Rocket Pharmaceuticals develops gene therapies for severe and rare inherited diseases, focusing on patients with limited treatment options. They use a method called AAV (Adeno-Associated Virus) to deliver corrected genes directly into patients' cells, targeting diseases that affect the heart, liver, eyes, or central nervous system. Unlike competitors, Rocket Pharmaceuticals emphasizes a patient-centric approach and combines scientific research with partnerships to generate revenue. Their goal is to create both the first and best gene therapies available, guided by their core values of Trust, Generosity, Curiosity, and Elevation.

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Victory Capital Management

California Institute for Regenerative Medicine

Biotechnology

Healthcare

Rocket Pharmaceuticals

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 Fanconi Anemia, commercial preparations for the potential approval of KRESLADI for severe LAD-I, and ongoing enrollment in the Phase 2, pivotal study of RP-A501 for Danon Disease and the Phase 1 study of RP-A601 for PKP2-ACM,” said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharmaceuticals. “As we build on the advancements made this quarter, we remain focused on execution for patients with rare and devastating diseases with limited treatment options.”. Recent Pipeline and Operational UpdatesAdvanced Fanconi Anemia (FA) program through key regulatory milestones

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

Aaron Ondrey joins Rocket as Chief Financial Officer, bringing more than 20 years of experience in financial management, strategic planning, commercial finance, capital allocation and mergers and acquisitions having held multiple finance leadership positions across the pharma and biotech industry.

Rocket Pharmaceuticals (RCKT) Appoints Aaron Ondrey as CFO

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 studies of KRESLADI™ (marnetegragene autotemcel) for severe Leukocyte Adhesion Deficiency-I (LAD-I) and RP-L102 for Fanconi Anemia (FA), in addition to the Phase 1 study of RP-L301 for Pyruvate Kinase Deficiency (PKD).“The positive updates presented at this year’s annual meeting demonstrate the sustained safety and efficacy across the totality of our LV hematology portfolio,” said Jonathan Schwartz, M.D., Chief Medical & Gene Therapy Officer, Rocket Pharmaceuticals. “Ahead of the upcoming PDUFA date, KRESLADITM continues to demonstrate 100% HSCT-free survival and significant reductions in infection-related hospitalizations following engraftment in patients with severe LAD-I. In our pivotal studies of RP-L102 for Fanconi Anemia, we continue to see maintained genetic and phenotypic correction combined with hematologic stabilization. Additionally, our Phase 1 study of RP-L301 for PKD shows sustained clinically meaningful hemoglobin improvement in all patients

Rocket Pharmaceuticals Presents Positive Data From Lv Hematology Portfolio At The 27Th Annual Meeting Of The American Society Of Gene And Cell Therapy (Asgct)

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 mutations in the FANCA gene affecting DNA repair and characterized by bone marrow failure (BMF), cancer predisposition, and congenital malformations. “The acceptance of the MAA for RP-L102 marks an important step forward in our goal of bringing this potential gene therapy treatment to patients impacted by this devastating childhood disorder. Currently, there are no existing options to potentially prevent BMF for patients with FA,” said Kinnari Patel, Pharm.D., MBA, President, Head of RD and Chief Operating Officer, Rocket Pharma. “We are appreciative of the patients, their families, and researchers who helped reach this meaningful milestone and continue participating in the clinical development program. We look forward to partnering closely with the EMA throughout the review process to make RP-L102 available to patients with FA who are in need of new treatment options.”

Rocket Pharmaceuticals Announces European Medicines Agency Acceptance Of Rp-L102 Marketing Authorization Application For The Treatment Of Fanconi Anemia

New Jersey’s biotech sector is recognized as a major hub of innovation and industry in the pharmaceutical and biotechnological landscapes. According to Genetic Engineering and Biotechnology News’ 2023 ranking, New Jersey is the 4th most important biotech hub in the U.S. with innovative biotech companies in its ecosystem.With $2.49 billion in venture capital investment in 2022 and the first half of 2023 and over 140,000 biotech jobs in the region, New Jersey is home to industry leaders such as Johnson  Johnson or Bristol Myers Squibb. But industry giants aren’t the only ones to call New Jersey home. Here are nine biotech companies to put on your radar.BioAegis TherapeuticsBioAegis Therapeutics, based in North Brunswick, New Jersey, is a clinical-stage biotech company that focuses on developing therapies based on plasma gelsolin, a naturally occurring human protein known for its role in modulating inflammation and immune response. The company aims to harness plasma gelsolin to treat a variety of conditions where inflammation plays a critical role.BioAegis’ flagship project involves the development of recombinant human plasma gelsolin (rhu-pGSN) for the treatment of acute and chronic inflammatory diseases, including acute respiratory distress syndrome (ARDS). ARDS is a severe lung condition often seen in critically ill patients, and it represents a significant area of unmet medical need

Biotech In New Jersey: Nine Companies Leading The Charge

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(TM) (marnetegragene autotemcel; marne-cel), a lentiviral (LV) vector-based gene therapy to treat severe leukocyte adhesion deficiency-I (LAD-I).

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

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

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

Rocket Pharmaceuticals to participate in the 43rd Annual J.P. Morgan Healthcare Conference.

Rocket Pharmaceuticals to Participate in the 43rd Annual J.P. Morgan Healthcare Conference

Rocket Pharmaceuticals prices upsized offering of 13.2M shares, strengthening its financial position with potential for additional shares through overallotment option.

Rocket Pharmaceuticals Secures $165M Through Upsized Public Stock Offering at $12.50 Per Share

Rocket Pharmaceuticals announces proposed public offering of common stock.

Rocket Pharmaceuticals Announces Proposed Public Offering of Common Stock

Develops gene therapies for rare diseases

Rocket Pharmaceuticals

Develops gene therapies for rare diseases

Rocket Pharmaceuticals

Develops gene therapies for rare diseases

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