RCKT · CIK 0001281895
What Rocket Pharmaceuticals, Inc. told the SEC could break it.
Rocket's most acute recent setback is clinical: in May 2025, two patients in the Phase 2 pivotal study of RP-A501 for Danon disease each experienced an unexpected serious adverse event, prompting a voluntary pause of a key program. It is also pre-revenue and burning cash fast — a $1.44 billion accumulated deficit, a $223.1 million net loss in 2025, and cash down to about $77.6 million from $163.6 million — leaving it dependent on continued equity and convertible-debt raises to fund its gene-therapy work. And its manufacturing is concentrated, with in-house GMP production at a single Cranbury, New Jersey facility and reliance on third-party manufacturers for AAV supplementation and for all of its LV-program plasmids, vectors, cell banks and final drug product.
3 self-disclosed vulnerabilities, pulled from its own filings, each in the company’s words, with the source. This is the risk register almost nobody reads.
In its own words
What could break it.
Other disclosures
- clinical-safety event in RP-A501 pivotal studyhigh
In May 2025, two patients in the Phase 2 pivotal study of RP-A501 (Danon disease) each experienced an unexpected serious adverse event, prompting a voluntary pause — a material setback for a key program.
“In May 2025, two patients participating in the Phase 2 pivotal study of RP-A501 each experienced an unexpected SAE.”
SEC filing →As of 2026
Liquidity & debt
- pre-revenue; high cash burn vs. declining cashmedium
Rocket has no product revenue, a $1.44B accumulated deficit and a $223.1M net loss in 2025; cash fell to ~$77.6M (from ~$163.6M), so it depends on continued equity/convertible-debt raises to fund its gene-therapy programs.
“As of December 31, 2025, we had an accumulated deficit of $1.44 billion.”
SEC filing →As of 2026
Supplier concentration
- single Cranbury, NJ GMP facility + third-party CMOs (LV programs)medium
Rocket's in-house gene-therapy manufacturing is concentrated at a single Cranbury, NJ GMP facility, and it relies on third-party manufacturers for AAV supplementation and for all LV-program plasmids, vectors, cell banks and final drug product.
“We commenced GMP manufacturing at our facility in Cranbury, New Jersey in 2022. We plan to supplement our own direct manufacturing capabilities with third-party manufacturers for our AAV programs. For our LV programs, we currently rely on third-party manufacturers to produce the plasmids, vectors, cell banks and final drug product for our clinical trials.”
SEC filing →As of 2026
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