Estimated readout windows: registry primary-completion date + 6-12 weeks. Windows already open are shown from "Now". Drug names link to ClinicalTrials.gov.
Approved · ActiveModality: ex vivo CRISPR/Cas9-edited autologous cell therapy
Aliases exagamglogene autotemcel, exa-cel
Target BCL11A
Partnerships Vertex Pharmaceuticals: joint development and commercialization; Vertex leads and has all decision-making; net profits/losses split 40% CRISPR / 60% Vertex; PRV proceeds sharing ($43M or 42% of net proceeds)
Indications
Sickle cell disease(Approved)
Transfusion-dependent beta thalassemia(Approved)
Status note Approved product; Vertex is the manufacturer and exclusive license holder. Phase 3 pediatric studies CLIMB-151 (SCD ages 5-11) and CLIMB-141 (TDT ages 5-11) ongoing with positive ASH December 2025 data.
Zugocabtagene geleucel
Phase 1 · ActiveModality: allogeneic CAR T cell therapy (CRISPR-edited)
Aliases zugo-cel, CTX112
Target CD19
Partnerships Eli Lilly: collaboration and clinical supply agreement to evaluate zugo-cel with pirtobrutinib in aggressive B-cell lymphomas
Status note Next-generation gene-edited healthy-donor allogeneic CAR T targeting CD19; edits designed to enhance potency, reduce exhaustion, evade immunity. RMAT designation for relapsed/refractory follicular lymphoma and marginal zone lymphoma.
CTX310
Phase 1 · ActiveModality: in vivo CRISPR/Cas9 gene editing (LNP-delivered Cas9 mRNA + gRNA)
Target ANGPTL3
Indications
Heterozygous/homozygous familial hypercholesterolemia, mixed dyslipidemia, severe hypertriglyceridemia(Phase 1)
Status note Most advanced in vivo program; Phase 1b prioritizes severe hypertriglyceridemia and refractory hypercholesterolemia after positive Phase 1 data presented November 2025.
Partnerships Sirius Therapeutics: collaboration, option and license agreement (May 2025); 50/50 development cost and profit share; CRISPR leads Phase 3/US commercialization; up to $87.5M milestones
Indications
Thromboembolic disease (VTE prevention in total knee arthroplasty)(Phase 2)
Status note Targets coagulation factor XI mRNA; semi-annual subcutaneous dosing potential. Two Phase 1 trials completed by Sirius.
Status note Regenerative medicine program; preclinical efficacy via direct administration. Separate non-exclusive IP license to Vertex for hypoimmune T1D cell therapies (milestones + royalties).
CTX340
Preclinical · ActiveModality: in vivo CRISPR/Cas9 gene editing
Target AGT (angiotensinogen)
Indications
Refractory hypertension(Stage not disclosed)
Status note IND-enabling studies ongoing.
CTX460
Preclinical · ActiveModality: in vivo gene editing (SyNTase platform)
Target SERPINA1
Indications
Alpha-1 antitrypsin deficiency(Stage not disclosed)
Status note First candidate from proprietary SyNTase editing platform.
CTX321
Preclinical · ActiveModality: in vivo CRISPR/Cas9 gene editing
Target LPA
Indications
Elevated lipoprotein(a)(Stage not disclosed)
Status note Next-generation LPA program with updated guide RNA (~2x potency vs CTX320 in preclinical testing); IND-enabling studies ongoing.
Per-asset detail extracted from the 10-Q filed 2026-08-03. Fields the filing does not state are marked "not disclosed" rather than filled from other sources.
Sponsor-matched active trials from ClinicalTrials.gov (7 as sponsor, 3 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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