ROCKET PHARMACEUTICALS (RCKT) Company Report

Rocket Pharmaceuticals is pivoting its focus towards cardiovascular AAV-based gene therapies to optimize capital and resources, while seeking partnerships for its ex vivo Lenti-based hematology programs, with the exception of Kresladi (LAD-I).
The Danon disease program's clinical trial is set to restart in the first half of 2026 after the hold was lifted, with plans to treat at least 12 patients without the complement inhibitor that was associated with previous safety events.
The company has addressed all CMC findings for Kresladi (LAD-I) and submitted its response to the FDA, with a PDUFA date of March 28.
Rocket's BAG3 program, targeting dilated cardiomyopathy, is entering the clinic in 2026.

3 days ago

Transcript

Rocket Pharmaceuticals Discusses AAV Pipeline Focus and Kresladi Approval Timeline

RCKT

New Projects/Investments

Product Launch

Guidance Update

3 days ago

Transcript

Rocket Pharmaceuticals Reports Q3 2025 Financial Results and Operational Progress

RCKT

Earnings

Guidance Update

Management Change

Rocket Pharmaceuticals reported cash, cash equivalents and investments of $222.8 million as of September 30, 2025, with an expected operational runway into the second quarter of 2027.
For the third quarter ended September 30, 2025, the company recorded a net loss of $50.3 million, or $0.45 per share, compared to a net loss of $66.7 million, or $0.71 per share, for the same period in 2024.
Key operational updates include the anticipated resumption of the pivotal Phase 2 trial for RP-A501 for Danon disease in the first half of 2026 and a Prescription Drug User Fee Act (PDUFA) target action date of March 28, 2026, for KRESLADI™ for severe LAD-I.
The company also announced leadership updates with the appointments of Dr. Syed Rizvi as Chief Medical Officer, Christopher Stevens as Chief Operating Officer, and Sarbani Chaudhuri as Chief Commercial & Medical Affairs Officer.

Nov 6, 2025, 9:04 PM

8-K

Rocket Pharmaceuticals' Gene Therapy Application Accepted by FDA

RCKT

Product Launch

Layoffs

New Projects/Investments

The U.S. Food and Drug Administration (FDA) has accepted Rocket Pharmaceuticals' resubmitted Biologics License Application (BLA) for Kresladi, a gene therapy for severe LAD-I, with a decision deadline set for March 28, 2026. This follows an earlier FDA rejection in June 2024.
Kresladi demonstrated 100% survival at 12 months post-treatment in clinical trials, and an analyst projects peak sales of $294 million in 2031 for the therapy.
Rocket Pharmaceuticals, a clinical-stage company with no current revenue, has a strong balance sheet with a current ratio of 6.39 and a debt-to-equity ratio of 0.07. The company plans a 30% workforce reduction to extend its operational runway into Q2 2027 and prioritize its AAV cardiovascular pipeline and Kresladi regulatory efforts.

Oct 14, 2025, 11:25 AM

Morningstar

Rocket Pharmaceuticals Announces FDA Acceptance of KRESLADI™ BLA Resubmission

RCKT

Product Launch

New Projects/Investments

Rocket Pharmaceuticals (RCKT) announced on October 14, 2025, that the FDA accepted the resubmission of its Biologics License Application (BLA) for KRESLADI™ (marnetegragene autotemcel; marne-cel).
KRESLADI™ is an investigational gene therapy for severe Leukocyte Adhesion Deficiency-I (LAD-I), a rare genetic immune disorder.
The FDA has set a PDUFA target action date of March 28, 2026, for the BLA, and Rocket is eligible for a Rare Pediatric Disease Priority Review Voucher (PRV) upon approval.
The BLA is supported by positive clinical data from a Phase 1/2 study, which showed 100% overall survival at 12 months post-infusion for all enrolled patients, with KRESLADI™ being well tolerated and meeting all primary and secondary endpoints.

Oct 14, 2025, 11:09 AM

8-K

Rocket Pharmaceuticals Announces FDA Acceptance of KRESLADI™ BLA Resubmission

RCKT

Product Launch

New Projects/Investments

Rocket Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has accepted the resubmission of the Biologics License Application (BLA) for KRESLADI™ (marnetegragene autotemcel; marne-cel), an investigational gene therapy for severe Leukocyte Adhesion Deficiency-I (LAD-I).
The Prescription Drug User Act (PDUFA) target action date for KRESLADI™ is set for March 28, 2026.
The BLA is supported by positive clinical efficacy and safety data from a global Phase 1/2 study, which demonstrated 100% overall survival at 12 months post-infusion for all enrolled patients, meeting all primary and secondary endpoints.
Rocket Pharmaceuticals is eligible for a Rare Pediatric Disease Priority Review Voucher if KRESLADI™ is approved.

Oct 14, 2025, 11:00 AM

Press Release

Rocket Pharmaceuticals Withdraws Fanconi Anemia Gene Therapy Filing Amid Strategic Shift

RCKT

Layoffs

New Projects/Investments

Guidance Update

Rocket Pharmaceuticals has voluntarily withdrawn its biologics license application for RP-L102 (Fanskya), a gene therapy for Fanconi Anemia.
This decision is due to a strategic shift to focus on programs with clearer regulatory and commercial prospects, following a corporate restructuring in July 2025 that included layoffs and a pivot toward cardiovascular disease programs.
The withdrawal is not related to safety or efficacy concerns, as data continue to show the therapy is generally well-tolerated with a favorable risk-benefit profile.
While internal investment in RP-L102 has ceased, Rocket Pharmaceuticals is exploring external partnerships to advance the therapy.

Oct 3, 2025, 4:14 PM

Morningstar

Rocket Pharmaceuticals Provides Updates on Gene Therapy Portfolio and Danon Program Clinical Hold Lift

RCKT

New Projects/Investments

Guidance Update

Rocket Pharmaceuticals has shifted its focus to its AAV cardiac gene therapy portfolio, which includes programs for Danon, PKP2, and BAG3, collectively representing over 100,000 patients in the U.S. and Europe.
The clinical hold on the Danon program was lifted in less than three months following the identification of capillary leak syndromes linked to a C3 inhibitor. The revised protocol involves a recalibrated dose closer to 4e13, removal of the C3 inhibitor, and a lower threshold for C5 inhibition.
The company plans to treat the next three Danon patients in early 2026, with the FDA not requiring enrollment of more than 12 patients for the pivotal trial.
For the PKP2 program, the prophylaxis regimen is similar to the revised Danon program, and the next milestone is FDA alignment on the Phase 2 pivotal trial design. The BAG3 program is expected to start its Phase 1 trial next year.

Sep 3, 2025, 8:26 PM

Earnings Transcripts

Quarterly earnings call transcripts for ROCKET PHARMACEUTICALS.

Ask Fintool AI Agent

Get instant answers from SEC filings, earnings calls & more

What did Gaurav Shah say about margins?What are ROCKET PHARMACEUTICALS's strategic priorities?How is ROCKET PHARMACEUTICALS positioned vs competitors?How did ROCKET PHARMACEUTICALS do last quarter?

Let Fintool AI Agent track ROCKET PHARMACEUTICALS's earnings for you

Get instant analysis when filings drop