Estimated readout windows: registry primary-completion date + 6-12 weeks. Windows already open are shown from "Now". Drug names link to ClinicalTrials.gov.
Partnerships University of Edinburgh: exclusive worldwide license to EXACT technology and optimized MECP2 cassettes (Mar 2022); up to $5.3M regulatory milestones ($0.3M paid for Phase 1/2), up to $25M sales milestones, low single-digit royalties
Indications
Rett syndrome (females)(Stage not disclosed)
Status note First clinical-stage program using EXACT technology. Completed dosing in Phase 1/2 trial for Rett syndrome and completed dosing in the registrational Embolden study (single-arm, baseline-controlled, open-label in females with Rett syndrome), designed to support a potential BLA submission.
Per-asset detail extracted from the 10-Q filed 2026-08-11. 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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