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.

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that Gaurav Shah, M.D., Chief Executive Officer, will deliver a company presentation virtually at the 40th Annual J.P. Morgan Healthcare Conference on Monday, Jan. 10 at 1:30 p.m. ET. A live audio webcast of the presentation will be available under “Events” in the Investors secti

Rocket Pharmaceuticals to Present at the 40th Annual J.P. Morgan Healthcare Conference

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Biotech stocks remain poised to eke out modest gains for 2021 after the stellar gains of the previous year. COVID-19 drug and vaccine developers experienced volatility throughout 2021 amid delays in programs and the reemergence of variant strains.

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CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces positive clinical updates from its ongoing Phase 2 registrational trials for Fanconi Anemia (FA) and Leukocyte Adhesion Deficiency-I (LAD-I) and its ongoing Phase 1 trial for Pyruvate Kinase Deficiency (PKD) at the 63rd American Society of Hematology (ASH) Annual Meeting. “The po

Rocket Pharmaceuticals Presents Positive Clinical Data from Company's Lentiviral Gene Therapies for Treatment of Fanconi Anemia, LAD-I and PKD at the 63rd American Society of Hematology (ASH) Annual Meeting

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces the appointment of Martin L. Wilson as General Counsel, Chief Compliance Officer and Senior Vice President. Mr. Wilson brings nearly 20 years of legal, compliance and executive experience and accomplishment within the life sciences industry. Mr. Wilson will be responsible for the

Rocket Pharmaceuticals Appoints Martin L. Wilson as General Counsel and Chief Compliance Officer

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, will host an in-person Investor and Analyst Event in Atlanta that will simultaneously be webcast on Tuesday, Dec. 14, 2021, at 7:30 a.m. ET to discuss updates from three of the Company's lentiviral vector (LVV)-based gene therapy programs: RP-L201 for Leukocyte Adhesion Deficiency-I (LAD-I), RP-

Rocket Pharmaceuticals to Host In-Person Investor and Analyst Event and Webcast on Tuesday, Dec. 14 at 7:30 a.m. ET to Discuss Updated Clinical Data Presented at the 63rd American Society of Hematology (ASH) Annual Meeting

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces Gaurav Shah, M.D., Chief Executive Officer, will participate in a fireside chat at the Evercore ISI 4th Annual HEALTHCONx Virtual Conference on Wednesday, Dec. 1, 2021 at 10:55 a.m. ET. A live audio webcast of the presentation will be available under “Events” in the Investors sec

Rocket Pharmaceuticals Announces Participation in Evercore ISI 4th Annual HEALTHCONx Virtual Conference

Rocket Pharmaceuticals Inc (NASDAQ: RCKT) has announced updated data from the Phase 1 trial evaluating a single intravenous infusion of RP-A501, an investigational gene therapy, for Danon Disease.  Danon disease is characterized by weakening of the heart muscle, weakening of skeletal muscles, and intellectual disability.

Rocket Pharma To Test Lower Dose Of Gene Therapy In Genetic Disorder Trial

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces updated data from the Phase 1 clinical trial evaluating a single intravenous infusion of RP-A501, the Company's investigational gene therapy, for the treatment of Danon Disease. This update, which includes interim safety and efficacy data from patients in the low-dose (6.7e13 vg/

Rocket Pharmaceuticals Announces Positive Updates from Phase 1 Clinical Trial of RP-A501 in Danon Disease

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, will host a conference call and live webcast on Monday, Nov. 15, 2021, at 8:30 a.m. ET. Rocket will present updated data from the ongoing Phase 1 clinical trial of RP-A501 in Danon Disease, which will include data being presented in an ePoster at the American Heart Association (AHA) Scientific S

Rocket Pharmaceuticals to Host Webcast on Nov. 15 at 8:30 a.m. ET to Present Updated Danon Disease Clinical Data

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces Gaurav Shah, M.D., Chief Executive Officer, will participate in a fireside chat at the Stifel 2021 Virtual Healthcare Conference on Tuesday, November 16, 2021 at 2:40 p.m. ET. A live audio webcast of the presentation will be available under “Events” in the Investors section of th

Rocket Pharmaceuticals Announces Participation in Stifel 2021 Virtual Healthcare Conference

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that updated data from the ongoing Phase 1 clinical trial of RP-A501 in Danon Disease will be presented at the American Heart Association (AHA) Scientific Sessions 2021 taking place virtually November 13-15, 2021. Details for Rocket's ePoster presentation are as follows: Title: R

Rocket Pharmaceuticals Announces Danon Disease Presentation at the American Heart Association (AHA) Scientific Sessions 2021

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces upcoming data presentations at the 63rd American Society of Hematology (ASH) Annual Meeting taking place virtually and in Atlanta, Georgia from December 11-14, 2021. Presentations will include updated clinical data from three of Rocket's lentiviral vector (LVV)-based gene therapy

Rocket Pharmaceuticals Announces Presentations Highlighting Three Lentiviral Gene Therapies at the 63rd American Society of Hematology (ASH) Annual Meeting

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today reports financial results for the quarter ending September 30, 2021 and updates on the Company's key pipeline developments, business operations, and upcoming milestones. “We are excited about the strong progress we made in the third quarter as we initiated treatment in our trial of RP-A501

Rocket Pharmaceuticals Reports Third Quarter 2021 Financial Results and Highlights Recent Progress

Rocket Pharmaceuticals Inc (NASDAQ: RCKT) has announced interim data updates from seven patients from the RP-L201 Phase 1/2 trial for Leukocyte Adhesion Deficiency-I (LAD-I). Data were shared at the Annual Congress of the European Society of Gene & Cell Therapy (ESGCT).

Rocket Pharma Posts Interim Data From Immunodeficiency Disorder Gene Therapy Trial

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces interim data updates from the RP-L201 Phase 1/2 clinical trial for the treatment of Leukocyte Adhesion Deficiency-I (LAD-I) at the 28th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT). Severe LAD-I is a rare pediatric disease that prevents patients from

Rocket Pharmaceuticals Announces Positive Clinical Data from RP-L201 Trial for the Treatment of LAD-I at the 28th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT)

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces data presentations at the 28th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT) taking place virtually October 19-22, 2021. Oral presentations will include clinical data from Rocket's lentiviral vector (LVV)-based gene therapy programs for Leukocyte Adhe

Rocket Pharmaceuticals Announces Presentations Highlighting Lentiviral Gene Therapies at the 28th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT)

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that Gaurav Shah, M.D., chief executive officer, will deliver an in-person company presentation at the 2021 Cell & Gene Meeting on the Mesa, being held as a hybrid conference virtually and in Carlsbad, CA. The Rocket Pharmaceuticals presentation will take place on Tuesday, Oc

Rocket Pharmaceuticals to Present at the 2021 Cell & Gene Meeting on the Mesa

Rocket Pharmaceuticals, Inc. is a clinical-stage, multi-platform biotechnology company developing gene therapies with a focus on direct on-target mechanisms of action for rare and devastating orphan diseases.

Rocket Pharmaceuticals Stock Forecast: Can It Rebound To $50 Levels? A Resounding Yes

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announced that Gaurav Shah, M.D., chief executive officer, will participate in a fireside chat on Tuesday, October 5, 2021, at 10:30 am ET at the Chardan Virtual 5th Annual Genetic Medicines Conference. In addition, Jonathan Schwartz, M.D., chief medical officer, will be part of a panel di

Rocket Pharmaceuticals to Participate in the Chardan Virtual 5th Annual Genetic Medicines Conference

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces an oral presentation of previously disclosed data from the ongoing Phase 1 trial of RP-A501 for the treatment of Danon Disease was given at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2021. The presentation overviewed data from the low-dose (6.7e13 vg/kg

Rocket Pharmaceuticals Presents Clinical Data from RP-A501 Trial in Danon Disease at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2021

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that previously disclosed data from the ongoing Phase 1 trial of RP-A501 in Danon Disease will be presented in a late-breaking oral presentation at the upcoming Heart Failure Society of America (HFSA) Annual Scientific Meeting 2021. The meeting will take place September 10-13, 20

Rocket Pharmaceuticals Announces Upcoming Danon Disease Presentation at the Heart Failure Society of America Annual Scientific Meeting 2021

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces the appointment of Isabel Carmona, J.D., as chief human resources officer and senior vice president. Ms. Carmona brings more than 25 years of experience in human resources and organizational leadership including the last 15 years within the life sciences industry. Ms. Carmona wil

Rocket Pharmaceuticals Appoints Isabel Carmona, J.D., as Chief Human Resources Officer

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces a securities purchase agreement with a fund affiliated with RTW Investments, LP, the Company's largest shareholder, for the purchase of an aggregate of 812,516 shares of common stock, par value $0.01 per share, at a purchase price of $32.48 per share, the closing price on August

Rocket Pharmaceuticals Announces $26.4 Million Private Placement

The FDA has lifted the clinical hold on Rocket Pharmaceuticals Inc's (NASDAQ:RCKT) Phase 1 trial of RP-A501 (gene therapy) for Danon Disease, allowing patient enrollment to resume.  The hold was removed after the Company addressed the FDA's requests to modify the trial protocol and other supporting documents with revised patient selection and management guidelines.

FDA Lifts Clinical Hold On Rocket Pharma's X-Linked Inherited Disorder Trial

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on the Company's Phase 1 clinical trial of RP-A501 for the treatment of Danon Disease, allowing patient enrollment to resume. The hold was removed after the Company addressed the FDA's requests to modif

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