Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping(Phase 3)
Status note Proprietary Fab targeting TfR1 conjugated to a PMO promoting skipping of DMD exon 51. Phase 1/2 DELIVER trial; global confirmatory Phase 3 FORZETTO commenced Q2 2026 with FDA-aligned design. FDA accepted the BLA for review in July 2026 (no action date stated in the filing). Orphan Drug designation from FDA, EMA, and Japan MHLW.
Status note Proprietary Fab targeting TfR1 conjugated to an ASO reducing toxic nuclear DMPK RNA. Evaluated in ACHIEVE, a global Phase 1/2 trial designed to be registrational. FDA Fast Track and Orphan Drug designations (FDA, EMA).
Status note Reduces DUX4 expression in muscle tissue. FDA cleared the IND application in July 2026 to initiate a Phase 1 clinical trial; placebo-controlled design with 96-week open-label extension. Filing describes traditional approval pathway pursuit in the U.S.
Status note Enzyme replacement therapy engineered via the FORCE platform to deliver the lysosomal enzyme GAA addressing the deficiency that causes Pompe disease.
Per-asset detail extracted from the 10-Q filed 2026-07-29. Fields the filing does not state are marked "not disclosed" rather than filled from other sources.
Sponsor-matched active trials from ClinicalTrials.gov (0 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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