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 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

With all due respect to Elton John, the real Rocket Man is in Cranbury, New Jersey.

Rocket Pharmaceuticals Primed for Takeoff

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 announces that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to RP-L201, its investigational gene therapy for the treatment of Leukocyte Adhesion Deficiency-I (LAD-I). RMAT designation was granted based on encouraging prel

Rocket Pharmaceuticals Receives FDA Regenerative Medicine Advanced Therapy Designation for RP-L201 Gene Therapy for Leukocyte Adhesion Deficiency-I

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 and operational results for the fourth quarter and year ended December 31, as well as guidance for anticipated 2021 milestones. “2021 is poised to be another exciting year for Rocket,” said Gaurav Shah, M.D., Chief Executive Officer of Rocket. “We anticipate clinical data

Rocket Pharmaceuticals Reports Fourth Quarter and Full Year 2020 Financial and Operational Results

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 announces participation at the following upcoming conferences: 10th Annual SVB Leerink Global Healthcare Conference Gaurav Shah, M.D., Chief Executive Officer of Rocket, is scheduled to participate in a fireside chat on Thursday, February 25, 2021, at 2:20 p.m. Eastern Time. Cowen's 41st Annual H

Rocket Pharmaceuticals Announces Participation at Upcoming Conferences

Rocket recently released impressive data for their gene therapy candidate for Danon Disease, RP-A501. The data demonstrated that RP-A501 was able to improve some of the hallmark metrics and symptoms. Rocket is gearing up for a potential commercial launch. The company is working on building out a new HQ with R&D and commercial capabilities.

Rocket Pharmaceuticals: Moving Closer To A Potential Launch

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 announces the Company's plans for the buildout of its new Research and Development (R&D) and Chemistry, Manufacturing and Controls (CMC) operation which will also serve as the Company's new headquarters in Cranbury, New Jersey. This new 103,720 ft2 facility will support clinical develo

Rocket Pharmaceuticals Announces Buildout of R&D and Manufacturing Facility to Support Development of Innovative Gene Therapy Pipeline

NEW YORK--(BUSINESS WIRE)---- $RCKT #GeneTherapy--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”), 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 and President of Rocket is scheduled to present on Monday, January 11, 2021, at 2:50 p.m. Eastern Time at the 39th Annual J.P. Morgan Healthcare Conference. A live audio webcast of the presentation will be available on the Inve

Rocket Pharmaceuticals Announces Participation at the 39th Annual J.P. Morgan Healthcare Conference

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NEW YORK--(BUSINESS WIRE)---- $RCKT #GeneTherapy--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces the closing of its previously announced upsized underwritten public offering of 5,339,286 shares of its common stock, including the exercise in full by the underwriters of their option to purchase an additional 696,428 shares, at the public offering price of $56.00 per share

Rocket Pharmaceuticals Announces Closing of Public Offering and Full Exercise of the Underwriters' Option to Purchase Additional Shares

Rocket Pharmaceuticals' (RCKT) AAV-based gene therapy candidate demonstrates early evidence of clinical benefit and tolerability in patients with Danon disease, which affects the cardiac muscle.

News for Rocket Pharma Stock (RCKT)