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: in vivo gene editing (ARCUS, LNP-delivered)
Target HBV cccDNA
Indications
Chronic hepatitis B (HBV)(Phase 1)
Status note Wholly owned in vivo ARCUS gene-editing program designed to eliminate covalently closed circular DNA (cccDNA), the source of HBV replication. Global first-in-human Phase 1/2a ELIMINATE-B trial enrolling at sites in Hong Kong, New Zealand, the U.S. and Moldova, expanding to the U.K.
PBGENE-DMD
Phase 1 · ActiveModality: in vivo gene editing (ARCUS, AAV-delivered)
Target dystrophin gene (exon 45-55 excision)
Indications
Duchenne muscular dystrophy (DMD)(Phase 1)
Catalysts
— End of 2026
Status note Wholly owned development candidate for DMD: two complementary ARCUS nucleases in a single AAV excise exons 45-55 of the dystrophin gene, restoring near full-length dystrophin protein expression. Phase 1/2 FUNCTION-DMD trial in ambulatory boys ages 2-7 with mutations between exons 45 and 55; two sites active (Arkansas Children's, Washington University). FDA Rare Pediatric Disease designation (Jun 2025), Orphan Drug Designation (Jul 2025), Fast Track (Feb 2026); PRV-eligible.
Partnerships Oncology rights licensed to Imugene Limited/Imugene US (Aug 2023; covers up to three additional research candidates). Autoimmune and non-cancer rights licensed to TG Therapeutics (Jan 2024; $10M upfront, up to $288.6M milestones; $7.5M clinical milestone achieved Mar 2026).
Indications
Diffuse large B-cell lymphoma (DLBCL)(Phase 1)
Progressive multiple sclerosis(Phase 1)
Status note Allogeneic CAR-T therapy. Imugene continues development in DLBCL with written FDA guidance aligned on the registrational pathway (dosing, population, endpoints, manufacturing). TG Therapeutics evaluating azer-cel in a Phase 1 trial in progressive multiple sclerosis.
PBGENE-3243
Preclinical · ActiveModality: in vivo gene editing (mitochondria-targeted ARCUS)
Target mutant mitochondrial DNA (m.3243)
Status note Wholly owned candidate for m.3243-associated mitochondrial disease: mitochondria-targeted ARCUS nucleases designed to eliminate mutant mitochondrial DNA and shift heteroplasmy. Development paused to prioritize PBGENE-HBV and PBGENE-DMD.
ECUR-506
Stage not disclosed · ActiveModality: in vivo gene editing (ARCUS gene insertion)
Target OTC gene (gene insertion)
Partnerships Developed by iECURE, Inc. under a development and license agreement (Aug 2021); Precision eligible for milestones and mid-single to low-double digit royalties; iECURE uses Precision's PCSK9-directed ARCUS nuclease.
Indications
Neonatal onset ornithine transcarbamylase (OTC) deficiency(Stage not disclosed)
Catalysts
— First half of 2026
Status note ARCUS-mediated targeted gene insertion approach for neonatal onset OTC deficiency. OTC-HOPE study ongoing in the U.K., U.S., Australia and Spain; FDA Regenerative Medicine Advanced Therapy (RMAT) designation. FDA alignment reached on primary/key secondary endpoints, comparators and study size that could support a BLA.
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.
Pipeline
Lead drug
PBGENE-DMD (IV)
Lead indication
Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD
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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