Passage BIO (PASG) Company Report

Passage Bio reported a preliminary, unaudited cash balance of approximately $46 million as of December 31, 2025, which is expected to fund operating expenses and capital expenditure requirements into the first quarter of 2027.
The company plans to initiate discussions with the FDA on a registrational study design for PBFT02 in FTD-GRN in 1H 2026 and will report updated interim safety and biomarker data from Dose 2 in FTD patients during the same period.
For its Huntington's disease preclinical program, Passage Bio expects to declare a clinical candidate in 2H 2026.
Interim data for PBFT02 in FTD-GRN patients, as of June 15, 2025, showed robust, durable increases in CSF Progranulin and early evidence of improvement in plasma NfL, with the therapy being generally well tolerated.

Jan 12, 2026, 12:01 PM

Passage Bio Updates on FTD GRN Program, FDA Strategy, and Financial Outlook

PASG

Guidance Update

New Projects/Investments

Passage Bio is advancing its lead gene therapy program for frontotemporal dementia with a granular mutation (FTD GRN), aiming to generate more progranulin using an AAV approach, which differentiates it from a recently failed competitive program.
The company is targeting an earlier patient population (CDR one or 0.5) for its FTD GRN study and is encouraged by recent FDA guidance on rare disease evidence principles, which may allow for single-arm studies given the disease's characteristics.
Key catalysts in the first half of next year include a data refresh and seeking FDA guidance on the registration path for the FTD GRN program.
Passage Bio has developed a high-productivity suspension manufacturing process for its gene therapy, capable of treating over a thousand patients per 200-liter batch, and has aligned with the FDA on an analytical comparability path.
The company's cash balance is expected to fund operations into 2027, with an annual spending of approximately $30 million.

Nov 12, 2025, 2:30 PM

Passage Bio Provides Update on FTD-GRN Gene Therapy Program and Financial Runway

PASG

New Projects/Investments

Guidance Update

Passage Bio's lead clinical program is for frontotemporal dementia with the granulin mutation (FTD-GRN), utilizing a gene therapy approach to generate more progranulin, which differentiates it from a competitor's recent Phase 3 disappointment.
The company aims to achieve CSF progranulin levels at or above the upper limit of normal (8 nanograms per mL), with early data from dose two showing levels near eight at 30 days.
Passage Bio is encouraged by recent FDA guidance on rare disease evidence principles, which suggests openness to single-arm studies for diseases like FTD-GRN, and plans to discuss the registration path with the FDA in the first half of 2026.
The company's cash balance is projected to last into Q1 2027, with an annual spend of approximately $30 million.
Passage Bio has developed a high-productivity suspension manufacturing process capable of treating over 1,000 patients per 200-liter batch, which they intend to use for their registrational cohort.

Nov 12, 2025, 2:30 PM

Passage Bio Outlines FTD-GRN Program Strategy and Regulatory Path

PASG

Guidance Update

New Projects/Investments

Passage Bio's lead clinical program is focused on frontotemporal dementia with the granulin mutation (FTD-GRN), with a preclinical program also in Huntington's disease.
To address a competitor's recent phase III disappointment, Passage Bio is differentiating its FTD-GRN approach by using AAVs to generate more progranulin and by targeting earlier-stage patients (CDR 1 or 0.5).
Early data from their dose two shows CSF progranulin levels near 8 nanograms per mL at 30 days, with the goal of reaching or exceeding the upper limit of normal (8 nanograms per mL).
The company plans an initial discussion with the FDA in the first half of 2026 to clarify the registration path, encouraged by recent rare disease evidence principles that may allow for single-arm studies under specific conditions.
Passage Bio's cash balance is projected to last into the first quarter of 2027, with an annual spend of approximately $30 million.

Nov 12, 2025, 2:30 PM

Passage Bio Reports Q3 2025 Financial Results and Provides Business Highlights

PASG

Earnings

Guidance Update

Passage Bio reported cash, cash equivalents, and marketable securities of $52.8 million as of September 30, 2025, with an anticipated cash runway into Q1 2027.
For the third quarter ended September 30, 2025, the company reported a net loss of $7.7 million, or $2.44 per basic and diluted share.
The company is actively enrolling Cohort 3 (FTD-GRN) and Cohort 4 (FTD-C9orf72) patients in its upliFT-D study.
Passage Bio has aligned with the FDA on an analytical approach for its high-productivity PBFT02 manufacturing process and expects to obtain regulatory feedback on the FTD-GRN registrational trial design in 1H 2026.

Nov 10, 2025, 9:15 PM

UniCure, MIRA, and Passage Bio Discuss Progress and Challenges in Neurodegenerative Gene Therapies

PASG

Product Launch

New Projects/Investments

Guidance Update

UniCure announced 75% statistically significant slowing of disease progression in Huntington's disease patients treated with AMT-130 after three years of follow-up, with a pre-BLA meeting expected in Q4 2025 and BLA submission in Q1 2026.
MIRA detailed its Parkinson's program, which has demonstrated three positive studies and two positive studies UPDRS against sham, and is leveraging AI technology to show physiological brain changes and strengthen pivotal study endpoints.
Passage Bio reported its lead clinical program for frontotemporal dementia (FTD) with granulin mutation shows high durable levels of target engagement of CSF progranulin and stabilization of plasma neurofilaments.
All three companies highlighted the advantages of local, targeted delivery for neurodegenerative gene therapies, emphasizing enhanced bioavailability and reduced systemic safety risks.
Panelists observed that regulatory bodies, including the FDA, are demonstrating increased flexibility for rare diseases with high unmet needs, supporting novel trial designs, biomarkers, and single-arm studies, as outlined in the FDA's rare disease evidence principles.

Oct 21, 2025, 7:30 PM

Genetic Medicines Conference Highlights Progress in Neurodegenerative Disease Gene Therapies

PASG

Product Launch

New Projects/Investments

uniQure reported remarkable findings from its Huntington's disease program (AMT-130), including a 75% statistically significant slowing of disease progression based on composite UHDRS, with a pre-BLA meeting expected in Q4 and BLA submission in Q1 2026.
Mira highlighted its Parkinson's program as the only treatment to have three positive studies and two positive UPDRS studies against SHAM, utilizing an AI partnership to demonstrate physiological changes in the brain and neuroprotective effects, attributing its incredibly good safety to local delivery and the AAV2 capsid.
Passage Bio presented its lead clinical program for frontotemporal dementia (FTD) with GRN mutation, showing high durable levels of target engagement of CSF progranulin and stabilization of plasma neurofilaments, with more data anticipated next year.
Panelists discussed key challenges in gene therapy for neurodegenerative diseases, such as delivery to the brain and the slow progression and heterogeneity of diseases, while also noting the FDA's increased flexibility for rare diseases with high unmet needs regarding trial designs and biomarkers.