Estimated readout windows: registry primary-completion date + 6-12 weeks. Windows already open are shown from "Now". Drug names link to ClinicalTrials.gov.
Stage not disclosed · TerminatedModality: gene therapy
Target granulin (GRN)
Partnerships University of Pennsylvania license terminated for PBFT02 (notice Jun 23, 2026, effective ~Sep 2026); Catalent development/manufacturing agreements terminated Jun 23, 2026.
Indications
Frontotemporal dementia with granulin mutations (FTD-GRN)(Stage not disclosed)
Catalysts
— Q4 2026
Status note Former lead product candidate. On Jun 23, 2026 the company terminated its Penn license for PBFT02 and its Catalent development/clinical-supply agreements in connection with the wind-down of its gene therapy programs and the Jun 24, 2026 merger agreement with Remix Therapeutics (expected close Q4 2026).
PBGM01
Stage not disclosed · ActiveModality: gene therapy
Target beta-galactosidase (GLB1)
Partnerships Outlicensed to Gemma Biotherapeutics (Jul 2024; amended May 2025): up to $15M initial payments, $5M business-milestone contingent, up to $114M development/commercial milestones, single-digit royalties. Gemma assumed Penn payment obligations.
Indications
GM1 gangliosidosis(Stage not disclosed)
Status note Outlicensed program for GM1 gangliosidosis. Company determined to wind down its own gene therapy programs; PBGM01 was outlicensed to Gemma. Clinical phase not disclosed in the excerpts.
PBKR03
Stage not disclosed · ActiveModality: gene therapy
Target galactocerebrosidase (GALC)
Partnerships Outlicensed to Gemma Biotherapeutics (Jul 2024; amended May 2025; economics as per PBGM01).
Indications
Krabbe disease(Stage not disclosed)
Status note Outlicensed program for Krabbe disease. Company determined to wind down its own gene therapy programs; PBKR03 was outlicensed to Gemma. Clinical phase not disclosed in the excerpts.
PBML04
Stage not disclosed · ActiveModality: gene therapy
Target arylsulfatase A (ARSA)
Partnerships Outlicensed to Gemma Biotherapeutics (Jul 2024; amended May 2025; economics as per PBGM01).
Indications
Metachromatic leukodystrophy (MLD)(Stage not disclosed)
Status note Outlicensed program for MLD. Company determined to wind down its own gene therapy programs; PBML04 was outlicensed to Gemma. Clinical phase not disclosed in the excerpts.
Huntington's disease program
Stage not disclosed · TerminatedModality: gene therapy
Target HTT
Partnerships Gemma collaboration (IND-enabling work) terminated by written notice May 21, 2026 (effective within 90 days).
Indications
Huntington's disease(Stage not disclosed)
Status note Active research program for Huntington's disease whose preclinical/IND-enabling work was conducted by Gemma under the Gemma Collaboration Agreement; company terminated that agreement on May 21, 2026 as part of the gene therapy wind-down.
TLE research program
Stage not disclosed · TerminatedModality: gene therapy
Partnerships Gemma collaboration terminated May 21, 2026.
Indications
Temporal lobe epilepsy (TLE)(Stage not disclosed)
Status note Paused research program in temporal lobe epilepsy; terminated along with the Gemma Collaboration Agreement on May 21, 2026 as part of the gene therapy wind-down.
Per-asset detail extracted from the 10-Q filed 2026-08-10. Fields the filing does not state are marked "not disclosed" rather than filled from other sources.
Leukodystrophy; White Matter Disease; Leukoencephalopathies
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Sponsor-matched active trials from ClinicalTrials.gov (1 as sponsor, 2 as collaborator); "Sponsor" = company is the lead sponsor, "Collaborator" = company is a collaborator. The BP column flags big-pharma involvement as lead sponsor or collaborator (excluding the company itself). The Mgmt guide column shows what management guided for readout timing when a guided program matches the trial (hover for the quote, click for the source). Trial listings are not a company pipeline slide.
Institutional holder positions from 13F filings. Q/Q change is vs the prior quarter's filing; % of portfolio is the fund's own reported portfolio weight.
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