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 · ActiveModality: AAVrh10-based gene therapy (intravenous)
Target frataxin (FXN)
Partnerships Adverum: exclusive license for FA cardiomyopathy materials/technology (Jan 2021; $7.5M upfront); Cornell: license agreements (May 2020) and investigator-initiated trial
Indications
Friedreich ataxia (FA) cardiomyopathy(Stage not disclosed)
Catalysts
— first half of 2028
Status note SUNRISE-FA Phase 1/2 ongoing; SUNRISE-FA 2 pivotal study initiated Jun 2026 with protocol/SAP finalized incorporating FDA feedback, intended to support BLA under accelerated approval.
Partnerships Stelios Therapeutics: acquired Jul 2021 (AAV gene therapies for ACM and TNNI3-HCM); up to $12.5M remaining development milestones
Indications
Arrhythmogenic cardiomyopathy caused by PKP2 mutations (PKP2-ACM)(Phase 1)
Status note HEROIC-PKP2 first-in-human, 52-week open-label dose-ascending trial; 10 participants dosed; interim clinical data update Jan 2026.
Per-asset detail extracted from the 10-Q filed 2026-08-12. Fields the filing does not state are marked "not disclosed" rather than filled from other sources.
Sponsor-matched active trials from ClinicalTrials.gov (7 as sponsor, 0 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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