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. 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 a positive safety update from the initial three patients treated with gene therapy RP-A501 under a modified protocol in its global, pivotal Phase 2 Danon disease trial. Patients received a recalibrated single dose of 3.8 × 10¹³ GC/kg plus a refined immunomodulatory regimen of rituximab, sirolimus and corticosteroids, administered sequentially with at least four weeks between infusions.
According to Rocket Pharmaceuticals, no thrombotic microangiopathy, capillary leak syndrome or other significant safety concerns have been seen in these patients so far. The 12‑patient, single‑arm Phase 2 trial remains pivotal, and the company is actively engaging with the U.S. FDA to align on dosing additional patients and completing the trial, with a regulatory pathway update and a broader Danon disease program update expected in the second half of 2026.
Rocket Pharmaceuticals (NASDAQ:RCKT) closed the sale of its FDA-granted Rare Pediatric Disease Priority Review Voucher for $180 million in gross proceeds.
The voucher was awarded after March 2026 approval of KRESLADI for LAD-I. Pro forma cash rose from $144.4 million to about $322.6 million, which Rocket expects will fund operations into Q2 2028.
Rocket Pharmaceuticals (NASDAQ:RCKT) will participate in two upcoming healthcare investor conferences in June 2026. CEO Gaurav Shah, M.D., will join fireside chats and management will host investor meetings at both events.
Live webcasts will be accessible via the Investors section of the company’s website.
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.