Estimated readout windows: registry primary-completion date + 6-12 weeks. Windows already open are shown from "Now". Drug names link to ClinicalTrials.gov.
Partnerships Alnylam: March 2026 research collaboration to discover and validate novel cardiovascular gene targets (upfront payment; target-by-target license)
Status note Investigational AAV-based gene therapy for MYBPC3-associated HCM, caused by insufficient myosin-binding protein C (MyBP-C). Clinical-stage; one-time intravenous infusion studied in a first-in-human trial. Company pursuing alignment with regulators on pivotal trial plans.
TN-401
Phase 1 · ActiveModality: gene therapy (AAV)
Target PKP2 gene
Indications
PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC/ACM)(Phase 1)
Catalysts
— None
Status note Investigational AAV-based gene therapy for ARVC caused by PKP2 mutations. Phase 1 trial evaluating two dose levels (3E13 vg/kg and 6E13 vg/kg) of a one-time IV infusion; pursuing alignment with regulators on pivotal trial plans.
TN-301
Phase 1 · ActiveModality: small molecule
Target HDAC6
Indications
Duchenne muscular dystrophy (DMD)(Phase 1)
Heart failure with preserved ejection fraction (HFpEF)(Phase 1)
Status note Selective HDAC6 inhibitor. Phase 1 SAD/MAD study in healthy volunteers (up to 300mg QD for 14 days). FDA granted Orphan Drug and Rare Pediatric Drug Designations as a DMD treatment.
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 (4 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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