Rocket Pharmaceuticals

98 articles with Rocket Pharmaceuticals

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

  3/29/2021

  — PRIME Designation to Facilitate Greater EMA Collaboration for Development and Potential Accelerated Regulatory Review in Europe — — LAD-I Program Now Holds All Available Accelerated Regulatory Designations in the U.S. and EU —

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

  3/22/2021

  Updated Data Demonstrate Sustained Improvements in Hemoglobin to Normal Range at 6-months in First Patient and Similar 3-month Improvements in Second Patient Treated

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

  3/9/2021

  — FDA RMAT Designation for RP-L201 to Help Expedite Development and Registration — — Enrollment Complete in Phase 1/2 Clinical Trial of RP-L201 for the Treatment of LAD-I —

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

  2/25/2021

  — Achieved Favorable Clinical Results Across Four First-in-Class Gene Therapy Programs for Danon Disease, FA, LAD-I and PKD — — Announced >50% Protein Expression with Stabilization and Biomarker Improvement Including Cardiac Output in Low Dose Cohort for Danon Disease Heart Failure Program —

• Rocket Pharmaceuticals Announces Participation at Upcoming Conferences - Feb 18, 2021

  2/18/2021

  Rocket Pharmaceuticals, Inc., a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders announces participation at the following upcoming conferences

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

  1/11/2021

  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

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

  1/6/2021

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

• Rocket Pharmaceuticals Announced Closing of Public Offering and Full Exercise of the Underwriters’ Option to Purchase Additional Shares

  12/14/2020

  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

• Rocket Pharmaceuticals Prices Upsized Public Offering of Common Stock - Dec 10, 2020

  12/10/2020

  Rocket Pharmaceuticals, Inc., a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, 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.

• Rocket Pharmaceuticals Announces Proposed Public Offering of Common Stock - Dec 09, 2020

  12/9/2020

  Rocket Pharmaceuticals, Inc., a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, announces that it has commenced an underwritten public offering of $175 million of shares of its common stock.

• 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

  12/8/2020

  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

• 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

  12/7/2020

  Rocket Pharmaceuticals Presents Positive Clinical Data from its Fanconi Anemia and Leukocyte Adhesion Deficiency-I Programs at the 62 nd American Society of Hematology Annual Meeting –Two of Three FA “Process B” Patients with at Least 12-Months Follow-Up and Three of Four Additional Patients with Shorter Follow-Up Demonstrate Evidence of Engraftment— – Follow-Up Data from Phase 1/2 Trial for Leukocyte Adhesion Deficiency-I

• 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

  12/6/2020

  Preliminary Data from First Patient Demonstrates Initial Safety and Tolerability of RP-L301, Near Doubling of Hemoglobin to Normal Range and Additional Normalization of Hemolysis Markers

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

  12/2/2020

  Rocket Pharmaceuticals to Hold Webcast for Investors Highlighting Data Presented at the 62 nd American Society of Hematology Annual Meeting — Rocket Management to Discuss Data Updates from the Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I) and Pyruvate Kinase Deficiency (PKD) Programs —

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

  11/13/2020

  — ~$3.7 Million Clinical Grant to Fund U.S. Phase 1 Study of RP-L401 Gene Therapy for Infantile Malignant Osteopetrosis —

• 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

  11/4/2020

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

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

  11/4/2020

  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.

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

  10/16/2020

  Longer-Term Data from the Registration-Enabling Phase 1/2 Trial for Leukocyte Adhesion Deficiency-I Demonstrate Safety and Efficacy of RP-L201

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

  10/1/2020

  Poster Presentation to Highlight Preclinical Data on RP-L401 for Infantile Malignant Osteopetrosis

• Rocket Pharmaceuticals Announces Participation at Upcoming Conferences - Sep 24, 2020

  9/24/2020

  Rocket Pharmaceuticals, Inc., a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, announces participation at the following upcoming conferences