Yeji

$RCKT

ROCKET PHARMACEUTICALS, INC.

Runway:10 mo
NasdaqMarket cap: —CIK 0001281895

Upcoming catalysts

4 surfaced
FDA approvalDate TBD~ low confidence
$RCKTROCKET PHARMACEUTICALS, INC.
kresladi · Treatment of pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I) due to biallelic variants in ITGB2 without an available human leukocyte antigen-matched sibling donor for allogeneic hematopoietic stem cell transplant
“The PRV was awarded following the U.S. Food and Drug Administration (FDA) accelerated approval of KRESLADI™ (marnetegragene autotemcel).”
FDA approvalDate TBD~ low confidence
$RCKTROCKET PHARMACEUTICALS, INC.
“the PRV was originally issued in connection with the FDA ffds approval of the Company ffds biologics license application for KRESLADI fffd (marnetegragene autotemcel), an autologous hematopoietic stem cell-based gene therapy indicated for the treatment of pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I) due to biallelic variants in ITGB2 without an available human leukocyte antigen-matched sibling donor for allogeneic hematopoietic stem cell transplant.”
NDA/BLA filedDate TBD
$RCKTROCKET PHARMACEUTICALS, INC.
mozafancogene autotemcel · Fanconi Anemia
“On October 3, 2025, Rocket Pharmaceuticals, Inc. ("Rocket" or the "Company") reported that it has voluntarily withdrawn its Biologics License Application ("BLA") to the U.S. Food and Drug Administration ("FDA") for mozafancogene autotemcel (also known as "Fanskya," "Fanca-cel," or "RP-L102"), an investigational gene therapy for the treatment of Fanconi Anemia ("FA").”
CRLDate TBD
$RCKTROCKET PHARMACEUTICALS, INC.
kresladi · Treatment of pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I) due to biallelic variants in ITGB2 without an available human leukocyte antigen-matched sibling donor for allogeneic hematopoietic stem cell transplant
“The U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) in response to Rocket's Biologics License Application for KRESLADI™ wherein the FDA requested limited additional Chemistry Manufacturing and Controls (CMC) information to complete its review.”

Drug pipeline

kresladi
Treatment of pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I) due to biallelic variants in ITGB2 without an available human leukocyte antigen-matched sibling donor for allogeneic hematopoietic stem cell transplant
mozafancogene autotemcel
Fanconi Anemia
rp-l201
Severe Leukocyte Adhesion Deficiency-I (LAD-I)
Heart Transplant
Danon Disease
RP-L401
Infantile Malignant Osteopetrosis
No intervention
Danon Disease
RP-A501
Danon Disease
RP-A601
PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)
RP-L301
Pyruvate Kinase Deficiency
RP-A701 is a recombinant viral vector composed of an AAV serotype rh.74 (AAVrh.74) capsid encapsulating the transgene, BCL2-associated Athanogene 3 (BAG3)
Dilated Cardiomyopathy (DCM)
RP-L102
Fanconi Anemia Complementation Group A
Safety and efficacy assessments
Fanconi Anemia Complementation Group A

Recent & active trials

NCTDrugPhaseStatusPrimary completion
NCT07527975RP-L102—Enrolling By Invitation2038-05-01
NCT06282432——Active Not Recruiting2036-10-04
NCT04437771Safety and efficacy assessments—Active Not Recruiting2034-01-17
NCT07486752——Not Yet Recruiting2032-12-01
NCT06644742——Recruiting2030-12-01
NCT06214507No intervention—Recruiting2029-12-01
NCT07137338RP-A701 is a recombinant viral vector composed of an AAV serotype rh.74 (AAVrh.74) capsid encapsulating the transgene, BCL2-associated Athanogene 3 (BAG3)Phase 1Recruiting2029-06-01
NCT06422351RP-L301Phase 2Suspended2029-01-01
NCT05885412RP-A601Phase 1Recruiting2028-09-01
NCT06092034RP-A501Phase 2Recruiting2028-04-01
NCT04248439RP-L102Phase 2Active Not Recruiting2026-05-05
NCT04105166RP-L301Phase 1Completed2025-06-09
NCT03882437RP-A501Phase 1Unknown2025-03-24
NCT04069533RP-L102Phase 2Unknown2025-02-01
NCT05548855Heart Transplant—Completed2023-10-31
NCT03812263rp-l201Phase 2Completed2023-09-12
NCT03825783rp-l201Phase 1Withdrawn2021-10-01
NCT04525352RP-L401Phase 1Terminated2021-05-21
NCT03814408RP-L102Phase 1Unknown2020-12-01

Recent catalysts

PDUFAMar 28, 2026· 70d ago· success
$RCKTROCKET PHARMACEUTICALS, INC.
kresladi · Treatment of pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I) due to biallelic variants in ITGB2 without an available human leukocyte antigen-matched sibling donor for allogeneic hematopoietic stem cell transplant
“The PDUFA date set by the FDA is March 28, 2026.”
FDA approvalMar 27, 2026· 71d ago
$RCKTROCKET PHARMACEUTICALS, INC.
kresladi · Treatment of pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I) due to biallelic variants in ITGB2 without an available human leukocyte antigen-matched sibling donor for allogeneic hematopoietic stem cell transplant
“the U.S. Food and Drug Administration (FDA) has approved KRESLADI™ (marnetegragene autotemcel), an autologous hematopoietic stem cell-based gene therapy indicated for the treatment of pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I) due to biallelic variants in ITGB2 without an available human leukocyte antigen-matched sibling donor for allogeneic hematopoietic stem cell transplant.”
NDA/BLA filedOct 14, 2025· 235d ago· unknown
$RCKTROCKET PHARMACEUTICALS, INC.
kresladi · Treatment of pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I) due to biallelic variants in ITGB2 without an available human leukocyte antigen-matched sibling donor for allogeneic hematopoietic stem cell transplant
“On October 14, 2025, Rocket Pharmaceuticals, Inc. (the \”
CRLOct 3, 2025· 246d ago
$RCKTROCKET PHARMACEUTICALS, INC.
mozafancogene autotemcel · Fanconi Anemia
“On October 3, 2025, Rocket Pharmaceuticals, Inc. (”
PDUFAJun 30, 2024· 706d ago· failure
$RCKTROCKET PHARMACEUTICALS, INC.
kresladi · Treatment of pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I) due to biallelic variants in ITGB2 without an available human leukocyte antigen-matched sibling donor for allogeneic hematopoietic stem cell transplant
“the U.S. Food and Drug Administration (FDA) has extended the Priority Review period for the Biologics License Application (BLA) for KRESLADI™ (marnetegragene autotemcel) for severe Leukocyte Adhesion Deficiency-I (LAD-I) by three months, to June 30, 2024, to allow additional time to review clarifying Chemistry, Manufacturing, and Controls (CMC) information submitted by Rocket in response to FDA information requests.”
CRLJun 28, 2024· 708d ago
$RCKTROCKET PHARMACEUTICALS, INC.
kresladi · Treatment of pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I) due to biallelic variants in ITGB2 without an available human leukocyte antigen-matched sibling donor for allogeneic hematopoietic stem cell transplant
“The U.S. Food and Drug Administration ("FDA") has issued a Complete Response Letter ("CRL") in response to the Company's Biologics License Application ("BLA") for KRESLADI™ wherein the FDA requested limited additional Chemistry Manufacturing and Controls ("CMC") information to complete its review.”
PDUFAMar 31, 2024· 797d ago
$RCKTROCKET PHARMACEUTICALS, INC.
rp-l201 · Severe Leukocyte Adhesion Deficiency-I (LAD-I)
“The PDUFA date set by the FDA is March 31, 2024.”
NDA/BLA filedOct 2, 2023· 978d ago· unknown
$RCKTROCKET PHARMACEUTICALS, INC.
kresladi · Treatment of pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I) due to biallelic variants in ITGB2 without an available human leukocyte antigen-matched sibling donor for allogeneic hematopoietic stem cell transplant
“On October 2, 2023, Rocket Pharmaceuticals, Inc. (the ”