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Passage Bio was launched with the vision to be a first in class fully-integrated biotech company developing life transforming AAV-delivered in vivo therapeutics for the treatment of rare CNS diseases. In collaboration with the University of Pennsylvania Gene Therapy Program, Philadelphia-based Passage Bio plans to advance a portfolio of 5 rare disease indications in the neuro/CNS genetic space through IND-enabling studies using best-in-class AAV technology and know-how. Subsequently, Passage Bio will be responsible for all clinical development and will collaborate with Penn`s Orphan Disease Center to support these efforts.
Generation Bio is innovating genetic medicines to provide durable, redosable treatments for people living with rare and prevalent diseases. The company`s non-viral platform incorporates a novel DNA construct called closed-ended DNA, or ceDNA; a unique cell-targeted lipid nanoparticle delivery system, or ctLNP; and a highly scalable capsid-free manufacturing process that uses its proprietary cell-free rapid enzymatic synthesis, or RES, to produce ceDNA. The platform is designed to enable multi-year durability from a single dose, to deliver large genetic payloads, including multiple genes, to specific tissues, and to allow titration and redosing to adjust or extend expression levels in each patient. RES has the potential to expand Generation Bio`s manufacturing scale to hundreds of millions of doses to support their mission to extend the reach of genetic medicine to more people, living with more diseases, around the world.
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