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Rocket Pharmaceuticals

RCKT Rare disease Lead: Approved 4 Biotech Distilled articles ↓

Cardiovascular Immunology Gene Therapy

Most recent annual filing: 2026-02-15

Pipeline (4 active programs)

RP-A501

Gene Therapy Target: LAMP2B, LAMP2

Danon Disease Phase 2 intravenous

RP-A601

Gene Therapy Target: PKP2

Arrhythmogenic Cardiomyopathy Phase 1 intravenous

RP-A701

Gene Therapy Target: BAG3

Dilated Cardiomyopathy Phase 1

KRESLADI

marnetegragene autotemcel · RP-L201

Gene Therapy Target: ITGB2, CD18

Leukocyte Adhesion Deficiency-I Approved CIEMAT

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 (4)

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.