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Precision BioSciences (DTIL) investor relations material
Precision BioSciences Sidoti's Year End Virtual Investor Conference summary
Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.Strategic focus and financial position
Company is exclusively focused on two in-vivo gene editing programs: PBGENE-HBV for hepatitis B and PBGENE-DMD for Duchenne muscular dystrophy.
Recent $75 million financing extends cash runway through 2028, supporting clinical milestones and data readouts.
ARCUS gene editing platform has demonstrated clinical validation in multiple indications, including partnerships and internal programs.
Ongoing discussions with potential partners for commercialization, especially for the HBV program.
Company maintains a disciplined, focused approach to capital allocation and program execution.
PBGENE-HBV (Hepatitis B) program progress
PBGENE-HBV targets the root cause of hepatitis B by eliminating cccDNA, aiming for a functional cure.
Phase 1 ELIMINATE-B trial has shown dose-dependent antiviral activity and a manageable safety profile across three cohorts.
Higher dose cohorts demonstrate sustained S antigen reduction, with some patients nearing levels where cure testing is possible.
Plans to complete cohort three dosing, explore shorter dosing intervals, and expand to more patients before phase 2.
Data readouts expected at major liver conferences and throughout 2026.
PBGENE-DMD (Duchenne muscular dystrophy) program progress
PBGENE-DMD uses gene editing to excise mutations in exons 45-55, potentially benefiting up to 60% of DMD patients.
Preclinical studies show restoration of functional dystrophin protein above the 5% threshold linked to milder disease.
Function-DMB trial designed as a phase 1/2/3 study, aiming to enroll first patients in 2026 and read out data by end of 2026.
Safety is prioritized with aggressive immunomodulation and top clinical sites.
Potential for BLA filing by end of 2028 if data are positive.
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