Precision BioSciences develops ARCUS-based in vivo editing for gene elimination, excision, and insertion. Delivery and evidence differ by program; the company describes AAV delivery for its DMD gene-excision approach.
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Technology, programs & evidence
Precision BioSciences develops ARCUS-based in vivo editing for gene elimination, excision, and insertion. Delivery and evidence differ by program; the company describes AAV delivery for its DMD gene-excision approach.
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Related news & literature
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Precision BioSciences said an independent safety board endorsed additional dosing in the Phase 1/2 FUNCTION-DMD study after reviewing the first PBGENE-DMD recipient's eight-week observation period. No serious adverse events or adverse events of special interest were reported in that patient. The AAV-delivered gene-editing program remains investigational; this single-patient review does not establish efficacy or broader safety.
Precision BioSciences reported updated PBGENE-HBV Phase 1 data covering 25 patients and 59 administrations. The company described pgRNA loss and liver-biopsy evidence of cccDNA reduction at the expected go-forward dose of 0.4 mg/kg, with no dose-limiting toxicities reported. It plans Phase 2 evaluation, including assessment after nucleos(t)ide analog withdrawal; viral cure has not been established.
Precision BioSciences updated its ARCUS in vivo editing portfolio in its third-quarter report. The company described ongoing ELIMINATE-B evaluation of PBGENE-HBV and the Phase 1/2 FUNCTION-DMD study of PBGENE-DMD, which uses AAV delivery. Planned data releases and management expectations should be distinguished from completed clinical outcomes.
A Nature Materials study describes mRNA-loaded LNPs that reprogram tumor-associated macrophages into Claudin18.2-targeting CAR macrophages. The lipid formulation also promotes TREM2 internalization and degradation, reshaping immune responses. The original abstract reports tumor suppression and longer survival with PD-1 blockade in mice, plus ex vivo patient-sample findings. These findings do not demonstrate clinical efficacy in treated patients.
CSPC announced initiation of the first Chinese center for SYS6055-003, a Phase 1/2 trial in relapsed or refractory B-cell non-Hodgkin lymphoma. SYS6055 uses lentiviral vectors to generate CD19-targeting CAR-T cells in vivo. The company said recruitment and screening are underway following October clinical-trial authorization. Center initiation does not establish patient dosing, efficacy, or marketing approval.
Azalea reported SITC preclinical findings for its dual-vector in vivo CAR-T platform, combining targeted CRISPR-Cas9 delivery with AAV-mediated CAR insertion at the TRAC locus. The company described leukemia clearance and protection after tumor rechallenge in selected mouse groups, plus dual-antigen and armored designs. These are animal findings, not evidence of efficacy or safety in patients.
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