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.

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.

Rocket Pharmaceuticals (RCKT) Surges on Encouraging Study Data

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 pricing of an upsized underwritten public offering of 4,642,858 shares of its common stock at a public offering price of $56.00 per share. The gross proceeds to Rocket from the offering are expected to be approximately $260 million, before deducting the underwriting disc

Rocket Pharmaceuticals Prices Upsized Public Offering of Common Stock

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NEW YORK--(BUSINESS WIRE)---- $RCKT #Danon--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 preliminary data from its open-label, Phase 1 clinical trial of RP-A501, the Company's adeno-associated viral vector (AAV)-based gene therapy candidate expressing LAMP2B for the treatment of Danon Disease. Danon Disease is a rare X-linked inherited disorder caused by genetic

Rocket Pharmaceuticals Announces Positive Gene Expression, Clinical Biomarker and Preliminary Functional Data from Phase 1 Trial of RP-A501 for the Treatment of Danon Disease

NEW YORK--(BUSINESS WIRE)---- $RCKT #ASH2020--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today presents updated interim data from its Fanconi Anemia (FA) and Leukocyte Adhesion Deficiency-I (LAD-I) programs at the 62nd American Society of Hematology (ASH) Annual Meeting. The data are highlighted in two oral presentations. “We are highly pleased with the data presented at ASH de

Rocket Pharmaceuticals Presents Positive Clinical Data from its Fanconi Anemia and Leukocyte Adhesion Deficiency-I Programs at the 62nd American Society of Hematology Annual Meeting

NEW YORK--(BUSINESS WIRE)---- $RCKT #ASH2020--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 preliminary clinical data from its Phase 1 clinical trial of RP-L301 for the treatment of Pyruvate Kinase Deficiency (PKD), showing significant improvement in hemoglobin levels and transfusion independence over the initial 3-months following therapy. These results were prese

Rocket Pharmaceuticals Announces Positive Preliminary Clinical Data from Phase 1 Trial of RP-L301 for the Treatment of Pyruvate Kinase Deficiency at the 62nd American Society of Hematology Annual Meeting

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 it will host a webcast on Monday, December 7 at 6:00PM EST to discuss presentations at the 62nd American Society of Hematology (ASH) Annual Meeting being held virtually December 5-8, 2020. The ASH presentations will highlight data from the Fanconi Anemia, Leukocyte Adhe

Rocket Pharmaceuticals to Hold Webcast for Investors Highlighting Data Presented at the 62nd American Society of Hematology Annual Meeting

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 the California Institute for Regenerative Medicine (CIRM) has awarded Rocket a $3.7 million CLIN2 grant award to support the clinical development of its lentiviral vector (LVV)-based gene therapy, RP-L401, for the treatment of Infantile Malignant Osteopetrosis (IMO), a

Rocket Pharmaceuticals Receives Funding from the California Institute for Regenerative Medicine for Phase 1 Clinical Trial of RP-L401 for Infantile Malignant Osteopetrosis

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 presentations at the upcoming 62nd American Society of Hematology (ASH) Annual Meeting being held virtually December 5-8, 2020. There will be two oral presentations highlighting clinical data from the Fanconi Anemia (FA) and Leukocyte Adhesion Deficiency-I (LAD-I) programs a

Rocket Pharmaceuticals to Present Data from its Fanconi Anemia, Leukocyte Adhesion Deficiency-I and Pyruvate Kinase Deficiency Programs at the 62nd American Society of Hematology Annual Meeting

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 reports financial results for the quarter that ended September 30, 2020, along with an update on the Company's key pipeline developments, business operations and upcoming milestones. “We are incredibly pleased with the steady progress we made across all five of our clinical programs t

Rocket Pharmaceuticals Reports Third Quarter 2020 Financial Results and Highlights Recent Progress

NEW YORK--(BUSINESS WIRE)---- $RCKT #ESID--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 clinical data at the European Society for Immunodeficiencies (ESID) 2020 Meeting being held virtually October 14-17, 2020. An oral presentation provides positive longer-term follow-up data from the Phase 1/2 clinical trial of RP-L201 for Leukocyte Adhesion Deficiency-I (LAD-

Rocket Pharmaceuticals Presents Positive LAD-l Clinical Update and Comprehensive IMO Preclinical Review at the European Society for Immunodeficiencies 2020 Meeting

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 two presentations at the European Society for Immunodeficiencies (ESID) 2020 Meeting to be held virtually October 14-17, 2020. An oral presentation will provide an update on data from the Phase 1/2 clinical trial of RP-L201 for Leukocyte Adhesion Deficiency-I (LAD-I). An e-p

Rocket Pharmaceuticals Announces Two Presentations at the European Society for Immunodeficiencies 2020 Meeting

NEW YORK--(BUSINESS WIRE)---- $RCKT #Danon--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare pediatric disorders, today announces it has treated the first patient in the higher dose cohort in its open-label, Phase 1 clinical trial of RP-A501, the Company’s adeno-associated viral vector (AAV)-based gene therapy for the treatment of Danon Disease.

Rocket Pharmaceuticals Announces First Patient Treated in Higher Dose Cohort in Phase 1 Clinical Trial of RP-A501 for Danon Disease

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 the U.S.

Rocket Pharmaceuticals Receives FDA Fast Track Designation for RP-L401 Gene Therapy for Infantile Malignant Osteopetrosis

NEW YORK--(BUSINESS WIRE)---- $RCKT--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 addition of four additional clinical trial sites across four clinical programs for Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Danon Disease and Infantile Malignant Osteopetrosis (IMO).

Rocket Pharmaceuticals Expands Clinical Sites for its FA, Danon, LAD-I and IMO Trials, Adding to its Global Centers of Excellence

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 reports financial results for the quarter that ended June 30, 2020, along with an update on the Company's key pipeline developments, business operations and upcoming milestones.

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