Estimated readout windows: registry primary-completion date + 6-12 weeks. Windows already open are shown from "Now". Drug names link to ClinicalTrials.gov.
Partnerships Indiana University (exclusive license, 2016; Wake Forest license expired Dec 2025)
Indications
Friedreich's ataxia(Phase 2)
Catalysts
— second half of 2026
Status note Subcutaneously administered recombinant FXN-delivery fusion protein (CPP platform). Breakthrough Therapy Designation granted Feb 2026; rolling BLA submitted (first module Jun 2026). Confirmatory global Phase 3 study with first dosing expected mid-2026.
Per-asset detail extracted from the 10-Q filed 2026-08-04. Fields the filing does not state are marked "not disclosed" rather than filled from other sources.
Sponsor-matched active trials from ClinicalTrials.gov (2 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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