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.

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.

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

NEW YORK--(BUSINESS WIRE)--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 first patient has received investigational therapy in the open-label, Phase 1 clinical trial of RP-L301, the Company’s lentiviral vector (LVV)-based gene therapy for the treatment of Pyruvate Kinase Deficiency (PKD), a rare monogenic red blood cell disorder. “We are

Rocket Pharmaceuticals Announces First Patient Treated in Phase 1 Trial of RP-L301 Gene Therapy for Pyruvate Kinase Deficiency

NEW YORK--(BUSINESS WIRE)-- #IMO--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 has received clearance from the U.S. Food and Drug Administration (FDA) for the Company’s Investigational New Drug (IND) application for RP-L401. RP-L401 is the Company’s lentiviral vector (LVV)-based gene therapy for the treatment of Infantile Malignant Osteopetrosi

Rocket Pharmaceuticals Announces FDA Clearance of IND for RP-L401 Gene Therapy for Infantile Malignant Osteopetrosis

Rocket Pharmaceuticals: Updating Our Bullish View For Recent Events - Still A BUY

Rocket Pharmaceuticals has been struggling lately, but the selling pressure may be coming to an end soon.

Rocket Pharmaceuticals Sees Hammer Chart Pattern: Time to Buy?

Rocket (RCKT) has seen solid earnings estimate revision activity over the past month, and belongs to a strong industry as well.

Why Rocket (RCKT) Stock Might be a Great Pick

Does Rocket Pharmaceuticals (RCKT) have what it takes to be a top stock pick for momentum investors? Let's find out.

Here's Why Rocket Pharmaceuticals (RCKT) is a Great Momentum Stock to Buy

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The remaining weeks of December are chock-full of clinical trial results and FDA approval decisions that savvy investors can take advantage of.

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As of late, it has definitely been a great time to be an investor in Rocket Pharmaceuticals, Inc. (RCKT).

Rocket Pharmaceuticals, Inc. (RCKT) Shares March Higher, Can It Continue?

RCKT has solid pipeline assets and plenty of catalysts during next 12 months for value creation. There are no near-term funding needs.

Blast Off With Rocket Pharmaceuticals

Rocket Therapeutics has remained under my radar despite making headway in the gene therapy pipeline with multiple INDs being accepted. I review the companies technology and pipeline candidates.

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