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 dosing completion of pivotal Phase 2 Cohort 4 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 dosing completion of pivotal Phase 2 Cohort 4 for RP-A501 in Danon Disease by mid-2027. (H1 2027). 3 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 - Oct 6, 2026discontinued Rocket discontinued Phase 2 Cohort 3 of RP-A501 after two patients experienced serious adverse events with C3-inhibitor immunomodulatory regimen.
In Phase 2 Cohort 3, two patients treated with immunomodulatory regimen containing a C3-inhibitor experienced serious adverse events. Cohort 3 is discontinued and data from the two subjects were not included.
Filing - Sep 30, 2026n/a Rocket Pharmaceuticals enters $150 million senior secured term loan facility with Hercules Capital, receives $35 million initial tranche.
On September 30, 2026, Rocket Pharmaceuticals, Inc., together with its subsidiaries entered into a Loan and Security Agreement providing for up to five tranches of senior secured term loans in an aggregate principal amount of up to $150.0 million.
Filing
6 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.