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.

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.

Rocket Pharmaceuticals to Participate in the 43rd Annual Cowen Health Care 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 reported financial and operational results for the fourth quarter and year ended December 31, 2022.

Rocket Pharmaceuticals Reports Fourth Quarter and Full Year 2022 Financial and Operational Results

Rocket Pharmaceuticals stock trades at ~$19.5 today - back in December 2021 it had hit highs of >$60. The highs were achieved when Rocket's in vivo gene therapy showed promise treating patients with a rare genetic heart condition known as Danon Disease.

Rocket Pharmaceuticals: Promising Gene Therapy Play With Discounted Entry Point

Rocket Pharmaceuticals, Inc. performed beautifully for me in the last half year. They have a number of catalysts in the next few months.

Rocket Pharmaceuticals Has A Lot More Upside

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 virtual fireside chat at the SVB Securities Global Biopharma Conference on Thursday, February 16, at 2:20 p.m. ET.

Rocket Pharmaceuticals to Present at the SVB Securities Global Biopharma Conference

Rocket Pharmaceuticals Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for RP-A501 Gene Therapy for Danon Disease

The mean of analysts' price targets for Rocket Pharmaceuticals (RCKT) points to a 163.4% upside in the stock. While this highly sought-after metric has not proven reasonably effective, strong agreement among analysts in raising earnings estimates does indicate an upside in the stock.

Wall Street Analysts Think Rocket Pharmaceuticals (RCKT) Could Surge 163.35%: Read This Before Placing a Bet

Rocket Pharmaceuticals (RCKT) witnesses a hammer chart pattern, indicating support found by the stock after losing some value lately. This coupled with an upward trend in earnings estimate revisions could mean a trend reversal for the stock in the near term.

Rocket Pharmaceuticals (RCKT) Could Find a Support Soon, Here's Why You Should Buy the Stock Now

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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 childhood disorders with high unmet need, today announces the addition of RP-A601 to Rocket's cardiac gene therapy portfolio as well as anticipated highlights for the year ahead across the Company's world-class pipeline of lentiviral and AAV gene therapy programs targeting rare hematologic and cardiovascular diseases. These announcements and anticipated highlights will be presented at the 41st Annual J.P. Morgan Healthcare Conference today at 2:15 p.m. PT by Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharma.

Rocket Pharmaceuticals Expands Cardiac Gene Therapy Portfolio with Addition of RP-A601 for PKP2-ACM and Announces Positive Updated Phase 1 Data for RP-A501 in Danon Disease

The consensus price target hints at a 170.9% 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 Believe Rocket Pharmaceuticals (RCKT) Could Rally 170.92%: Here's is How to Trade

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 childhood disorders with high unmet need, today announces that Gaurav Shah, M.D., Chief Executive Officer, is scheduled to present at the 41st Annual J.P. Morgan Healthcare Conference on Monday, January 9, 2023, at 2:15 p.m. PT.

Rocket Pharmaceuticals to Present at 41st Annual J.P. Morgan 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 childhood disorders with high unmet need, today announces updates from the Company's end-of-Phase 1 meeting with the U.S. Food and Drug Administration (FDA) regarding RP-A501, the Company's investigational adeno-associated virus (AAV)-based gene therapy for Danon Disease. During the meeting, Rocket reviewed the positive Phase 1 dataset with the FDA and proposed a study design and endpoints for ongoing clinical development of the investigational gene therapy.

Rocket Pharmaceuticals Provides Update on Anticipated Registration Path for RP-A501 in Danon Disease Following End-of-Phase 1 FDA Meeting

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