Acuitas CSO on CRISPR Delivery Breakthrough and Innovations in Lipid Nanoparticles

07/07/2025 36 min
Acuitas CSO on CRISPR Delivery Breakthrough and Innovations in Lipid Nanoparticles

Listen "Acuitas CSO on CRISPR Delivery Breakthrough and Innovations in Lipid Nanoparticles"

Episode Synopsis

Ying Tam, Chief Scientific Officer at Acuitas Therapeutics, discusses the company's role in delivering the first personalized CRISPR gene editing treatment to an infant with CPS-1, achieving treatment in just six months from diagnosis. Tam shares insights on evolving lipid nanoparticle technology beyond COVID vaccines, including LNPs that allow for next-generation immune-targeting delivery that is 10x more potent, and progress in delivering genetic medicines to stem cells and solid tumors. He also addresses the challenges of repeat dosing, a new biodegradable LNP formulation, and expanding genetic medicine beyond the liver to treat blood disorders and cancer.

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