Estimated readout windows: registry primary-completion date + 6-12 weeks. Windows already open are shown from "Now". Drug names link to ClinicalTrials.gov.
Phase 1 · ActiveModality: ex vivo prime editing (autologous HSCT)
Target NCF1
Indications
Chronic granulomatous disease (CGD)(Phase 1)
Catalysts
— H1 2027
Status note Ex vivo Prime Editing for CGD correcting the ΔGT mutation in NCF1. Phase 1/2 ongoing in patients undergoing autologous hematopoietic stem cell transplantation; RMAT designation granted Jun 2026.
PM577a
Phase 1 · ActiveModality: in vivo prime editor (LNP-delivered)
Aliases PM577
Target ATP7B
Indications
Wilson disease (H1069Q)(Phase 1)
Catalysts
— 2027
Status note Lead in vivo program. CTA cleared in New Zealand (Jun 2026) and IND cleared by FDA (Jul 2026); global Phase 1/2 trial initiating. Follow-on editors for additional ATP7B mutations in development.
PM647
Preclinical · ActiveModality: in vivo prime editor (LNP-delivered)
Status note In vivo program for AATD; IND application submission and/or CTA filing on track for Q3 2026. (Jul 2026 arbitration: Tribunal confirmed PM647 is within the company's field under the Beam Collaboration Agreement.)
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 (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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