Estimated readout windows: registry primary-completion date + 6-12 weeks. Windows already open are shown from "Now". Drug names link to ClinicalTrials.gov.
Status note Global Phase 1/2 ELEVATE-44-201: Cohort 1 (6 mg/kg) completed; Cohort 2 (12 mg/kg) fully enrolled; Cohort 3 (up to 18 mg/kg) to follow if needed; expansion cohort planned to support potential US accelerated approval. FDA Rare Pediatric Disease Designation.
Status note Global Phase 1/2 ELEVATE-45-201: Cohort 1 dosing complete; Cohort 2 dosing ongoing.
VX-670
Phase 1 · ActiveModality: EEV-based genetic medicine
Target CUG repeats
Partnerships Vertex: global research collaboration; Vertex running GALILEO Phase 1/2 clinical trial
Indications
Myotonic dystrophy type 1(Phase 1)
Catalysts
— H2 2026
Status note GALILEO global Phase 1/2 completed enrollment and continues dosing in MAD portion; assessing safety, splicing index, and muscle function/strength.
ENTR-601-50
Stage not disclosed · ActiveModality: EEV-conjugated PMO (endosomal escape vehicle)
Target DMD
Indications
Duchenne muscular dystrophy (exon 50 amenable)(Stage not disclosed)
Status note Regulatory applications to be submitted following review of data from lead programs; timeline guidance pending.
Duchenne muscular dystrophy (exon 51 amenable)(Stage not disclosed)
Status note IND-enabling studies completed; regulatory application not yet filed.
ENTR-801
Preclinical · ActiveModality: oligonucleotide
Target USH2A
Indications
Usher syndrome type 2A (exon 13 amenable)(Preclinical)
Status note First ocular clinical candidate (announced December 2025), designed to restore functional usherin protein. Second ocular candidate expected to be announced H2 2026.
Per-asset detail extracted from the 10-Q filed 2026-08-05. Fields the filing does not state are marked "not disclosed" rather than filled from other sources.
Sponsor-matched active trials from ClinicalTrials.gov (3 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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