Earnings summaries and quarterly performance for ROCKET PHARMACEUTICALS.
Executive leadership at ROCKET PHARMACEUTICALS.
Gaurav Shah
Detailed
Chief Executive Officer
CEO
AO
Aaron Ondrey
Detailed
Chief Financial Officer
CS
Chris Stevens
Detailed
Chief Operating Officer
JM
John Militello
Detailed
Vice President of Finance, Treasurer, Principal Accounting Officer
JS
Jonathan Schwartz
Detailed
Chief Medical & Gene Therapy Officer
MW
Martin Wilson
Detailed
General Counsel and Chief Corporate Officer, Senior Vice President
SC
Sarbani Chaudhuri
Detailed
Chief Commercial & Medical Affairs Officer
Board of directors at ROCKET PHARMACEUTICALS.
Research analysts who have asked questions during ROCKET PHARMACEUTICALS earnings calls.
DG
Dae Gon Ha
Stifel
1 question for RCKT
Also covers: ABEO, BEAM, CRBP +10 more
DH
David Hoang
Citigroup
1 question for RCKT
Also covers: ACAD, ALKS, AVDL +8 more
Eric Joseph
JPMorgan Chase & Co.
1 question for RCKT
Also covers: BEAM, BLUE, ENTA +9 more
Gil Blum
Needham & Company
1 question for RCKT
Also covers: ABSI, AUTL, CELC +9 more
JS
Josh Schimmer
Cantor Fitzgerald
1 question for RCKT
Also covers: BBIO, BYSI, CRNX +3 more
MF
Mani Foroohar
Leerink Partners
1 question for RCKT
Also covers: ADVM, ALNY, ARWR +9 more
Patrick Dolezal
LifeSci Capital
1 question for RCKT
Also covers: ADVM, LRMR, VYNE
RP
Raju Prasad
William Blair
1 question for RCKT
Also covers: BOLD
YW
Yaron Werber
TD Cowen
1 question for RCKT
Also covers: ALEC, AMGN, ARGX +14 more
Recent press releases and 8-K filings for RCKT.
Rocket Pharmaceuticals Provides Strategic Update and Pipeline Progress at J.P. Morgan Healthcare Conference
RCKT
New Projects/Investments
Guidance Update
Product Launch
- Rocket Pharmaceuticals reported $222.8 million in cash, cash equivalents, and restricted cash as of September 30, 2025, which is expected to fund operations into Q2 2027, excluding any potential sale of a Priority Review Voucher (PRV).
- The company is strategically refocusing on cardiovascular gene therapies, deprioritizing internal investments in Fanconi anemia and PKD while seeking partner options for these programs.
- A key near-term catalyst is the potential approval and commercialization of Kresladi (for severe LAD-1) by March 28, 2026, which could yield a PRV and extend the cash runway.
- Clinical progress includes resuming dosing for the Danon disease program in H1 2026 with a recalibrated dose, initiating a Phase I trial for BAG3 DCM in 2026, and defining the registrational pathway for PKP2 ACM with the FDA.
Jan 14, 2026, 7:15 PM
Rocket Pharmaceuticals Outlines 2026 Strategic Priorities and Financial Runway
RCKT
Guidance Update
New Projects/Investments
Product Launch
- Rocket Pharmaceuticals reported $222.8 million in cash, cash equivalents, and restricted cash as of September 30, 2025, which is expected to fund operations into the second quarter of 2027, excluding any potential Priority Review Voucher (PRV) sale.
- The company has refocused its pipeline on cardiovascular gene therapies, deprioritizing internal investments in Fanconi anemia and PKD while seeking partner options for these programs.
- Key catalysts for 2026 include the PDUFA date for Kresladi (severe LAD1) on March 28, 2026, the resumption of dosing for the Danon disease pivotal phase 2 study in the first half of 2026, and the initiation of the first-in-human phase 1 trial for BAG3 DCM.
Jan 14, 2026, 7:15 PM
Rocket Pharmaceuticals outlines strategic focus on cardiovascular gene therapies and 2026 milestones
RCKT
New Projects/Investments
Guidance Update
Product Launch
- Rocket Pharmaceuticals is strategically refocusing on its AAV platform for cardiovascular gene therapies, including Danon disease, PKP2-ACM, and BAG3 DCM, while deprioritizing internal investments in Fanconi anemia and PKD.
- As of September 30, 2025, the company held $222.8 million in cash, cash equivalents, and restricted cash, projected to fund operations into Q2 2027, with potential for further extension from a Priority Review Voucher (PRV) upon Kresladi approval.
- Key 2026 milestones include the March 28, 2026, PDUFA date for Kresladi (severe LAD1), resuming dosing in the pivotal phase 2 trial for Danon disease in the first half of the year, initiating the phase 1 study for BAG3 DCM, and seeking FDA alignment for the PKP2-ACM pivotal phase 2 study.
- Rocket maintains in-house AAV manufacturing capabilities at its 100,000 sq ft facility in Cranbury, New Jersey, supporting its current and future commercial programs.
Jan 14, 2026, 7:15 PM
Rocket Pharmaceuticals Provides Clinical Program Updates and Strategic Focus
RCKT
New Projects/Investments
Guidance Update
Product Launch
Dec 2, 2025, 12:55 PM
Rocket Pharmaceuticals Provides Updates on Gene Therapy Programs and Strategic Focus
RCKT
New Projects/Investments
Guidance Update
Product Launch
- 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.
Dec 2, 2025, 12:55 PM
Rocket Pharmaceuticals Discusses AAV Pipeline Focus and Kresladi Approval Timeline
RCKT
New Projects/Investments
Product Launch
Guidance Update
Dec 2, 2025, 12:55 PM
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
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
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
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
Quarterly earnings call transcripts for ROCKET PHARMACEUTICALS.
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