Welcome to our dedicated page for Rocket Pharmaceu news (Ticker: RCKT), a resource for investors and traders seeking the latest updates and insights on Rocket Pharmaceu stock.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is a fully integrated, late-stage biotechnology company developing investigational genetic therapies for rare and often life-threatening disorders. The RCKT news feed on Stock Titan aggregates company announcements, press releases and third-party coverage related to its AAV and lentiviral gene therapy programs.
Investors following Rocket’s news can track updates on its adeno-associated viral (AAV) cardiovascular portfolio, which includes RP-A501 for Danon disease, RP-A601 for PKP2-arrhythmogenic cardiomyopathy (PKP2-ACM) and RP-A701 for BAG3-associated dilated cardiomyopathy (BAG3-DCM). News items frequently cover clinical trial milestones, such as FDA lifting a clinical hold on the pivotal Phase 2 trial of RP-A501, preliminary Phase 1 data for RP-A601, and IND clearance and Fast Track designation for RP-A701.
The news stream also highlights developments in Rocket’s lentiviral (LV) hematology portfolio, including regulatory progress for KRESLADI (marnetegragene autotemcel; marne-cel) in severe leukocyte adhesion deficiency-I (LAD-I). Articles may discuss FDA acceptance of the resubmitted Biologics License Application (BLA), the setting of a Prescription Drug User Fee Act (PDUFA) target action date, and clinical data showing survival and infection outcomes in treated patients.
Beyond pipeline updates, RCKT news includes financial results, strategic corporate reorganization and pipeline prioritization announcements, leadership and board changes, equity inducement grants and participation in major healthcare investor conferences. By reviewing this page regularly, readers can follow how Rocket’s clinical, regulatory and corporate events may influence sentiment around RCKT stock and gain context on the company’s progress in gene therapy for rare cardiovascular and hematologic diseases.
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.
Rocket Pharmaceuticals (NASDAQ: RCKT), a late-stage biotechnology company focused on genetic therapies for rare disorders, has announced its participation in two major healthcare conferences in September 2025.
CEO Gaurav Shah, M.D. will participate in fireside chats at the 2025 Cantor Fitzgerald Global Healthcare Conference on September 3 at 3:20 PM ET and the Morgan Stanley 23rd Annual Global Healthcare Conference on September 9 at 9:15 AM ET. Management will also host investor meetings at both events. Webcasts will be available on the company's investor relations website.
Rocket Pharmaceuticals (NASDAQ:RCKT) announced the FDA has lifted the clinical hold on its pivotal Phase 2 trial of RP-A501 for Danon disease treatment in under three months. The trial will resume with a recalibrated lower dose of 3.8 x 10¹³ GC/kg in three patients, treated sequentially with four-week intervals.
The modified protocol includes an adjusted immunomodulatory regimen similar to the Phase 1 pediatric cohort, removing prophylactic C3 complement inhibitor while maintaining other components. Six patients have been treated to date in the Phase 2 study. The trial's co-primary endpoints include LAMP2 protein expression improvements and left ventricular mass reductions, with troponin change as a key secondary endpoint.