Rocket Pharmaceuticals Inc.
RCKT evidence brief
What we can confirm
- BioSniper Score:
- 4.8 / 10
- Market Cap:
- $373.8M
What it means
What to watch
Prepared research question
What matters most for RCKT—catalysts, financial risk and supporting evidence?
Traceable citations · Unknowns marked
BioSniper AI · RCKT
Rocket Pharmaceuticals Inc. (RCKT) is a fully integrated, commercial-stage biotechnology company focused on developing and commercializing genetic therapies for rare and life-threatening diseases, with a primary emphasis on inherited cardiovascular disorders and one FDA-approved product for an ultra-rare indication.
Key risk: No product revenue
BioSniper Score
Based on 2 of 6 signals
Limited data coverage
Market Cap
$373.8M
Cash Runway
—
Lead Asset
—
Next Catalyst
—
Financial data as of Jun 30, 2026.
Tracking 14 clinical trials for Rocket Pharmaceuticals Inc..
Rocket Pharmaceuticals Inc. (RCKT) is a fully integrated, commercial-stage biotechnology company focused on developing and commercializing genetic therapies for rare and life-threatening diseases, with a primary emphasis on inherited cardiovascular disorders and one FDA-approved product for an ultra-rare indication.
- Rocket Pharmaceuticals is a fully integrated, late-stage biotechnology company focused on genetic therapies for rare and often fatal diseases [sec · filed 2026-04-06].
- The company's first FDA-approved product, KRESLADI™, was approved in March 2026 under the accelerated approval pathway for the treatment of severe Leukocyte Adhesion Deficiency-I [sec_mda · as of 2026-08-10].
- As of 2026-06-30, the company reported cash & equivalents of $224.5M, shareholders' equity of $368.8M, and net income of $123.2M, with $0.0M in total revenue [sec].
- The company operates a ~100,000 sq. ft. in-house AAV cGMP manufacturing facility in Cranbury, New Jersey to support clinical and future commercial activities [sec · filed 2026-04-06].
- In September 2026, the company announced FDA alignment on continued enrollment and dosing for the pivotal Phase 2 trial of RP-A501 for Danon disease [news · published 2026-09-15].
What works
- First FDA-approved product (KRESLADI™) achieved in March 2026, demonstrating regulatory execution capability [sec · filed 2026-04-06].
- Strong cash position of $224.5M as of 2026-06-30, bolstered by a $180M sale of a Rare Pediatric Disease Priority Review Voucher [sec_mda · as of 2026-08-10].
- In-house AAV cGMP manufacturing facility provides vertical integration and potential for scaled production [sec · filed 2026-04-06].
- Leadership team has a proven track record of over 20 successful U.S. and international drug approvals and launches [sec · filed 2026-04-06].
What to weigh
- No product revenue generated to date; KRESLADI™ is not expected to generate material revenue in the near term due to the ultra-rare patient population [sec_mda · as of 2026-08-10].
- The company implemented a 30% workforce reduction and deprioritized Fanconi Anemia and Pyruvate Kinase Deficiency programs as part of a strategic realignment [sec · filed 2026-04-06].
- Reliance on a single approved product for an ultra-rare indication creates concentration risk; commercial scalability is uncertain [sec_mda · as of 2026-08-10].
- The RP-A501 program for Danon disease was previously under FDA clinical hold, indicating regulatory and clinical development risks [sec · filed 2026-04-06].
From the filings
Quoted directly from source documents.
FDA approval of KRESLADI™
“On March 27, 2026, Rocket announced FDA approval of KRESLADI™.”
SEC filings · April 6, 2026
Workforce reduction of approximately 30%
“the Company implemented a reduction in the workforce of approximately 30%”
SEC filings · April 6, 2026
Catalyst timeline
- 2020-07-06 · Phase 1 Start: Gene Therapy for Pyruvate Kinase Deficiency (PKD) — Initiation of a Phase I clinical trial evaluating safety of autologous CD34+ cells transduced with a lentiviral vector carrying the coRPK gene.
- 2023-08-29 · Phase 1 Start: RP-A601 (PKP2-ACM) — Start of a Phase 1 dose escalation trial for an AAVrh.74 vector containing the human PKP2a coding sequence in subjects with arrhythmogenic cardiomyopathy.
- 2023-09-05 · Phase 2 Start: RP-A501 (Danon Disease) — Initiation of a Phase 2 study evaluating gene therapy for Danon disease.
Risks & what to watch (4)›
Key risks
- No product revenue — KRESLADI™ is not expected to generate material revenue in the near term [sec_mda · as of 2026-08-10].
- Workforce reduction — 30% workforce reduction and deprioritization of two programs indicate operational strain [sec · filed 2026-04-06].
- Clinical hold history — RP-A501 was under FDA clinical hold, highlighting regulatory risk in pivotal trials [sec · filed 2026-04-06].
- Ultra-rare market — KRESLADI™ addresses an ultra-rare indication, limiting commercial upside [sec_mda · as of 2026-08-10].
What to watch
- Progress of the RP-A501 pivotal Phase 2 trial for Danon disease following FDA alignment in September 2026 [news · published 2026-09-15].
- Commercial launch execution and revenue generation from KRESLADI™ for LAD-I [sec_mda · as of 2026-08-10].
- Advancement of cardiovascular pipeline programs RP-A601 (PKP2-ACM) and RP-A701 (BAG3-DCM) [sec_mda · as of 2026-08-10].
- Cash runway and expense management following the 30% workforce reduction and PRV sale [sec · filed 2026-04-06].
Sources: Catalysts · News · SEC filings · SEC MD&A · Generated October 9, 2026 · How we verify sources →
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Evidence & data
Score breakdownBased on 2 of 6 signals · derived from public data
Pipeline inputs are unavailable for this score. It uses only the available signals shown below.
Cash runway data unavailable
D/E 0.15 · 1% dilution
No tracked pipeline phase
No tracked pipeline candidates
No upcoming catalysts tracked
0 buys · 14 sells (180d)
Regulatory
Financials
Institutional Holdings
Source: SEC Form 13F · as of Mar 2026Institutional holders
196
Total value held
$344.2M
| Holder | Shares | Value |
|---|---|---|
| rtw investments, lp | 17,687,772 | $63.3M |
| blackrock, inc. | 7,834,795 | $28.0M |
| citigroup inc | 5,107,708 | $18.3M |
| vanguard portfolio management llc | 4,690,215 | $16.8M |
| state street corp | 4,485,989 | $16.1M |
| vanguard capital management llc | 3,775,736 | $13.5M |
| maverick capital ltd | 3,567,372 | $12.8M |
| newtyn management, llc | 3,165,900 | $11.3M |
| nuveen, llc | 3,128,753 | $11.2M |
| millennium management llc | 3,118,124 | $11.2M |
Showing top 10 of 196 institutional holders of Rocket Pharmaceuticals Inc.. Open the full 13F history after sign-in.
Short Interest
as of May 19, 2026- % of float
- 24.2%
- Days to cover
- 7.9
- Shares short
- 21,549,583
- Daily short volume
- -
Short percent of float and days-to-cover reflect reported short positioning. Daily short volume is the exchange-reported short share of a single day's trading volume and is noisier. See the most-shorted biotech ranking →
RCKT short interestGovernance
Frequently asked questions
What is the AI investment thesis for Rocket Pharmaceuticals Inc.?
Rocket Pharmaceuticals Inc. (RCKT) is a fully integrated, commercial-stage biotechnology company focused on developing and commercializing genetic therapies for rare and life-threatening diseases, with a primary emphasis on inherited cardiovascular disorders and one FDA-approved product for an ultra-rare indication.
Who is the CEO of Rocket Pharmaceuticals Inc.?
Dr. Gaurav D. Shah M.D. is the chief executive officer of Rocket Pharmaceuticals Inc. (RCKT).
Where is Rocket Pharmaceuticals Inc. headquartered?
Rocket Pharmaceuticals Inc. (RCKT) is headquartered in Cranbury, NJ, United States.
What therapeutic areas does Rocket Pharmaceuticals Inc. focus on?
Rocket Pharmaceuticals Inc. develops therapies across Rare Disease, Gene Therapy, Cardiovascular, Hematology.
The AI overview and BioSniper Score are neutral, data-derived signals built from public SEC filings, ClinicalTrials.gov and FDA records — not investment advice.