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Pipeline (4 active programs)

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

Capital (as filed)

Cash

$225M cash and cash equivalents · Jun 30, 2026 (10-Q)

Short-term investments

$59.2M Jun 30, 2026

Operating cash flow

−$39.6M quarter Mar 31, 2026 → Jun 30, 2026, year-to-date difference (10-Q)

As stated in coverage

$270M cash, runway Q3 2028 per the update of Oct 6, 2026 — the company's own words as read there, not the filed figure

Cash, investments and operating cash flow are the company's own XBRL facts as filed with the SEC, each shown with its period end and tag. As-stated figures come from the company's own words as read in Biotech Distilled coverage, dated, and routinely count securities and planned financing that a balance sheet does not. Offering rows are the SEC cover pages as filed. Not investment advice. Figures as of Nov 7, 2026.

Biotech Distilled coverage (8)

• 2026-10-07 UPDATE: Rocket Pharmaceuticals (RCKT) - The Webinar Showed Everything Except the Answer 🔒 Paid
• 2026-09-16 UPDATE: Rocket Pharmaceuticals (RCKT) - The FDA Answered, and the Answer Has a Date Attached 🔒 Paid
• 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 are subscriber-only; those marked ★ Distilled Pro are for Distilled Pro members.

Verdict history (8 calls)

Every verdict Biotech Distilled has published on Rocket Pharmaceuticals, oldest first — the full history, never a selection. How calls are scored: the track record.

1. 2026-03-06 Watch Rocket Pharmaceuticals (RCKT): Scientific Deep Dive for Lead and Pipeline Products Compelling mid-to-late-stage genetic assets but severe AAV toxicity history and manufacturing risks warrant waiting for the PDUFA decision and post-dilution entry.
2. 2026-03-28 Hold UPDATE: Rocket Pharmaceuticals (RCKT) - Rocket Deploys KRESLADI Patch, But Internal Code Shows Severe Tech Debt KRESLADI accelerated approval is a genuine win but the commercial market is tiny, financial overhang from ATM and PRV-pending is suffocating, and clean Danon Phase 2 safety logs are still needed before establishing a position.
3. 2026-04-29 Hold speculative UPDATE: Rocket Pharmaceuticals (RCKT) - The PRV Lands at $180M, Runway Bought Through Q2 2028 The $180M PRV sale materially narrows the dilution gap and extends runway to Q2 2028, improving asymmetry enough to warrant a starter position, though the AAV toxicity question remains the binding constraint.
4. 2026-05-09 Speculative buy speculative UPDATE: Rocket Pharmaceuticals (RCKT) - The Danon Clock is Running Danon Phase 2 dosing reinitiated on schedule and pro forma cash of $322.6M removes the forced-raise tail risk, but the capillary leak binary remains unresolved so conviction and sizing are unchanged.
5. 2026-08-03 Buy core UPDATE: Rocket Pharmaceuticals (RCKT) - The Canary Came Back Out of the Mine Three sequential Danon patients dosed at the recalibrated dose with zero capillary leak or TMA materially reduces the platform-failure tail risk, improving asymmetry enough to upgrade from speculative to core buy.
6. 2026-08-11 Buy core UPDATE: Rocket Pharmaceuticals (RCKT) - The Observation Window Closed Clean All three Danon patients completed the full protocol-defined safety observation window with no TMA or capillary leak, confirming and extending the August 3 upgrade rather than adding a new leg.
7. 2026-09-16 Buy core UPDATE: Rocket Pharmaceuticals (RCKT) - The FDA Answered, and the Answer Has a Date Attached FDA confirmed the 12-patient pivotal population, recalibrated dose, and co-primary endpoints intact, retiring regulatory uncertainty but introducing timeline risk as dosing runs through mid-2027 and the efficacy read lands near the Q2 2028 runway.
8. 2026-10-07 Speculative buy speculative UPDATE: Rocket Pharmaceuticals (RCKT) - The Webinar Showed Everything Except the Answer No efficacy data from the recalibrated dose was shown, a Hercules debt facility places a lien on IP ahead of equity, a Phase 1 transplant raises durability questions, and the runway barely reaches the 2028 readout, making new money better treated as a lottery ticket.

• Long call · opened May 11, 2026 at $3.59 · -33.6% vs XBI +13.0% (Nov 6, 2026)

Every verdict Biotech Distilled has published on this company, in order, with the post it came from — the full history, never a selection. Position figures are the same ones shown on /track-record, computed by the same method. Editorial opinion, not investment advice.

Timing, in the company's words (1 quote from 1 filing)

Sentences quoted verbatim from the company's SEC filings, newest filing first. Each sits under the filing it appeared in; the "matched to" tag names the calendar row it was read for.

• 2026-08-10 10-Q in filings ↓

  • “The Company is actively engaging with the FDA to align on the path to dosing additional patients and completing the pivotal Phase 2 trial and expects to provide an update on the regulatory pathway in the second half of 2026.”

    matched to RP-A501 (Danon disease) · trial initiation · read as second half of 2026

Timing sentences quoted from the company's own SEC filings, each under the filing it appeared in. The 'matched to' tag is the calendar row the sentence was read for; the filing is the source. Not investment advice.

Filings & releases (29)

SEC filings from EDGAR and press releases captured from the company's investor-relations site, newest first. Releases captured from May 7, 2026; filings from March 2026. Summaries come from the BD update that read the document; "BD extracted" counts point at the pipeline table above. All companies →

• Oct 15, 2026ReleaseRocket Pharmaceuticals to Participate in Chardan’s 10th Annual Healthcare Conference
• Oct 7, 2026ReleaseRocket Pharmaceuticals Highlights FDA-Aligned Pivotal Phase 2 Trial and Updated Clinical Findings for RP-A501 Following Danon Disease Investor WebinarBD extracted 1 program update from this document → RCKT pipeline
• Oct 6, 2026ReleaseRocket Pharmaceuticals Secures Strategic Credit Facility for Up to $150 Million from Hercules Capital→ BD updateSummary on the 8-K
• Oct 6, 20268-KCurrent report: material agreement, debt / obligation, unregistered sale of equity · EX-99.1 EXHIBIT 99.1→ BD updateRocket closed a $150M Hercules debt facility and hosted a pivotal-program webinar showcasing 7-year Phase 1 durability data and the first Phase 2 efficacy numbers — two developments that materially reshape both the cash runway and the clinical risk picture heading into the mid-2028 readout.BD extracted 1 program update from this document → RCKT pipeline
• Sep 15, 2026ReleaseRocket Pharmaceuticals Announces FDA Alignment on Path to Complete Pivotal Phase 2 Trial of RP-A501 in Danon Disease→ BD updateSummary on the 8-K
• Sep 15, 20268-KCurrent report: Reg FD disclosure · EX-99.1 EXHIBIT 99.1→ BD updateThe FDA just handed Rocket a clear, locked regulatory runway for the Danon pivotal: 12 patients, co-primary endpoints preserved, and continued enrollment green-lit — the exact alignment the August 10 update said was the "tell worth watching."BD extracted 1 program update from this document → RCKT pipeline
• Sep 2, 202625-NSEDelisting notice
• Aug 27, 20268-KCurrent report: officer / director change
• Aug 10, 2026ReleaseRocket Pharmaceuticals Reports Second Quarter 2026 Financial Results and Highlights Recent Progress
• Aug 10, 20268-KCurrent report: results of operations · EX-99.1 EXHIBIT 99.1
• Aug 10, 202610-QQuarterly report
• Aug 3, 2026ReleaseRocket Pharmaceuticals Announces Positive Clinical Safety Update from Initial Three Patients Treated with RP-A501 Under Modified Phase 2 Protocol for Danon DiseaseBD extracted 1 program update from this document → RCKT pipeline
• Aug 3, 20268-KCurrent report: Reg FD disclosure · EX-99.1 EXHIBIT 99.1
• Jun 30, 20268-KCurrent report: officer / director change
• Jun 12, 2026ReleaseRocket Pharmaceuticals Announces Closing of Sale of Rare Pediatric Disease Priority Review Voucher for $180 Million
• Jun 12, 20268-KCurrent report: acquisition / disposition, Reg FD disclosure · EX-99.1 EXHIBIT 99.1
• May 28, 2026ReleaseRocket Pharmaceuticals to Participate in Upcoming Investor Conferences
• May 21, 20268-KCurrent report: shareholder vote
• May 7, 2026ReleaseRocket Pharmaceuticals Reports First Quarter 2026 Financial Results and Highlights Recent Progress
• May 7, 20268-KCurrent report: results of operations · EX-99.1 EXHIBIT 99.1
• May 7, 202610-QQuarterly report
• Apr 28, 20268-KCurrent report: material agreement, Reg FD disclosure · EX-99.1 EXHIBIT 99.1
• Apr 27, 2026SC TO-ISC TO-I
• Apr 6, 2026DEFA14AProxy soliciting material
• Apr 6, 2026DEF 14AProxy statement
• Mar 27, 20268-KCurrent report: Reg FD disclosure · EX-99.1 EXHIBIT 99.1
• Mar 10, 20268-KCurrent report: material agreement, agreement terminated
• Mar 10, 2026424B5Prospectus supplement (offering)
• Mar 2, 2026S-3Shelf registration