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Precision BioSciences
Precision BioSciences (DTIL) investor relations material
Precision BioSciences Guggenheim Securities Emerging Outlook: Biotech Summit 2026 summary
Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.Guggenheim Securities Emerging Outlook: Biotech Summit 2026 summary11 Feb, 2026
Company overview and technology focus
Specializes in in vivo gene editing using proprietary ARCUS technology, with 20 years of expertise and a focus on two main programs: chronic hepatitis B and Duchenne muscular dystrophy.
ARCUS enables precise gene edits, targeting diseases with significant unmet needs and offering solutions where other technologies face limitations.
Duchenne muscular dystrophy (DMD) program
PBGENE-DMD uses ARCUS to excise exons 45-55, producing a near full-length dystrophin protein, aiming for broader applicability and improved function over micro-dystrophin therapies.
Preclinical data show 10-15% dystrophin in heart and up to 25% in skeletal muscle, exceeding the 5% threshold linked to clinical benefit.
The IND for PBGENE-DMD was recently cleared, with plans to dose ambulatory patients in a single-dose, non-escalating phase I trial focused on safety.
Manufacturing emphasizes high full-to-empty capsid ratios (>85%) to enhance safety and tolerability, with robust oversight of CDMO partners.
Aggressive immunosuppression and comprehensive supportive care are planned to maximize safety in pediatric patients.
Clinical trial timelines and data expectations
Initial DMD trial will treat 3-5 patients in 2024, with early safety and dystrophin expression data expected by year-end.
Early biopsies will provide dystrophin data, with staggered dosing to ensure patient safety.
DMD: Functional benefit of 80% dystrophin?
DMD: How lower AAV dose impacts safety?
HBV: How does dose intensification impact cccDNA?
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Next Precision BioSciences earnings date
Q4 202527 Mar, 2026
Precision BioSciences
Next Precision BioSciences earnings date
Q4 202527 Mar, 2026
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Frequently asked questions
Precision BioSciences Inc. is a U.S.-based biotechnology company focused on developing gene editing technologies. The company utilizes its proprietary ARCUS genome editing platform to research and develop treatments for genetic disorders and cancers. Its work spans cell and gene therapies, with applications in both in vivo and allogeneic CAR T-cell therapies. Precision BioSciences collaborates with academic institutions and pharmaceutical companies to advance its research and clinical programs. The company is headquartered in Durham, North Carolina, and its shares are listed on the NASDAQ.
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