease Priority Review Voucher, as well as presentations at healthcare conferences.
Rocket Pharmaceuticals (NASDAQ: RCKT) reported Q1 2026 results and operational updates on May 7, 2026. Key items: KRESLADI received FDA accelerated approval for severe LAD-I, the company monetized a $180 million PRV, pro forma cash is ~$322.6M, and dosing resumed in the pivotal Phase 2 RP-A501 Danon study.
Rocket expects cash runway into Q2 2028 and plans a Danon program update in H2 2026; first patient dosing for RP-A701 is anticipated mid-2026.
Rocket Pharmaceuticals (NASDAQ: RCKT) entered a definitive agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for $180 million. Proceeds are non-dilutive and will support advancement of prioritized cardiovascular gene therapy programs. Rocket says the sale extends its cash runway into Q2 2028.
The PRV was awarded after FDA accelerated approval of KRESLADI (marnetegragene autotemcel). The PRV program was reauthorized in February 2026.
Rocket Pharmaceuticals (NASDAQ: RCKT) announced FDA accelerated approval of KRESLADI (marnetegragene autotemcel) for pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I) due to biallelic ITGB2 variants without an HLA-matched sibling donor.
The approval is based on increased neutrophil CD18/CD11a expression and carries a Rare Pediatric Disease Priority Review Voucher; continued approval requires confirmatory long-term clinical benefit data.
Rocket Pharmaceuticals (NASDAQ: RCKT) reported Q4 and full-year 2025 results and operational updates. Cash, cash equivalents and investments were $188.9 million, with expected runway into the second quarter of 2027. Net loss improved to $223.1 million ($2.01/share).
Key pipeline milestones: RP-A501 pivotal Phase 2 to resume in 1H 2026; KRESLADI™ BLA resubmission accepted with a PDUFA date of March 28, 2026; RP-A701 Phase 1 dosing expected mid-2026; ongoing FDA engagement for RP-A601.
Rocket Pharmaceuticals (NASDAQ: RCKT) announced participation in two investor conferences in early March 2026. Management will host meetings and CEO Gaurav Shah will join a fireside chat.
Conference dates: TD Cowen on March 2, 2026 at 11:10 AM ET and Leerink on March 10, 2026 at 9:20 AM ET. Webcasts will be available on the company's investor website.
Rocket Pharmaceuticals (NASDAQ: RCKT) will participate in the 44th Annual J.P. Morgan Healthcare Conference in San Francisco.
Gaurav Shah, M.D., CEO, is scheduled to present on Wednesday, January 14, 2026 at 11:15 a.m. PT and will host investor meetings during the conference. A webcast of the presentation will be available on the company’s Investors website.
Rocket Pharmaceuticals (NASDAQ: RCKT) will participate in the 8th Annual Evercore Healthcare Conference in Miami on December 2, 2025.
Gaurav Shah, M.D., chief executive officer, will appear in a fireside chat on Tuesday, December 2 at 7:55 a.m. ET and will host investor meetings. A webcast of the presentation will be available on the company’s Investors website.
Rocket Pharmaceuticals (NASDAQ: RCKT) reported Q3 2025 results and program updates on November 6, 2025. Cash, cash equivalents and investments were $222.8M, which the company expects will fund operations into the second quarter of 2027 (excluding any Priority Review Voucher proceeds).
Regulatory and clinical milestones include FDA acceptance of a resubmitted BLA for KRESLADI with a PDUFA date of March 28, 2026, FDA agreement to resume the pivotal Phase 2 trial of RP-A501 for Danon disease with three additional patients dosed at 3.8×10^13 GC/kg in 1H 2026, and ongoing development of RP-A601 and RP-A701. Leadership additions were announced for CMO, COO, and Chief Commercial & Medical Affairs.
Rocket Pharmaceuticals (NASDAQ: RCKT) announced the FDA has accepted the resubmission of the Biologics License Application for KRESLADI™ (marnetegragene autotemcel) for severe Leukocyte Adhesion Deficiency-I (LAD-I).
The Prescription Drug User Fee Act (PDUFA) target action date is March 28, 2026. The BLA is supported by global Phase 1/2 data showing 100% overall survival at 12 months, met all primary and secondary endpoints, demonstrated reductions in significant infections, improvement in skin lesions and wound healing, and was well tolerated with no treatment-related serious adverse events. Rocket is eligible for a Rare Pediatric Disease Priority Review Voucher if approved.
Rocket Pharmaceuticals (NASDAQ: RCKT) said on October 6, 2025 its Compensation Committee approved inducement equity awards for new Chief Medical Officer Dr. Syed Rizvi as a hiring incentive under Nasdaq Rule 5635(c)(4).
The grants include a nonstatutory stock option to buy 383,854 shares (10-year term, exercise price = Nasdaq close on Oct 6, 2025), 283,018 restricted stock units (On-Hire RSUs) and 62,893 additional RSUs. Vesting: the option and On-Hire RSUs vest 1/3 at one year then quarterly over two years; Additional RSUs vest 50% at one year and 50% one year later. Grants were approved as a material inducement to employment.