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 autologous HSC base editing
Aliases risto-cel, formerly BEAM-101
Target HBG1/HBG2
Indications
Sickle cell disease (SCD)(Phase 1)
Catalysts
— as early as year-end 2026
Status note Lead program; Phase 1/2 BEACON trial ongoing. Base edits mimic hereditary persistence of fetal hemoglobin (HPFH) SNPs to raise HbF. One patient death reported (respiratory failure, 4 months post-infusion). In vivo HSC-targeted LNP is the next-wave approach for SCD (lead optimization).
BEAM-302
Phase 1 · ActiveModality: in vivo LNP base editing (liver-targeted)
Target SERPINA1
Indications
Alpha-1 antitrypsin deficiency (AATD)(Phase 1)
Status note Corrects the E342K (PiZZ) mutation in a one-time treatment. Phase 1/2 open-label trial ongoing (Part A lung disease, Part B liver disease). FDA alignment on a potential accelerated approval pathway based on AAT biomarkers over 12 months; dose selection for registrational development to be finalized on trial data.
BEAM-301
Phase 1 · ActiveModality: in vivo LNP base editing (liver-targeted)
Target G6PC
Indications
Glycogen storage disease type 1a (GSDIa)(Phase 1)
Catalysts
— 2026
Status note Corrects the R83C mutation in G6PC; open-label, multi-cohort single-ascending-dose Phase 1/2 in the U.S.
BEAM-304
Preclinical · ActiveModality: in vivo LNP base editing (liver-targeted)
Target PAH
Indications
Phenylketonuria (PKU)(Preclinical)
Catalysts
— 2026
Status note Newly announced; initial development targets the two most prevalent PAH variants (R408W first).
Status note Enables the ESCAPE platform (non-genotoxic conditioning); Phase 1 healthy volunteer trial, dosing completion expected 1H 2026.
Pfizer collaboration development candidate (undisclosed)
Stage not disclosed · ActiveModality: in vivo base editing (liver-targeted)
Partnerships Pfizer: exclusive worldwide license opt-in exercised December 2025 on a liver-targeted development candidate from the 2021 research collaboration (three rare-genetic-disease targets)
Status note Pfizer opted in at completion of the 4-year research term; no target or stage disclosed.
Per-asset detail extracted from the 10-Q filed 2026-08-04. 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, 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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