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RCKT Rocket Pharmaceuticals

AAV gene therapy (RP-A501 related franchise context)

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Thesis

Editorial summary — not a primary filing

Gene therapy platform across rare cardiac and hematologic indications; DMD program is earlier than peers.

Next gate

2026-12-01EstimatedPh2 readout

Phase 2 update

110 days · Estimated: Estimated window

Survive-to-Event

ok

Cash covers the next gate with ~9 months spare (curated estimate).

Unverified estimate — not from a live SEC parse

Cash runway (~387d)100%
Days to gate (110d)28%
Show math

$280M cash ÷ $22M/mo burn ≈ 387 days. Source: curated SEC quarter figures.

Cash $280M as of 2026-03-31 (SEC)

Pipeline spine

  1. Preclinical
  2. Ph1
  3. Ph2
  4. Ph3
  5. Filed
  6. Approved

Rivals in this race

Open race →
SRPTApprovedokCAPRFiledokSLDBPh1ok

Risks

  • Clinical execution
  • Capital intensity
  • Crowded DMD field
  • Regulatory path length

Source trail

  1. SECCash from latest reported quarter (curated)as of 2026-03-31
  2. ClinicalTrials.govNCT05775848as of 2026-07-01
  3. CompanyIR / press disclosuresas of 2026-07-01