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Precision BioSciences (DTIL) investor relations material
Precision BioSciences Chardan’s 9th Annual Genetic Medicines Conference summary
Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.Advances in genome editing technologies
Recent years have seen significant progress in protein engineering, enabling the development of non-CRISPR nucleases and optimized DNA-modifying enzymes for broader genome editing applications.
New gene editing platforms, such as Arcus and engineered recombinases, offer alternatives to CRISPR, with unique features for precise DNA recognition and cutting.
These technologies are being leveraged for gene insertion, large genomic changes, and targeting viral genomes.
Applications and therapeutic strategies
Gene insertion enables correction of diseases with multiple mutations by allowing large DNA fragments to be integrated at specific genomic sites.
Safe harbor sites, such as PCSK9, are used for targeted gene insertion, minimizing risk and enabling robust, durable expression of therapeutic genes.
Multi-gene insertions are being explored for complex diseases and advanced CAR T therapies.
Gene insertion is particularly suited for pediatric indications and rapidly dividing tissues, ensuring long-term therapeutic effect.
Technology optimization and delivery
Product profiles are shaped by optimizing delivery modalities, nuclease/recombinase choice, insertion site, and construct design.
Approaches include using AAV or LNPs for delivery, with strategies for repeat dosing and decoupling donor DNA from editing enzymes.
Arcus nucleases enable high-efficiency gene insertion in both dividing and non-dividing cells, with the ability to insert and remove DNA in a single step.
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