COMPANY
Rocket Pharmaceuticals
FDA-approved therapeutic company Clinical Phase 2
Hiring
| Title | Location (from posting) | Employer posted | First seen |
|---|---|---|---|
| Facilities Technician I | Location not stated | Not stated | Oct 16, 2026 |
| Senior Manager, Quality Control Operations | Location not stated | Not stated | Oct 16, 2026 |
| Senior Accountant | Location not stated | Not stated | Oct 16, 2026 |
| Vice President, Access, Value Evidence & Pricing | Location not stated | Not stated | Oct 16, 2026 |
| Director, Business Insights & Analytics | Location not stated | Not stated | Oct 16, 2026 |
Timeline
- Debt Debt financing: $150M (up to, as announced), announced Oct 6, 2026. Source: SEC filing. source ↗
- Trial start —: BAG3-DCM Natural History Study source ↗
- Trial start 1: A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy source ↗
- Trial start Phase 2: Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency source ↗
- FDA FDA New drug/biologic (novel): kresladi (marnetegragene autotemcel) — BLA125806, Mar 26, 2026. Applicant at approval: Rocket Pharmaceuticals, Inc. source ↗
- Trial start —: PKP2-ACM Natural History Study source ↗
- Trial start —: Danon Disease Natural History Study source ↗
- Trial start Phase 2: A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease source ↗
- Trial start 1: A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM) source ↗
- Trial start —: Natural History of Danon Disease source ↗
- Trial start —: Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I) source ↗
- Trial start I: Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of RP-L102 source ↗
- Trial start Phase 1: A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis source ↗
- Trial start Phase 2: Gene Therapy for Fanconi Anemia, Complementation Group A source ↗
- Trial start Phase 1: Gene Therapy for Pyruvate Kinase Deficiency (PKD) source ↗
- Trial start —: Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene Therapy source ↗
- Trial start Phase 2: Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A source ↗
- Trial start Phase 1/Phase 2: A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I source ↗
- Trial start Phase 1: Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B source ↗
- Trial start Phase 1: A Gene Therapy Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I source ↗
- Trial start Phase 1: A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A source ↗
Upcoming milestones
- 2028-06 (ESTIMATED) · Phase 2 · A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease · NCT06092034
- 2028-09 (ESTIMATED) · 1 · A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM) · NCT05885412
- 2029-06 (ESTIMATED) · 1 · A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy · NCT07137338
- 2029-12 (ESTIMATED) · · Danon Disease Natural History Study · NCT06214507
- 2030-12 (ESTIMATED) · · PKP2-ACM Natural History Study · NCT06644742
- 2032-12 (ESTIMATED) · · BAG3-DCM Natural History Study · NCT07486752
- Jan 17, 2034 (ESTIMATED) · · Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene Therapy · NCT04437771
- Oct 4, 2036 (ESTIMATED) · · Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I) · NCT06282432
Funding
- Debt financing: $150M (up to, as announced), announced Oct 6, 2026. Source: SEC filing. source ↗
Clinical trials (lead sponsor) 19
| Record | Title | Phase | Status | Start | Primary completion |
|---|---|---|---|---|---|
| NCT06092034 | A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease | Phase 2 | Recruiting | Sep 5, 2023 | 2028-06 (estimated) |
| NCT03882437 | Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B | Phase 1 | Completed | Apr 17, 2019 | Jul 3, 2025 (actual) |
| NCT07137338 | A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy | 1 | Recruiting | Apr 17, 2026 | 2029-06 (estimated) |
| NCT06214507 | Danon Disease Natural History Study | Not listed | Recruiting | Dec 20, 2023 | 2029-12 (estimated) |
| NCT06422351 | Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency | Phase 2 | Suspended | 2026-04 | 2029-01 (estimated) |
| NCT05885412 | A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM) | 1 | Recruiting | Aug 29, 2023 | 2028-09 (estimated) |
| NCT07527975 | Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of RP-L102 | I | Enrolling by invitation | Feb 1, 2022 | 2038-05 (estimated) |
| NCT04437771 | Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene Therapy | Not listed | Active, not recruiting | Jun 1, 2020 | Jan 17, 2034 (estimated) |
| NCT07486752 | BAG3-DCM Natural History Study | Not listed | Not yet recruiting | 2026-05 | 2032-12 (estimated) |
| NCT06644742 | PKP2-ACM Natural History Study | Not listed | Recruiting | 2026-03 | 2030-12 (estimated) |
| NCT04248439 | Gene Therapy for Fanconi Anemia, Complementation Group A | Phase 2 | Active, not recruiting | Jul 15, 2020 | May 5, 2026 (estimated) |
| NCT06282432 | Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I) | Not listed | Active, not recruiting | Mar 9, 2022 | Oct 4, 2036 (estimated) |
| NCT04105166 | Gene Therapy for Pyruvate Kinase Deficiency (PKD) | Phase 1 | Completed | Jul 6, 2020 | Jun 9, 2025 (actual) |
| NCT04069533 | Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A | Phase 2 | Unknown status (ClinicalTrials.gov) | Nov 28, 2019 | 2025-02 (estimated) |
| NCT05548855 | Natural History of Danon Disease | Not listed | Completed | Sep 26, 2022 | Oct 31, 2023 (actual) |
| NCT03812263 | A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I | Phase 1/Phase 2 | Completed | Aug 30, 2019 | Sep 12, 2023 (actual) |
| NCT04525352 | A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis | Phase 1 | Terminated | Nov 19, 2020 | May 21, 2021 (actual) |
| NCT03825783 | A Gene Therapy Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I | Phase 1 | Withdrawn | Apr 15, 2019 | 2021-10 (estimated) |
| NCT03814408 | A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A | Phase 1 | Unknown status (ClinicalTrials.gov) | Jan 11, 2019 | 2020-12 (estimated) |
FDA records (last 2 years)
- FDA New drug/biologic (novel): kresladi (marnetegragene autotemcel) — BLA125806, Mar 26, 2026. Applicant at approval: Rocket Pharmaceuticals, Inc. source ↗
Open jobs 13
| Title | Location (from posting, not HQ) | Employer posted | First seen | Level |
|---|---|---|---|---|
| Facilities Technician I | Not listed | Not stated | Oct 16, 2026 | Entry |
| Senior Manager, Quality Control Operations | Not listed | Not stated | Oct 16, 2026 | Senior |
| Senior Accountant | Not listed | Not stated | Oct 16, 2026 | Senior |
| Vice President, Access, Value Evidence & Pricing | Not listed | Not stated | Oct 16, 2026 | Executive |
| Director, Business Insights & Analytics | Not listed | Not stated | Oct 16, 2026 | Senior |
| Manager, External Affairs | Not listed | Not stated | Oct 16, 2026 | Mid |
| Specialist, IT – GxP Technical Support | Not listed | Not stated | Oct 16, 2026 | Entry |
| Senior Engineer, IT - GxP Infrastructure & Systems | Not listed | Not stated | Oct 16, 2026 | Senior |
| Manager, IT - GxP Systems & Compliance | Not listed | Not stated | Oct 16, 2026 | Mid |
| Director, Internal & Employee Communications | Not listed | Not stated | Oct 16, 2026 | Senior |
| Senior Director, HEOR and Pricing | Not listed | Not stated | Oct 16, 2026 | Senior |
| Senior Director, Analytical Development | Not listed | Not stated | Oct 16, 2026 | Senior |
| Director, Gene Therapy Clinical Science | Not listed | Not stated | Oct 16, 2026 | Senior |
Connect
See your connections at Rocket Pharmaceuticals on LinkedIn (search by name, results not checked)