Rocket Pharmaceuticals, Inc. (RCKT)
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Rocket Pharmaceuticals, Inc., together with its subsidiaries, operates as a multi-platform biotechnology company that focuses on developing gene therapies for rare and devastating diseases. It has three clinical-stage ex vivo lentiviral vector programs for fanconi anemia, a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells; leukocyte adhesion deficiency-I, a genetic disorder that causes the immune system to malfunction; and pyruvate kinase deficiency, a rare red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia. The company also has a clinical stage in vivo adeno-associated virus program for Danon disease, a multi-organ lysosomal-associated disorder leading to early death due to heart failure. It has license agreements with Fred Hutchinson Cancer Research Center; Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT), Centro de Investigacion Biomedica En Red, and Fundacion Instituto de investigacion Sanitaria Fundacion Jimenez Diaz; CIEMAT and UCL Business PLC; The Regents of the University of California; and REGENXBIO, Inc. Rocket Pharmaceuticals, Inc. is headquartered in Cranbury, New Jersey.
NEWS

Rocket Pharmaceuticals to Participate in the 8th Annual Evercore Healthcare Conference
businesswire.com
2025-11-25 07:00:00CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that the Company will participate in the 8th Annual Evercore Healthcare Conference in Miami. Gaurav Shah, M.D., Chief Executive Officer, will take part in a fireside chat on Tuesday, December 2, at 7:55 a.m. ET and host investor meetings. A webcast of the p.

Rocket Pharmaceuticals Reports Third Quarter 2025 Financial Results and Highlights Recent Progress
businesswire.com
2025-11-06 16:00:00CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and recent operational results for the third quarter ending September 30, 2025. “During the third quarter, we maintained disciplined execution and sharpened our strategic focus on Rocket's AAV cardiovascular gene therapy portfolio,” said Gaurav Sha.

ROCKET ALERT: Bragar Eagel & Squire, P.C. is Investigating Rocket Pharmaceuticals, Inc. on Behalf of Long-Term Stockholders and Encourages Investors to Contact the Firm
globenewswire.com
2025-10-23 18:03:00Bragar Eagel & Squire, P.C. Litigation Partner Brandon Walker Encourages Investors Who Suffered Losses In Rocket (RCKT) To Contact Him Directly To Discuss Their Options If you are a long-term stockholder in Rocket securities between February 27, 2025 to May 26, 2025 and would like to discuss your legal rights, call Bragar Eagel & Squire partner Brandon Walker or Marion Passmore directly at (212) 355-4648.

RCKT Stock Up as FDA Accepts Resubmitted BLA for Gene Therapy
zacks.com
2025-10-15 12:35:21Rocket Pharmaceuticals surges as the FDA accepts its resubmitted BLA for Kresladi, a gene therapy targeting the rare LAD-I disorder.

FDA Acceptance Of Rocket Pharmaceuticals' Lead Gene Therapy Application Reestablishes Momentum For Stock
benzinga.com
2025-10-14 14:27:11On Tuesday, the U.S. Food and Drug Administration (FDA) accepted Rocket Pharmaceuticals, Inc.'s (NASDAQ:RCKT) resubmission of the Biologics License Application (BLA) for Kresladi (marnetegragene autotemcel; marne-cel), a lentiviral vector (LV)-based investigational gene therapy for severe Leukocyte Adhesion Deficiency-I (LAD-I).

Rocket Pharmaceuticals Announces FDA Acceptance of BLA Resubmission of KRESLADI™ for the Treatment of Severe Leukocyte Adhesion Deficiency-I (LAD-I)
businesswire.com
2025-10-14 07:00:00CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today 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), a lentiviral vector (LV)-based investigational gene therapy for severe Leukocyte.

Rocket Pharmaceuticals Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
businesswire.com
2025-10-10 16:00:00CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that on October 6, 2025, the Compensation Committee of Rocket's Board of Directors approved the grant of inducement equity incentive awards to its newly-hired Chief Medical Officer, Dr. Syed Rizvi. The inducement awards consist of (i) a nonstatutory stock o.

Rocket Pharmaceuticals withdraws US application of gene therapy for rare blood disorder
reuters.com
2025-10-03 07:11:38Rocket Pharmaceuticals said on Friday that it has withdrawn its application for U.S approval of its experimental gene therapy for a rare inherited blood disorder.

Kuehn Law Encourages Investors of Rocket Pharmaceuticals, Inc. to Contact Law Firm
prnewswire.com
2025-09-19 13:30:00NEW YORK , Sept. 19, 2025 /PRNewswire/ -- Kuehn Law, PLLC, a shareholder litigation law firm, is investigating whether certain officers and directors of Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) breached their fiduciary duties to shareholders.

Rocket Pharmaceuticals, Inc. (RCKT) Presents At Morgan Stanley 23rd Annual Global Healthcare Conference Transcript
seekingalpha.com
2025-09-09 12:36:40Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT ) Morgan Stanley 23rd Annual Global Healthcare Conference September 9, 2025 9:15 AM EDT Company Participants Gaurav Shah - CEO & Director Conference Call Participants Michael Ulz - Morgan Stanley, Research Division Presentation Michael Ulz Equity Analyst All right. Good morning, everyone, and thanks for joining us at the Morgan Stanley Global Healthcare Conference.

RCKT Stock Rises as FDA Lifts Clinical Hold on Danon Disease Study
zacks.com
2025-08-21 12:15:28Rocket Pharmaceuticals' shares soar as the FDA lifts its clinical hold on the pivotal Danon disease gene therapy study.

Gene Therapy Trial Can Restart After Patient Death, FDA Says. The Sector Remains Depressed.
barrons.com
2025-08-20 11:02:00The past year has been a disappointing one for gene therapies.

FDA Lifts Rocket Pharmaceuticals Study Hold For Rare Disease Gene Therapy
benzinga.com
2025-08-20 09:31:04Rocket Pharmaceuticals Inc. RCKT stock is trading higher on Wednesday after the U.S. Food and Drug Administration (FDA) lifted the clinical hold on its pivotal Phase 2 trial of RP-A501 for Danon disease.

US FDA lifts clinical hold on Rocket Pharmaceuticals' gene therapy trial
reuters.com
2025-08-20 07:09:11Rocket Pharmaceuticals said on Wednesday that the U.S. Food and Drug Administration has lifted the clinical hold on its gene therapy mid-stage trial.

Rocket Pharmaceuticals Announces FDA Has Lifted the Clinical Hold on the Pivotal Phase 2 Trial of RP-A501 for the Treatment of Danon Disease
businesswire.com
2025-08-20 07:00:00CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on the Company's pivotal Phase 2 trial of RP-A501 for the treatment of Danon disease. The hold was lifted in under three months, underscoring the efficiency of the FDA's review pr.
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Rocket Pharmaceuticals to Participate in the 8th Annual Evercore Healthcare Conference
businesswire.com
2025-11-25 07:00:00CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that the Company will participate in the 8th Annual Evercore Healthcare Conference in Miami. Gaurav Shah, M.D., Chief Executive Officer, will take part in a fireside chat on Tuesday, December 2, at 7:55 a.m. ET and host investor meetings. A webcast of the p.

Rocket Pharmaceuticals Reports Third Quarter 2025 Financial Results and Highlights Recent Progress
businesswire.com
2025-11-06 16:00:00CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and recent operational results for the third quarter ending September 30, 2025. “During the third quarter, we maintained disciplined execution and sharpened our strategic focus on Rocket's AAV cardiovascular gene therapy portfolio,” said Gaurav Sha.

ROCKET ALERT: Bragar Eagel & Squire, P.C. is Investigating Rocket Pharmaceuticals, Inc. on Behalf of Long-Term Stockholders and Encourages Investors to Contact the Firm
globenewswire.com
2025-10-23 18:03:00Bragar Eagel & Squire, P.C. Litigation Partner Brandon Walker Encourages Investors Who Suffered Losses In Rocket (RCKT) To Contact Him Directly To Discuss Their Options If you are a long-term stockholder in Rocket securities between February 27, 2025 to May 26, 2025 and would like to discuss your legal rights, call Bragar Eagel & Squire partner Brandon Walker or Marion Passmore directly at (212) 355-4648.

RCKT Stock Up as FDA Accepts Resubmitted BLA for Gene Therapy
zacks.com
2025-10-15 12:35:21Rocket Pharmaceuticals surges as the FDA accepts its resubmitted BLA for Kresladi, a gene therapy targeting the rare LAD-I disorder.

FDA Acceptance Of Rocket Pharmaceuticals' Lead Gene Therapy Application Reestablishes Momentum For Stock
benzinga.com
2025-10-14 14:27:11On Tuesday, the U.S. Food and Drug Administration (FDA) accepted Rocket Pharmaceuticals, Inc.'s (NASDAQ:RCKT) resubmission of the Biologics License Application (BLA) for Kresladi (marnetegragene autotemcel; marne-cel), a lentiviral vector (LV)-based investigational gene therapy for severe Leukocyte Adhesion Deficiency-I (LAD-I).

Rocket Pharmaceuticals Announces FDA Acceptance of BLA Resubmission of KRESLADI™ for the Treatment of Severe Leukocyte Adhesion Deficiency-I (LAD-I)
businesswire.com
2025-10-14 07:00:00CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today 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), a lentiviral vector (LV)-based investigational gene therapy for severe Leukocyte.

Rocket Pharmaceuticals Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
businesswire.com
2025-10-10 16:00:00CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that on October 6, 2025, the Compensation Committee of Rocket's Board of Directors approved the grant of inducement equity incentive awards to its newly-hired Chief Medical Officer, Dr. Syed Rizvi. The inducement awards consist of (i) a nonstatutory stock o.

Rocket Pharmaceuticals withdraws US application of gene therapy for rare blood disorder
reuters.com
2025-10-03 07:11:38Rocket Pharmaceuticals said on Friday that it has withdrawn its application for U.S approval of its experimental gene therapy for a rare inherited blood disorder.

Kuehn Law Encourages Investors of Rocket Pharmaceuticals, Inc. to Contact Law Firm
prnewswire.com
2025-09-19 13:30:00NEW YORK , Sept. 19, 2025 /PRNewswire/ -- Kuehn Law, PLLC, a shareholder litigation law firm, is investigating whether certain officers and directors of Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) breached their fiduciary duties to shareholders.

Rocket Pharmaceuticals, Inc. (RCKT) Presents At Morgan Stanley 23rd Annual Global Healthcare Conference Transcript
seekingalpha.com
2025-09-09 12:36:40Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT ) Morgan Stanley 23rd Annual Global Healthcare Conference September 9, 2025 9:15 AM EDT Company Participants Gaurav Shah - CEO & Director Conference Call Participants Michael Ulz - Morgan Stanley, Research Division Presentation Michael Ulz Equity Analyst All right. Good morning, everyone, and thanks for joining us at the Morgan Stanley Global Healthcare Conference.

RCKT Stock Rises as FDA Lifts Clinical Hold on Danon Disease Study
zacks.com
2025-08-21 12:15:28Rocket Pharmaceuticals' shares soar as the FDA lifts its clinical hold on the pivotal Danon disease gene therapy study.

Gene Therapy Trial Can Restart After Patient Death, FDA Says. The Sector Remains Depressed.
barrons.com
2025-08-20 11:02:00The past year has been a disappointing one for gene therapies.

FDA Lifts Rocket Pharmaceuticals Study Hold For Rare Disease Gene Therapy
benzinga.com
2025-08-20 09:31:04Rocket Pharmaceuticals Inc. RCKT stock is trading higher on Wednesday after the U.S. Food and Drug Administration (FDA) lifted the clinical hold on its pivotal Phase 2 trial of RP-A501 for Danon disease.

US FDA lifts clinical hold on Rocket Pharmaceuticals' gene therapy trial
reuters.com
2025-08-20 07:09:11Rocket Pharmaceuticals said on Wednesday that the U.S. Food and Drug Administration has lifted the clinical hold on its gene therapy mid-stage trial.

Rocket Pharmaceuticals Announces FDA Has Lifted the Clinical Hold on the Pivotal Phase 2 Trial of RP-A501 for the Treatment of Danon Disease
businesswire.com
2025-08-20 07:00:00CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on the Company's pivotal Phase 2 trial of RP-A501 for the treatment of Danon disease. The hold was lifted in under three months, underscoring the efficiency of the FDA's review pr.










