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 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

Rocket Pharmaceuticals Announces FDA Lifts Clinical Hold on Danon Disease Trial of RP-A501

Rocket Pharmaceuticals, Inc. (RCKT) CEO Gaurav Shah on Q2 2021 Results - Earnings Call Transcript

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 June 30, 2021 and updates on the Company's key pipeline developments, business operations, and upcoming milestones. “We are grateful to the FDA for its support and for working with us on our Danon program toward resuming our trial, which we

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

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 audio webcast on Monday, Aug. 9, 2021, at 4:30 p.m. ET to report second quarter 2021 financial results and recent business highlights. Investors may access the conference call by dialing (866) 939-3921 from locations in the United States or +1 (678) 302-3550

Rocket Pharmaceuticals to Host Conference Call on Aug. 9 at 4:30 p.m. ET to Discuss Second Quarter Results and Recent Business Highlights

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Rocket Pharmaceuticals Inc (NASDAQ: RCKT) has announced positive data from its Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), and Pyruvate Kinase Deficiency (PKD) gene therapy programs. Data were presented at the American Society of Gene and Cell Therapy Annual Meeting.

Rocket Pharmaceuticals Announces Positive Data From RP-L102, RP-L201 Gene Therapy Programs

CRANBURY, N.J.--(BUSINESS WIRE)-- #ASGCT--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 data from its Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), and Pyruvate Kinase Deficiency (PKD) gene therapy programs presented at the 24th American Society of Gene and Cell Therapy (ASGCT) Annual Meeting. “We are very excited to report positive

Rocket Pharmaceuticals Presents Positive Clinical Data from Fanconi Anemia, Leukocyte Adhesion Deficiency-I, and Pyruvate Kinase Deficiency Programs at 24th Annual Meeting of the American Society of Gene and Cell Therapy

CRANBURY, N.J.--(BUSINESS WIRE)---- $RCKT #GeneTherapy--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 March 31, 2021 and updates on the Company's key pipeline developments, business operations, and upcoming milestones. “I am pleased that we began 2021 with sustained momentum across clinical, regulatory and manufacturing activities as reflect

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

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 clinical data presentations at the upcoming 24thAmerican Society of Gene and Cell Therapy (ASGCT) Annual Meeting taking place May 11-14, 2021. Investigators will review new data from Rocket's Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), and Fanconi A

Rocket Pharmaceuticals Announces Upcoming Clinical Data Presentations at the 24th Annual Meeting of the American Society of Gene and Cell Therapy

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 interim data from the Company's Phase 1/2 clinical trial studying RP-L201, its lentiviral-based gene therapy for the treatment of severe Leukocyte Adhesion Deficiency-I (LAD-I). Severe LAD-I is a rare pediatric disease that prevents patients from adequately combating inf

Rocket Pharmaceuticals Reports Positive Long-Term Clinical Data from RP-L201 Trial for the Treatment of Leukocyte Adhesion Deficiency-I at the Clinical Immunology Society 2021 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 that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to RP-L201, the Company's investigational gene therapy for Leukocyte Adhesion Deficiency-I (LAD-I). PRIME designation was granted based on encouraging preliminary safety and efficacy data

Rocket Pharmaceuticals Receives EMA Priority Medicines (PRIME) Designation for RP-L201 Gene Therapy for Treatment of Leukocyte Adhesion Deficiency-I

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 preliminary clinical data from its Phase 1 trial of RP-L301 for the treatment of Pyruvate Kinase Deficiency (PKD), showing durable normalization of hemoglobin levels up to 6 months following therapy and similar 3-month trends in the second patient treated. Additionally, r

Rocket Pharmaceuticals Announces Updated Positive Preliminary Clinical Data from Phase 1 Trial of RP-L301 for the Treatment of Pyruvate Kinase Deficiency

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