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Precision BioSciences (DTIL) investor relations material

Precision BioSciences H. C. Wainwright Genetic Medicines Virtual Conference summary

Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.
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H. C. Wainwright Genetic Medicines Virtual Conference summary14 Oct, 2025

Conference overview

  • The event highlighted next-generation approaches in genetic medicine, featuring key opinion leaders and innovative biotechnology companies in gene editing, RNA medicines, and gene therapy.

  • Precision Biosciences presented its Arcus gene editing platform and its differentiated approach to treating Duchenne muscular dystrophy (DMD).

Arcus gene editing platform

  • Arcus is derived from homoendonucleases, not CRISPR, offering unique advantages such as staggered cuts, small size, and single-component simplicity.

  • The compact size enables delivery of two nucleases in one AAV, allowing efficient editing at two sites in the dystrophin gene.

  • This design supports broader applicability and higher efficacy compared to multi-AAV or single-exon approaches.

PVGene DMD program rationale and design

  • DMD was prioritized due to high unmet need and the potential for transformative impact.

  • The program targets the exon 45-55 hotspot, relevant to up to 60% of DMD patients, by excising the intervening DNA to restore near full-length dystrophin.

  • Arcus enables an all-in-one AAV approach, overcoming limitations of other gene editing and exon skipping strategies.

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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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