Rocket Pharmaceuticals, Inc. RCKT
Rocket Pharmaceuticals, Inc. (Nasdaq: RCKT) is a rare genetic drug developer. Its lead program, RP-A501, is in Phase 2 for Danon disease. Its next catalyst on record is RP-A501: Company expects to complete dosing of the pivotal Phase 2 study for RP-A501 in Danon Disease by mid-2027. (H1 2027).
Upcoming catalysts
No catalyst is dated to the next 30 days or to an FDA action date.
Next further out: RP-A501 — Company expects to complete dosing of the pivotal Phase 2 study for RP-A501 in Danon Disease by mid-2027. (H1 2027). 6 catalysts on record beyond the next 30 days; subscribers see each with its filing sentence.
Pipeline
| Program | Indication | Modality | Stage |
|---|---|---|---|
| KRESLADI (RP-L201) | LAD-I | gene therapy | Approved |
| RP-A501 | Danon disease (pivotal Phase 2) | gene therapy | Phase 2 |
| RP-A601 | PKP2 cardiomyopathy | gene therapy | Phase 1 |
| RP-A701 | BAG3 cardiomyopathy | gene therapy | Phase 1 |
Recent results and decisions
- Oct 6, 2026positive First three Cohort 4 patients at the recalibrated dose with modified immunomodulation showed no TMA, capillary leak or other complement-mediated events; 1 patient with Grade 3+ serious TEAE (elevated GGT, resolved), all discharged within 1-2 weeks.
No evidence of thrombotic microangiopathy, capillary leak or other complement-mediated events
Filing - Sep 4, 2026positive Pivotal Cohort 4 with recalibrated dose and modified immunomodulatory regimen showed favorable safety profile with no evidence of thrombotic microangiopathy, capillary leak or other complement-mediated toxicities in the first three patients.
Pivotal Cohort 4 (recalibrated dose with modified IM regimen) No evidence of thrombotic microangiopathy, capillary leak or other complement-mediated events
Filing - Aug 10, 2026positive All three initial patients dosed at the recalibrated 3.8e13 GC/kg dose with modified immunomodulation completed the protocol-defined safety observation period with no TMA, capillary leak syndrome or other significant safety concerns.
All three patients have completed the protocol-defined safety observation period, with no thrombotic microangiopathy (TMA), capillary leak syndrome, or other significant safety concerns observed at the recalibrated dose with the refined immunomodulatory regimen.
Filing
4 more on record.
For subscribers
- Cash runway against each catalyst, with the company's own runway statement
- Track record: every Phase 3 readout, FDA decision and guidance slip since 2014
- Expected move for each dated catalyst, from comparable past events
- Phase 2 evidence behind each Phase 3 readout
- Companies on the same target or class
- Full catalyst list with filing sentences, deals, registered trials, Excel export
Other phase 2 rare genetic companies
The free Monday brief
Last week's readouts and FDA decisions and the dated catalysts in the week ahead, each with its filing sentence.