Estimated readout windows: registry primary-completion date + 6-12 weeks. Windows already open are shown from "Now". Drug names link to ClinicalTrials.gov.
Phase 3 · ActiveModality: in vivo CRISPR-based gene editing therapeutic
Aliases lonvoguran ziclumeran, NTLA-2002
Target kallikrein B1 (KLKB1)
Partnerships Wholly owned.
Indications
Hereditary angioedema (HAE)(Phase 3)
Catalysts
— 2H 2026
— 1H 2027
Status note Wholly-owned investigational in vivo CRISPR therapeutic for HAE (inactivates the KLKB1 gene in the liver) delivered as a single IV infusion. Positive topline data from the global Phase 3 HAELO trial reported Apr 2026. Rolling BLA initiated Apr 2026; launch readiness activities underway.
nex-z
Phase 3 · ActiveModality: in vivo CRISPR-based gene editing therapeutic
Aliases nexiguran ziclumeran, NTLA-2001
Target transthyretin (TTR)
Partnerships Co-development and co-promotion (Co/Co) agreement with Regeneron Pharmaceuticals for ATTR amyloidosis (2018). License and other agreements include up to $130M in contingent payments to Rewrite Therapeutics.
Indications
Transthyretin (ATTR) amyloidosis(Phase 3)
Catalysts
— 2H 2026
Status note Investigational in vivo CRISPR therapeutic for ATTR amyloidosis (inactivates the TTR gene in the liver) delivered as a single IV infusion via LNP technology. Phase 3 MAGNITUDE and MAGNITUDE-2 studies ongoing.
Per-asset detail extracted from the 10-Q filed 2026-08-06. Fields the filing does not state are marked "not disclosed" rather than filled from other sources.
Sponsor-matched active trials from ClinicalTrials.gov (6 as sponsor, 1 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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