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.

EMA accepted Rocket Pharmaceuticals, Inc. regulatory application of RP-L102 for the treatment of patients with Falconi Anemia. A U.S. FDA BLA filing of RP-L102 for the treatment of patients with Falconi Anemia is expected in the 1st half of 2024. PDUFA date of June 30th of 2024 to determine if Kresladi should be approved for the treatment of patients with leukocyte adhesion deficiency-I.

Rocket Pharmaceuticals: Multiple Regulatory Submissions Make It A Must Watch

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 Medicines Agency (EMA) accepted the Marketing Authorization Application (MAA) for RP-L102, its lentiviral (LV) vector-based investigational gene therapy for Fanconi Anemia (FA), complementation group A, a rare genetic disorder caused by mu.

Rocket Pharmaceuticals Announces European Medicines Agency Acceptance of RP-L102 Marketing Authorization Application for the Treatment of Fanconi Anemia

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

Why Is Rocket Pharmaceuticals (RCKT) Down 13.6% 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 the appointment of Aaron Ondrey, as Chief Financial Officer, and the further evolution of its corporate leadership team through key appointments with existing team members. These leadership announcements build on and expand Rocket's preparedness for its pla.

Rocket Pharmaceuticals Announces Appointment of Aaron Ondrey as Chief Financial Officer and Additional Updates to Corporate Leadership Team

Rocket Pharmaceuticals, Inc. stock surged 76% post a "Buy" recommendation, fueled by positive Phase 2 trial news for RP-A501 in Danon disease. RP-A501, targeting a rare disease without current treatments, promises significant market potential, pending trial outcomes and approval. Financial analysis shows Rocket with a strong liquidity position and a 25-month cash runway, despite a notable monthly cash burn.

Rocket Pharmaceuticals' Promising Path In Gene Therapy

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

Rocket (RCKT) Registers Narrower-Than-Expected Loss in Q4

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 operational results for the fourth quarter and year ended December 31, 2023. “I am pleased with the strong results Rocket delivered in 2023, as we closed another successful year of progress across all six disclosed gene therapy programs spannin.

Rocket Pharmaceuticals Reports Fourth Quarter and Full Year 2023 Financial Results and Highlights Recent Progress

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 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). The FDA extended the review period.

Rocket Pharmaceuticals Announces Update on FDA Review Timeline of KRESLADI™ (marnetegragene autotemcel) for the Treatment of Severe Leukocyte Adhesion Deficiency-I (LAD-I)

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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 Gaurav Shah, M.D., Chief Executive Officer, is scheduled to present at the 42nd Annual J.P. Morgan Healthcare Conference on Monday, January 8, 2024, at 3:45 p.m. PT. A webcast of the presentation will be available under “Events” in the Investors sectio.

Rocket Pharmaceuticals to Present at 42nd Annual J.P. Morgan Healthcare Conference

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CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced the appointment of R. Keith Woods as an independent, non-executive director to its Board of Directors. Mr. Woods is a seasoned executive with more than 30 years of experience spanning commercialization, global operations, business strategy and supply.

Rocket Pharmaceuticals Appoints R. Keith Woods to Board of Directors

Rocket (RCKT) gains 40% in three months on positive regulatory updates on its key candidates.

Rocket (RCKT) Surges 40% in 3 Months on Regulatory Updates

The consensus price target hints at a 167.1% upside potential for Rocket Pharmaceuticals (RCKT). While empirical research shows that this sought-after metric is hardly effective, an upward trend in earnings estimate revisions could mean that the stock will witness an upside in the near term.

Wall Street Analysts See a 167.09% Upside in Rocket Pharmaceuticals (RCKT): Can the Stock Really Move This High?

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial results for the quarter ending September 30, 2023, and updates from the Company's key pipeline developments, business operations and upcoming milestones. “I'm very pleased with Rocket's results this quarter, as we realized significant regulat.

Rocket Pharmaceuticals Reports Third Quarter 2023 Financial Results and Highlights Recent Progress

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced presentations at the 30th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT) in Brussels, Belgium, taking place October 24-27. Updated data will be presented from the Phase 1 clinical trial of RP-L301 for Pyruvate Kinase Defici.

Rocket Pharmaceuticals Announces Presentations Highlighting Lentiviral and AAV Gene Therapies at the 30th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT)

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and 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 Biologics License Application (BLA) and granted Priority Review for RP-L201 (marnetegragene autotemcel), a lentiviral vector (LV)-based investigational gene therapy for severe Leukocyte.

Rocket Pharmaceuticals Announces FDA Acceptance of Biologics License Application with Priority Review for RP-L201 (marnetegragene autotemcel) for the Treatment of Severe Leukocyte Adhesion Deficiency-I (LAD-I)

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that Gaurav Shah, M.D., Chief Executive Officer, is scheduled to participate in a fireside chat at the Chardan 7th Annual Genetic Medicines Conference in New York City on Tuesday, October 3, at 1:00 p.m. ET. A webcast of the fireside chat will be avai.

Rocket Pharmaceuticals to Participate in the Chardan 7th Annual Genetic Medicines Conference

Intellia Therapeutics

Rocket Pharmaceuticals has two programs approaching commercialization. By contrast, Intellia's candidates won't be ready for quite some time.

Better Gene Editing Stock: Intellia Therapeutics vs. Rocket Pharmaceuticals

Rocket Pharmaceuticals achieves a significant milestone in their RP-A501 therapy for Danon Disease, strengthening their position in the gene therapy industry. RP-A501 aims to restore normal cardiac function and offer a lifetime of benefit to patients with Danon Disease. Rocket and the FDA are in alignment on the pivotal study design for RP-A501, positioning RCKT as a pioneer in genetic disorders related to the heart.

Rocket Pharmaceuticals: Update On RP-A501 Strengthens Bullish Outlook

Rocket Pharmaceuticals is advancing one of its rare disease therapies into phase 2. It has another medicine that could get approved for sale relatively soon.

Rocket Pharmaceuticals Stock Is Blasting Off, but Here's Why You Should Tread Carefully

Rocket Pharmaceucticals (RCKT) soars 53.8% in a week on news of alignment with the FDA for the mid-stage study of RP-A501, an investigational gene therapy for treating Danon Disease.

Here's Why Rocket Pharmaceuticals (RCKT) Rises 53.8% in a Week

Rocket Pharmaceuticals announced yesterday it had reached agreement with the FDA on a potentially pivotal study of in vivo gene therapy candidate RP-A501. The drug candidate has faced a long journey to get to this point - now a historic opportunity awaits. Data from the study - which could be used to support accelerated approval - will not arrive for at least 15 months.

Rocket Pharmaceuticals: Soaring On Pivotal Danon Disease Study Agreement - More Upside Ahead?

Shares of Rocket Pharmaceuticals (NASDAQ: RCKT ) popped sharply higher on Wednesday, making it one of the top-trending tickers. Yesterday, the biotechnology specialist — which focuses on rare childhood disorders with high unmet needs — announced an encouraging clinical trial development.

Why Is Rocket Pharmaceuticals (RCKT) Stock Up 35% Today?

The company also announced a public stock offering that it said was worth $175 million. The clinical-stage biotech focuses on genetic therapies for rare diseases.

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