Develops one-time gene therapies for patients with neurodegenerative diseases, including frontotemporal dementia.
10-K for FY2025, filed Mar 3, 2026
No revenue reported yet.
10-K for FY2025, filed Mar 3, 2026
Third parties obtain licenses to research, develop, manufacture and commercialize product candidates, and may receive transition services; Gemma receives rights to pediatric programs under sublicense agreements.
10-K for FY2025, filed Mar 3, 2026
8-K, filed Sep 3, 2026
On September 2, 2026, the Company, Merger Sub, Peregrine Merger Sub, LLC, a Delaware limited liability company and a wholly owned…View ↗
Portfolio refocused
The earlier report described clinical programs for frontotemporal dementia, Krabbe disease and metachromatic leukodystrophy, plus other candidates; the latest centers clinical development on its frontotemporal dementia gene therapy and says pediatric programs were outlicensed to Gemma.
10-K for FY2022, filed Mar 6, 2023 10-K for FY2025, filed Mar 3, 2026
Broader disease targets
The earlier report described the lead gene therapy for progranulin-deficient frontotemporal dementia; the latest also discusses testing that therapy in another genetic form of frontotemporal dementia and potential use for ALS.
10-K for FY2022, filed Mar 6, 2023 10-K for FY2025, filed Mar 3, 2026We are currently developing PBFT02, which utilizes an AAV1 capsid to deliver a functional copy of the granulin gene, or GRN, encoding for…View ↗
Competes with AviadoBio, Kyowa Kirin, QurAlis, Denali Therapeutics, Takeda Pharmaceutical, Vesper Bio, Arkuda Therapeutics, Johnson & Johnson, Transposon Therapeutics, Evox Therapeutics, Latus Bio, uniQure, and Voyager Therapeutics
10-K for FY2025, filed Mar 3, 2026For the treatment of FTD, there are no approved disease-modifying therapies. We consider our most direct competitor with respect to PBFT02…View ↗
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