Passage BIO, Inc.
PASG evidence brief
What we can confirm
- BioSniper Score:
- 5.2 / 10
- Market Cap:
- $16.1M
- Lead Asset:
- pbft02 (Phase 1/Phase 2)
What it means
What to watch
Prepared research question
What matters most for PASG—catalysts, financial risk and supporting evidence?
Traceable citations · Unknowns marked
BioSniper AI · PASG
Passage BIO, Inc. is a clinical-stage genetic medicines company focused on developing PBFT02, a gene replacement therapy for frontotemporal dementia (FTD), that has initiated a strategic review and is winding down its principal programs after the FDA required a randomized controlled registrational trial.
Key risk: Program termination risk
BioSniper Score
Based on 4 of 6 signals
Market Cap
$16.1M
Cash Runway
—
Lead Asset
pbft02 (Phase 1/Phase 2)
Next Catalyst
—
Financial data as of Jun 30, 2026.
Tracking 1 pipeline candidate · 1 clinical trial for Passage BIO, Inc..
Similar companies
Passage BIO, Inc. is a clinical-stage genetic medicines company focused on developing PBFT02, a gene replacement therapy for frontotemporal dementia (FTD), that has initiated a strategic review and is winding down its principal programs after the FDA required a randomized controlled registrational trial.
- Lead product candidate PBFT02 is a gene replacement therapy using an AAV1 capsid to deliver a functional GRN gene for FTD-GRN [sec_mda · as of 2026-08-10].
- As of 2026-06-30, the company reported cash & equivalents of $24.2M and a net loss of -$7.8M for the quarter [sec].
- The company initiated a strategic review process in April 2026 intended to maximize shareholder value [news · published 2026-04-20].
- During Q2 2026, the company terminated or began winding-down its principal program, collaboration, license, and manufacturing arrangements [sec_mda · as of 2026-08-10].
- As of March 31, 2026, the accumulated deficit was $712.3 million [sec_mda · as of 2026-05-12].
What works
- PBFT02 demonstrated robust, durable elevation in CSF PGRN levels and improvement in plasma NfL, a disease progression biomarker, compared to natural history [news · published 2025-08-12].
- PBFT02 administration resulted in improvements in two disease progression biomarkers, reducing brain atrophy and stabilizing plasma NfL levels [news · published 2026-04-20].
- The company aligned with the FDA on an analytical approach to establish comparability of a high-productivity, suspension-based manufacturing process for PBFT02 [news · published 2025-11-10].
What to weigh
- The company has incurred recurring losses and negative cash flows from operations, with an accumulated deficit of $712.3 million as of March 31, 2026 [sec_mda · as of 2026-05-12].
- FDA feedback from a recent Type C meeting indicated a randomized controlled registrational trial will be required for PBFT02 in FTD-GRN, increasing development risk [news · published 2026-04-20].
- During Q2 2026, the company terminated or began winding-down its principal program, collaboration, license, and manufacturing arrangements, signaling a major strategic shift [sec_mda · as of 2026-08-10].
- Cash runway was previously projected into 1Q 2027, but with only $24.2M in cash as of 2026-06-30 and ongoing losses, the timeline is uncertain [news · published 2025-11-10] [sec].
From the filings
Quoted directly from source documents.
PBFT02 is a gene replacement therapy for FTD-GRN using an AAV1 capsid.
“Our lead clinical product candidate was PBFT02, a gene replacement therapy that utilizes an adeno-associated virus serotype 1, or AAV1, capsid to deliver a functional granulin gene, or GRN , encoding progranulin, or PGRN, to the brain via intra cisterna magna, or ICM, administration.”
SEC MD&A · August 10, 2026
The company initiated a strategic review in April 2026.
“The Company initiated a strategic review process intended to maximize shareholder value”
News · April 20, 2026
FDA required a randomized controlled registrational trial for PBFT02 in FTD-GRN.
“FDA feedback from recent Type C meeting indicated a randomized controlled registrational trial will be required for PBFT02 in FTD-GRN”
News · April 20, 2026
The company began winding down its principal programs in Q2 2026.
“During the second quarter of 2026, we terminated or began winding-down our principal program, collaboration, license, and manufacturing arrangements.”
SEC MD&A · August 10, 2026
Risks & what to watch (4)›
Key risks
- Program termination risk — The company has terminated or is winding down its principal programs, including PBFT02, as of Q2 2026 [sec_mda · as of 2026-08-10].
- Regulatory requirement for RCT — FDA requires a randomized controlled trial for PBFT02 in FTD-GRN, increasing cost and timeline [news · published 2026-04-20].
- Cash runway uncertainty — With $24.2M cash as of 2026-06-30 and ongoing losses, the company may need additional funding soon [sec].
- Accumulated deficit — Accumulated deficit of $712.3M as of March 31, 2026, with no product revenue [sec_mda · as of 2026-05-12].
What to watch
- Outcome of the strategic review process initiated in April 2026 [news · published 2026-04-20].
- Any updates on the winding-down of principal programs and potential asset sales [sec_mda · as of 2026-08-10].
- Cash position and any financing activities given the $24.2M cash as of 2026-06-30 [sec].
- Regulatory feedback on FTD-GRN registrational trial design, previously expected in 1H 2026 [news · published 2025-11-10].
Sources: News · SEC filings · SEC MD&A · Generated September 24, 2026 · How we verify sources →
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Evidence & data
Score breakdownBased on 4 of 6 signals · derived from public data
Cash runway data unavailable
D/E 4.65 · 0% dilution
Lead: Phase 1/Phase 2
1 candidate
No upcoming catalysts tracked
1 buy · 0 sells (180d)
Pipeline
Regulatory
Financials
Short Interest
as of May 22, 2026- % of float
- 5.6%
- Days to cover
- 1.2
- Shares short
- 145,029
- Daily short volume (Oct 9, 2026)
- 66.5%
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 →
PASG short interestGovernance
Frequently asked questions
What is the AI investment thesis for Passage BIO, Inc.?
Passage BIO, Inc. is a clinical-stage genetic medicines company focused on developing PBFT02, a gene replacement therapy for frontotemporal dementia (FTD), that has initiated a strategic review and is winding down its principal programs after the FDA required a randomized controlled registrational trial.
What is Passage BIO, Inc.'s lead drug candidate?
Passage BIO, Inc.'s most advanced tracked candidate is pbft02 (Phase 1/Phase 2) for Frontotemporal Dementia.
Who is the CEO of Passage BIO, Inc.?
Dr. William Chou M.D., Ph.D. is the chief executive officer of Passage BIO, Inc. (PASG).
Where is Passage BIO, Inc. headquartered?
Passage BIO, Inc. (PASG) is headquartered in Philadelphia, PA, United States.
What therapeutic areas does Passage BIO, Inc. focus on?
Passage BIO, Inc. develops therapies across Neurology.
The AI overview and BioSniper Score are neutral, data-derived signals built from public SEC filings, ClinicalTrials.gov and FDA records — not investment advice.