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PBGENE-LLY3 is an investigational gene editing therapy developed by Precision BioSciences for central nervous system (CNS) related disorders. It employs the ARCUS gene editing platform, which is based on proprietary meganucleases engineered to enable targeted gene modification. PBGENE-LLY3 is designed as a CNS-directed gene modulator, aiming to correct, modulate, or eliminate disease-driving genetic defects within the nervous system. As of 2025, development was discontinued at a preclinical phase. Details about the exact target gene or specific CNS disease indication have not been publicly disclosed.
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