Clinical Research

Rocket Pharmaceuticals Inc.

Address
9 Cedarbrook Drive

Sponsor Overview

assignment
6
sponsored trials
science
14
molecules
location_city
3
research centres
public
3
countries
person_search
5
investigators

Specializations

Cardiac and Lysosomal Storage Disorders

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.

Primary Immunodeficiency

Clinical research covers Leukocyte Adhesion Deficiency-I, a severe inherited disorder of immune cell trafficking and host defense.

  • Hematopoietic stem cell gene therapy
  • ITGB2
  • Immune cell function

Work in this domain addresses restoration of leukocyte adhesion and immune competence in affected patients.

Bone Marrow Failure Syndromes

Studies include Fanconi anemia subtype A, with emphasis on inherited DNA repair defects and related hematologic failure.

  • FANCA gene correction
  • Blood count normalization
  • Bone marrow stability

This area also involves monitoring for clonality and long-term blood cell recovery after gene-based treatment.

Inherited Anemias and Red Blood Cell Disorders

Research extends to pyruvate kinase deficiency, an inherited red blood cell disorder associated with chronic hemolytic anemia.

  • Pyruvate kinase gene therapy
  • Transfusion dependence
  • Anemia reduction

Clinical interest also includes broader pyruvate kinase deficiency terminology used across adult and pediatric hematology research.

Long-Term Gene Therapy Follow-Up

The sponsor funds extended follow-up across multiple inherited diseases to assess durable effects after autologous cell therapy and lentiviral vector treatment.

  • Transgene persistence
  • Replication-competent lentivirus
  • Long-term safety

This research area spans ongoing observation of hematologic stability, phenotypic correction, and treatment durability in rare genetic disease populations.

Sponsored Trials

Trial Name Country Status
Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I). Phase I/II clinical study to evaluate the safety and efficacy of the infusion of autologous hematopoietic stem cells transduced with a lentiviral vector encoding the ITGB2 geneSpain SpainNot Recruiting
Long-Term Follow-Up (LTFU) for Gene Therapy of Pyruvate Kinase Deficiency (PKD): A Phase I Clinical Trial to Evaluate the Safety of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the Codon Optimized Red Cell Pyruvate Kinase (coRPK) Gene in Adult and Pediatric Subjects with PKDSpain SpainNot Recruiting
Long-Term Follow-up of Autologous CD34+ Cells Transduced with Lentiviral Vector Carrying FANCA Gene in Patients with Fanconi Anemia Subtype ASpain SpainRecruiting
Long-Term Follow-up: Phase I/II clinical study to evaluate the safety and efficacy of the infusion of RP-L102Spain SpainRecruiting
Phase 2 Evaluation of rAAV9-LAMP2B Gene Therapy in Male Danon Disease Patients: Efficacy and Safety Assessment of Intravenous AdministrationGermany GermanyItaly ItalyRecruiting
Phase I Evaluation of Autologous CD34+ Cells Transduced with Lentiviral Vector Encoding Codon-Optimized PKLR Gene in Pyruvate Kinase DeficiencySpain SpainNot Recruiting

Data & Insights

science
Molecule Portfolio
Compounds under investigation
4
Exclusive Molecules
out of 14 total (29% not shared with other sponsors)

This sponsor works with 14 molecules, of which 4 are not being studied by other sponsors.

hub
Site Partnerships
Collaboration with research centres
33%
Return partnership rate
1 of 3 sites collaborated multiple times

This sponsor has worked with 3 sites across their trial portfolio.

Therapeutic Focus

  • 1.
    Congenital, Hereditary, and Neonatal Diseases and Abnormalities4 trials
    165 other sponsors
  • 2.
    Hemic and Lymphatic Diseases2 trials
    249 other sponsors

Partnered Research Centres

3 centres
location_city
assignment133
Azienda Ospedaliera Universitaria Meyer IRCCS
Address
Viale Gaetano Pieraccini 24, 50139 Florence
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location_city
assignment25
Deutsches Herzzentrum Muenchen Des Freistaates Bayern Klinik An Der Technischen Universitaet Muenchen
Address
Lazarettstrasse 36, Neuhausen-Nymphenburg, 80636 Munich
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location_city
assignment798
Hospital Universitario Fundacion Jimenez Diaz
Address
Avenida De Los Reyes Catolicos 2, 28040 Madrid
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