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.

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.

Why Shares of Rocket Pharmaceuticals Are Taking Off on Wednesday

Rocket Pharmaceuticals reaches alignment with the Food and Drug Administration regarding its global Phase 2 trial for a treatment of Danon disease.

Rocket Pharmaceuticals Stock Jumps Most Since 2020. New Gene Therapy Is Potential 'Value Driver.

Rocket Pharmaceuticals Inc (NASDAQ:RCKT) stock is soaring today, after the biotech name reached an "alignment" with the U.S. Food & Drug Administration (FDA) regarding a mid-stage trial design for its Danon disease therapy, RP-A501, for which there are currently no FDA-approved treatments.

Rocket Pharmaceuticals Stock Blasts Off on FDA Buzz

Rocket Pharmaceuticals Inc (RCKT) shares surged 32% in pre-market trading on Wednesday after the biotechnology company developing gene therapy announced that it has reached an agreement with the US Food and Drug Administration (FDA) over the global Phase 2 trial of RP-A501 to address Danon disease, a fatal inherited cardiomyopathy.  Rocket said its study would evaluate the use of the investigational gene therapy RP-A501 to treat 12 patients with Danon disease.

Rocket Pharmaceuticals stock takes flight on FDA agreement

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of investigational genetic therapies for rare childhood disorders with high unmet need, announced today the pricing of an underwritten public offering of (i) 7,812,500 shares of its common stock at a public offering price of $16.00 per share and (ii) to certain investors, pre-funded warrants to purchase 3,126,955 shares of comm.

Rocket Pharmaceuticals Announces Pricing of Public Offering of Common Stock

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 alignment has been reached with the Food and Drug Administration (FDA) on the global Phase 2 pivotal trial of RP-A501 for Danon Disease. Danon Disease is a uniformly fatal inherited cardiomyopathy that leads to mortality in the majority of male p.

Rocket Pharmaceuticals Reaches FDA Alignment on Pivotal Phase 2 Trial Design for RP-A501 in Danon Disease

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 June 30, 2023, and updates from the Company's key pipeline developments, business operations and upcoming milestones. “The second quarter of 2023 marked a period of strong forward momentum across our cardiovascu.

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

Rocket Pharmaceuticals is developing gene therapies for rare diseases, with positive results and safety profiles from clinical trials. The company's financial performance includes $360 million in cash and investments, with increased expenses and a net loss for the quarter. RCKT stock shows mixed signals, but the company has a stable capital structure and increased institutional fund holdings.

Rocket Pharmaceuticals: Thriving Gene Therapy With Multiple Catalysts

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The FDA grants fast track tag and orphan drug designation to Rocket's (RCKT) investigational gene therapy candidate, RP-A601, for cardiac indication. The stock gains 2.3% following the news.

Rocket (RCKT) Heart Disease Drug Gets Fast Track & Orphan Drug Tag

Shares of Rocket Pharmaceuticals RCKT, +0.17% gained 2.3% premarket on Thursday after the late-stage biotech company said the U.S. Food and Drug Administration granted fast-track and orphan-drug designations to its gene therapy candidate for treatment of an inherited heart disease. Rocket's RP-A601 gene therapy is designed to treat plakophilin-2 related arrhythmogenic cardiomyopathy, which is caused by genetic mutations and is associated with cardiac structural abnormalities and sudden cardiac death.

Rocket Pharmaceuticals shares rise on FDA fast-track designation for heart disease treatment

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 granted Fast Track and Orphan Drug designations to RP-A601, the Company's gene therapy candidate for the treatment of plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM). RP-A601 is Rocke.

Rocket Pharmaceuticals Receives FDA Fast Track and Orphan Drug Designations for RP-A601 Gene Therapy for PKP2 Arrhythmogenic Cardiomyopathy (ACM)

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 European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to RP-A501, the Company's investigational adeno-associated virus (AAV)-based gene therapy for the treatment of Danon Disease, a devastating and fatal inherited.

Rocket Pharmaceuticals Receives European Medicines Agency (EMA) Priority Medicines (PRIME) Designation for RP-A501 Gene Therapy for Danon Disease

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 granted Regenerative Medicine Advanced Therapy (RMAT) designation to RP-L301, the Company's investigational lentiviral-based gene therapy for Pyruvate Kinase Deficiency (PKD), a rare blood disorder.

Rocket Pharmaceuticals Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for RP-L301 Gene Therapy for Pyruvate Kinase Deficiency (PKD)

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 positive data from the Pyruvate Kinase Deficiency (PKD), Fanconi Anemia, Severe Leukocyte Adhesion Deficiency-I (LAD-I) and Danon Disease clinical programs and PKP2-arrhythmogenic cardiomyopathy (PKP2-ACM) preclinical program presented at the 26th Ann.

Rocket Pharmaceuticals Presents Positive Data from LV Hematology and AAV Cardiovascular Gene Therapy Programs at the 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT)

Today, we peek back in on Rocket Pharmaceuticals, Inc. for the first time in 2023. The company is seeing some momentum from positive trial developments and new-found enthusiasm from the analyst community so far here in May.

Rocket Pharmaceuticals: A Catalyst-Rich Year Ahead

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 it has received clearance from the U.S. Food and Drug Administration (FDA) for the Company's Investigational New Drug (IND) application for RP-A601, an AAV.rh74-based gene therapy candidate for the treatment of arrhythmogenic cardiomyopathy due t.

Rocket Pharmaceuticals Announces FDA Clearance of IND for Clinical Trial of RP-A601 for PKP2 Arrhythmogenic Cardiomyopathy (ACM)

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 March 31, 2023, and updates from the Company's key pipeline developments, business operations and upcoming milestones. “I am very pleased with our first quarter progress, as we continued to advance our industry.

Rocket Pharmaceuticals Reports First 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 that Gaurav Shah, M.D., Chief Executive Officer, is scheduled to participate in a fireside chat at the Bank of America Securities 2023 Healthcare Conference in Las Vegas, Nevada on Wednesday, May 10, 2023 at 4:20 p.m. PT. A live audio webcast of the p.

Rocket Pharmaceuticals to Participate in the Bank of America Securities 2023 Healthcare Conference

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 expansion of its leadership team to support its growing and industry-leading pipeline of AAV and LV gene therapy assets and evolution towards commercial stage. Jonathan Schwartz, M.D.

Rocket Pharmaceuticals Announces Leadership Expansion to Support Company on Evolution Towards Commercial Stage

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 the Genetic Medicines Corporate Panel at the 43rd Annual Cowen Health Care Conference taking place in Boston, MA on Monday, March 6, 2023 at 10:30 a.m. ET.

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