Rocket Pharmaceuticals Inc. — Sponsor Intelligence | 6 EU Trials

Filter these 6 trials by condition, phase, country and more.

Open in Search →

6 Registered trials

3 Recruiting

3 Completed trials

3 Countries active

14 Unique IMPs

100% Results reported

Phase II

Phase I/II

Phase I (Other)

Phase II: 1 Phase I/II: 3 Phase I (Other): 2

Oversight events:

2 temporary halts across 1 trial

View oversight register →

100%

3 of 3 completed trials have published summary results (EU CTR Article 37 — 12-month obligation).

View full Transparency Scoreboard →

Top indications

Pyruvate kinase deficiency anaemia 2 Leukocyte adhesion deficiency type I 1

Top IMPs

AUTOLOGOUS CD34+ HEMATOPOIETIC STEM CELLS TRANSDUCED EX VIVO WITH A LENTIVIRAL VECTOR ENCODING THE CODON-OPTIMIZED VERSION OF PKLR GENE 2 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 2 ADENO-ASSOCIATED VIRUS SEROTYPE 9 VECTOR CONTAINING THE HUMAN LAMP2 ISOFORM B TRANSGENE 1 AUTOLOGOUS CD34+ HAEMATOPOIETIC STEM CELLS GENETICALLY MODIFIED WITH LENTIVIRAL VECTOR ENCODING THE CD18 GENE 1 BUSULFAN 1 ECULIZUMAB 1 FILGRASTIM 1 PLERIXAFOR 1

All trials

6 total

Multi-Center, Open Label Gene Therapy Study of RP-A501 in Male Patients with Danon Disease Recruiting

2023-506480-34-00 Phase II 2 countries ADENO-ASSOCIATED VIRUS SEROTYPE 9 VECTOR CONTAINING THE HUMAN LAMP2 ISOFORM B TR…
Long-Term Follow-up: Phase I/II clinical study to evaluate the safety and efficacy of the infusion of RP-L102 Recruiting

2022-501082-52-00 Phase I/II 1 country AUTOLOGOUS CD34+ENRICHED CELLS FROM PATIENTS WITH FANCONI ANEMIA SUBTYPE A TRANS…
Long-Term Follow-up: Phase I/II clinical study to evaluate the safety and efficacy of the infusion of autologous CD34+ cells transduced with… Recruiting

2024-511523-33-00 Phase I/II 1 country AUTOLOGOUS CD34+ENRICHED CELLS FROM PATIENTS WITH FANCONI ANEMIA SUBTYPE A TRANS…
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… Ended

2022-501086-41-00 Phase I/II 1 country AUTOLOGOUS CD34+ HAEMATOPOIETIC STEM CELLS GENETICALLY MODIFIED WITH LENTIVIRAL …
Long-Term Follow-Up (LTFU) for Gene Therapy of Pyruvate Kinase Deficiency (PKD): A Phase I Clinical Trial to Evaluate the Safety of the Infu… Ended

2022-501526-38-00 Phase I (Other) 1 country AUTOLOGOUS CD34+ HEMATOPOIETIC STEM CELLS TRANSDUCED EX VIVO WITH A LENTIVIRAL V…
Gene Therapy for Pyruvate Kinase Deficiency (PKD): A Phase I Clinical Trial to Evaluate the Safety of the Infusion of Autologous CD34+ Cells… Ended

2024-511520-13-00 Phase I (Other) 1 country AUTOLOGOUS CD34+ HEMATOPOIETIC STEM CELLS TRANSDUCED EX VIVO WITH A LENTIVIRAL V…

Showing 1–6 of 6 trials

← All sponsors A–Z