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Rocket Pharmaceuticals Inc.
Sponsor

Rocket Pharmaceuticals Inc.

United States 9 Cedarbrook Drive, 08512-3618, United States

About the sponsor

Research activity includes Danon disease, with a focus on heart muscle involvement and the correction of LAMP2 deficiency in cardiomyopathy.

  • Cardiac gene therapy
  • Myocardial tissue expression
  • Left ventricular mass

The sponsor’s interest in this area centers on inherited conditions affecting cardiac function and cardiac remodeling.

About Specializations

Rocket Pharmaceuticals Inc. runs clinical trials in conditions such as Congenital aplastic anaemia, Pyruvate kinase deficiency anaemia, Glycogen storage disease type II, Leukocyte adhesion deficiency, among others.

Recruiting now

Ongoing trials

Clinical trials run by this sponsor – those enrolling participants first.

Condition
Country
City
Type of trial
Recruitment status
Quick filters

6 clinical trials

Congenital aplastic anaemia

Recruiting

Study on the Safety and Effects of Infusing Modified CD34+ Cells for Patients with Fanconi Anemia Subtype A

InvestigationalNo placebo
Countries:SpainSpain
  • Participants:0–17 years
  • Substances:Autologous Cd34+Enriched Cells From Patients With Fanconi Anemia Subtype A Transduced Ex Vivo With Lentiviral Vector Carrying The Fanconi Anemia Complementation Group A Gene
  • Sponsor:Rocket Pharmaceuticals Inc.

Glycogen storage disease type II

Recruiting

Study on Gene Therapy with AAV9.LAMP2B for Male Patients with Danon Disease

Registered drugNo placebo
Countries:GermanyGermany
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Eculizumab
  • Sponsor:Rocket Pharmaceuticals Inc.

Congenital aplastic anaemia

Recruiting

Long-Term Study on the Safety and Effects of RP-L102 Infusion for Patients with Fanconi Anemia Subtype A

InvestigationalNo placebo
Countries:SpainSpain
  • Participants:0–17 years · 18–64 years
  • Substances:Autologous Cd34+Enriched Cells From Patients With Fanconi Anemia Subtype A Transduced Ex Vivo With Lentiviral Vector Carrying The Fanconi Anemia Complementation Group A Gene
  • Sponsor:Rocket Pharmaceuticals Inc.

Pyruvate kinase deficiency anaemia

Not recruiting

Study on Gene Therapy for Pyruvate Kinase Deficiency Using Autologous CD34+ Cells in Adults and Children

Registered drugNo placebo
Countries:SpainSpain
  • Participants:0–17 years · 18–64 years
  • Substances:Busulfan
  • Sponsor:Rocket Pharmaceuticals Inc.

Pyruvate kinase deficiency anaemia

Not recruiting

Study on Long-Term Safety of Gene Therapy for Pyruvate Kinase Deficiency Using Autologous CD34+ Cells in Adults and Children

InvestigationalNo placebo
Countries:SpainSpain
  • Participants:0–17 years · 18–64 years
  • Substances:Autologous Cd34+ Hematopoietic Stem Cells Transduced Ex Vivo With A Lentiviral Vector Encoding The Codon-Optimized Version Of Pklr Gene
  • Sponsor:Rocket Pharmaceuticals Inc.

Leukocyte adhesion deficiency+1

Not recruiting

Study on Long-Term Safety and Efficacy of Gene Therapy for Leukocyte Adhesion Deficiency-I Using RP-L201 in Patients with LAD-I

InvestigationalNo placebo
Countries:SpainSpain
  • Participants:0–17 years · 18–64 years
  • Substances:Autologous Cd34+ Haematopoietic Stem Cells Genetically Modified With Lentiviral Vector Encoding The Cd18 Gene
  • Sponsor:Rocket Pharmaceuticals Inc.

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