Precision BioSciences unveils mitoARCUS program PBGENE-PMM; HBV program on track for 2024 filing

PRECISION BIOSCIENCES INC

Announced PBGENE-PMM, first mitoARCUS candidate targeting m.3243 primary mitochondrial myopathy; CTA/IND submission targeted for 2025.
Lead PBGENE-HBV for chronic hepatitis B on track for CTA/IND in 2024; data shows potential to eliminate cccDNA and integrated HBV DNA.
PBGENE-NVS with Novartis aims for in vivo transgene insertion for sickle cell and beta thalassemia; PBGENE-DMD with Lilly subsidiary excises ~500k base pair dystrophin hot spot.
Partner iECURE-OTC for OTC deficiency showed >20% gene insertion efficiency at 12 months; CTA/IND submission targeted for 2023.
Company highlights ARCUS platform advantages: unique cut, size, simplicity enabling viral/mitochondrial genome elimination, gene excision, and insertion.

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