Rocket Pharma

Rocket Pharmaceuticals, Inc. is a clinical-stage biotechnology company, which engages in the development of gene therapy treatment options for rare and devastating pediatric diseases. Its multi-platform development approach applies the lentiviral vector (LVV) and adeno-associated viral vector (AAV) gene therapy platforms. The firm's clinical program is a LVV-based gene therapy for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer. The company was founded on July 7, 1999 and is headquartered in Cranbury, NJ.

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Rocket Pharmaceuticals, Inc. is a clinical-stage biotechnology company, which engages in the development of gene therapy treatment options for rare and devastating pediatric diseases. Its multi-platform development approach applies the lentiviral vector and adeno-associated viral vector gene therapy platforms. Its clinical program is an LVV-based gene therapy for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer. The company was founded on July 7, 1999 and is headquartered in Cranbury, NJ.

On Monday, Rocket Pharmaceuticals, Inc. RCKT presented long-term safety and efficacy results from the Phase 1 study of RP-A501 in male patients with Danon disease.

Rocket Pharmaceuticals' Gene Therapy Progress Boosts Confidence in Future Developments: Analyst

CRANBURY, 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, presented long-term safety and efficacy results from the Phase 1 RP-A501 study which showed that RP-A501 was generally well tolerated and all evaluable Danon disease patients demonstrated LAMP2 protein expression at 12 months (sustained up to 60 months) and reduction of le.

Rocket Pharmaceuticals Announces New England Journal of Medicine Publication of Phase 1 RP-A501 Long-Term Data and Presents at Late-Breaking Scientific Sessions at 2024 American Heart Association Conference

CRANBURY, 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 be presenting new data on the results of the RP-A501 Phase 1 trial to treat patients with Danon disease at the American Heart Association's 2024 Late-Breaking Science sessions being held from November 16-18, 2024 in Chicago, Illinois.

Rocket Pharmaceuticals to Present Updated Data from the Phase 1 Clinical Trial of RP-A501 for Danon Disease at the American Heart Association's 2024 Late-Breaking Science Sessions

RCKT incurs narrower-than-expected third-quarter 2024 loss. The company provides updates on its pipeline candidates.

Rocket Pharmaceuticals' Q3 Earnings Beat, Pipeline in Focus

CRANBURY, 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 UBS Global Healthcare Conference in Rancho Palos Verdes, CA. Gaurav Shah, M.D., Chief Executive Officer, will take part in a fireside chat on Tuesday, November 12, at 5:00 p.m. PT. A webcast of the fireside chat will.

Rocket Pharmaceuticals to Participate in the UBS Global Healthcare Conference

CRANBURY, 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, 2024. “Rocket made meaningful progress during the third quarter, notably with the completion of enrollment in the RP-A501 program for Danon disease, low dose cohort enrollmen.

Rocket Pharmaceuticals Reports Third Quarter 2024 Financial Results and Highlights Recent Progress

Enrollment completed for pivotal single-arm phase 2 study, using RP-A501 for the treatment of male patients with Danon disease. Promise for RP-A501 program lies with the potential to receive FDA Accelerated Approval for the treatment of male patients with Danon disease. The Danon disease treatment market is expected to reach $2.82 billion by 2030.

Rocket: Danon Disease Treatment Program Should Carry The Tide Until Regulatory Moves

Rocket completes enrollment of patients in a pivotal phase II study evaluating RP-A501 for treating male patients with Danon disease.

RCKT Completes Enrollment in Rare Heart Disease Study, Stock Rises

CRANBURY, 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 all patients have been enrolled in the global, pivotal Phase 2 clinical trial evaluating RP-A501 to treat male patients with Danon disease. After the two-patient safety run-in, followed by harmonized global site activations, the remaining 10 patients w.

Rocket Pharmaceuticals Announces Completion of Enrollment in Phase 2 Pivotal Trial of RP-A501 for the Treatment of Danon Disease

CRANBURY, 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 the appointment of Mikael Dolsten, M.D., Ph.D., as an independent director to its Board of Directors. Dr. Dolsten is an accomplished industry executive with extensive global pharmaceutical management experience. “As an industry-leading pharmaceutical execut.

Rocket Pharmaceuticals Announces Appointment of Mikael Dolsten, M.D., Ph.D., to Board of Directors

The recent regulatory setbacks related to RCKT's pipeline candidate, Kresladi (marnetegragene autotemcel) weigh heavily on the stock.

Rocket Stock Down More Than 30% in Past Six Months: Here's Why

CRANBURY, 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 Morgan Stanley 22nd Annual Global Healthcare Conference and the 2024 Cantor Fitzgerald Global Healthcare Conference in New York. Gaurav Shah, M.D., Chief Executive Officer, will take part in a fireside chat and manag.

Rocket Pharmaceuticals to Participate in Upcoming Investor Conferences

CRANBURY, 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 quarter ending June 30, 2024. “Over the quarter, Rocket has been advancing its clinical pipeline as we progressed our RP-A501 and RP-A601 cardiac programs, targeting Danon disease and PKP2-ACM, and continued t.

Rocket Pharmaceuticals Reports Second Quarter 2024 Financial Results and Highlights Recent Progress

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Per the FDA, Rocket (RCKT) needs to submit additional manufacturing information on Kresladi gene therapy to secure approval for severe leukocyte adhesion deficiency-I (LAD-I), a rare genetic disorder.

FDA Rejects Rocket's (RCKT) Gene Therapy BLA for Rare Disease

Rocket Pharmaceuticals (RCKT) shares declined in intraday trading Friday after the biopharmaceutical firm announced that the Food and Drug Administration (FDA) wants more data about its gene therapy to treat a rare immune disorder in children before granting approval.

FDA Again Delays Approval of Rocket Pharmaceuticals' Gene Therapy Treatment

CRANBURY, 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 needs, today announced a regulatory update for KRESLADI™ (marnetegragene autotemcel; marne-cel), a lentiviral (LV) vector-based gene therapy to treat severe leukocyte adhesion deficiency-I (LAD-I). The U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter.

Rocket Pharmaceuticals Provides Regulatory Update on KRESLADI™ (marnetegragene autotemcel; marne-cel)

Rocket Pharmaceuticals (RCKT) reported earnings 30 days ago. What's next for the stock?

Why Is Rocket Pharmaceuticals (RCKT) Down 4.8% Since Last Earnings Report?

CRANBURY, 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 European Commission (EC), based on a positive opinion issued by the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA), has granted orphan medicinal product designation for RP-A601, the Company's adeno-associated.

Rocket Pharmaceuticals Receives Orphan Medicinal Product Designation from the European Commission for RP-A601 for PKP2 Arrhythmogenic Cardiomyopathy

CRANBURY, 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 longer-term data updates from its lentiviral (LV) vector hematology portfolio presented at the 27th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT). Data updates demonstrate the continued safety and efficacy of the Phase 1/2 pivotal.

Rocket Pharmaceuticals Presents Positive Data from LV Hematology Portfolio at the 27th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT)

Rocket Pharmaceuticals' (RCKT) first-quarter earnings beat estimates. Devoid of marketed drugs, the company did not report any revenues during the quarter.

Rocket's (RCKT) Q1 Loss Narrower Than Expected, Sales Nil

CRANBURY, 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 quarter ending March 31, 2024. “Rocket has had a strong start to 2024. This quarter has been marked by meaningful progress across all our clinical programs, most notably with the EMA's review of RP-L102 for Fa.

Rocket Pharmaceuticals Reports First Quarter 2024 Financial Results and Highlights Recent Progress

Rocket Pharmaceuticals (RCKT) might move higher on growing optimism about its earnings prospects, which is reflected by its upgrade to a Zacks Rank #2 (Buy).

Rocket Pharmaceuticals (RCKT) Upgraded to Buy: Here's Why

Rocket Pharmaceuticals, Inc. has potential FDA approvals for its gene therapies on the horizon. The company focuses on developing gene therapies for rare diseases, targeting a market that is expected to triple in value by the end of the decade. Rocket's pipeline includes promising therapies for severe leukocyte adhesion deficiency-I, Danon Disease, and Fanconi Anemia, among others.

Rocket Pharmaceuticals: Cautiously Optimistic (Rating Upgrade)

The EMA accepts Rocket's (RCKT) regulatory submission seeking approval for RP-L102, a gene therapy for a rare genetic disorder called fanconi anemia that affects the bone marrow.

News for Rocket Pharma Stock (RCKT)