RP-A501
Gene Therapy Target: LAMP2B, LAMP2
Rocket Pharmaceuticals
Most recent annual filing: 2026-02-15
- RP-A501 Danon disease Ph2 readout2H 2026RP-A501 Danon disease Ph2 readout, 2H 2026, material
- RP-A501 Danon disease Ph2 start2H 2026RP-A501 Danon disease Ph2 start, 2H 2026, notable
- RP-A701 BAG3-DCM Ph1 start2H 2026RP-A701 BAG3-DCM Ph1 start, 2H 2026, routine
- RP-A601 PKP2-ACM pivotal readout2H 2026RP-A601 PKP2-ACM pivotal readout, 2H 2026, routine
Bar width shows how precise the guidance is — a pin is a stated date, a wide band a six-month window. Deeper blue means more of the thesis rides on the event; it is not a view on the stock.
Pipeline (4 active programs)
RP-A601
Gene Therapy Target: PKP2
RP-A701
Gene Therapy Target: BAG3
KRESLADI
marnetegragene autotemcel · RP-L201 Gene Therapy Target: ITGB2, CD18
Discontinued (2)
- RP-L102 — Fanconi Anemia · no longer allocating internal resources towards regulatory filings and commercial activities; no longer pursuing BLA and EMA submissions; exploring external partnership options
- RP-L301 — Pyruvate Kinase Deficiency · no longer allocating internal resources towards RP-L301 and does not plan to initiate enrollment in a Phase 2 study; exploring external partnership options
Biotech Distilled coverage (6)
- 2026-08-11 UPDATE: Rocket Pharmaceuticals (RCKT) - The Observation Window Closed Clean 🔒 Paid
- 2026-08-03 UPDATE: Rocket Pharmaceuticals (RCKT) - The Canary Came Back Out of the Mine 🔒 Paid
- 2026-05-09 UPDATE: Rocket Pharmaceuticals (RCKT) - The Danon Clock is Running 🔒 Paid
- 2026-04-29 UPDATE: Rocket Pharmaceuticals (RCKT) - The PRV Lands at $180M, Runway Bought Through Q2 2028 🔒 Paid
- 2026-03-28 UPDATE: Rocket Pharmaceuticals (RCKT) - Rocket Deploys KRESLADI Patch, But Internal Code Shows Severe Tech Debt 🔒 Paid
- 2026-03-06 Rocket Pharmaceuticals (RCKT): Scientific Deep Dive for Lead and Pipeline Products 🔒 Paid
Articles open on Substack. Some deep dives are subscriber-only.