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Generation Bio

Founded: 2017

Location: Cambridge, MA

Employee range: 50 - 200

Clinical stage: Clin0

Therapy area: Hemophilia A

Drug types: GEN, HEM, OPH, GER, VAC, HEP, PED

Lead product: Undisclosed

Product link: https://generationbio.com/our-portfolio/

Funding: $230M IPO Jun 2020; $110M C Jan 2020; $100M B Feb 2018; $25M A Jan 2018

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Short description:

Gene Therapy

Drug notes:

7 undisclosed programs RD PKU, Wilson disease, Gaucher disease, Leber congenital amaurosis 10, Stargardt disease, wet AMD, ETAP

Long description:

Generation Bio is developing a new class of genetic medicines. Using their proprietary non-viral genetic medicine platform, Generation Bio is creating scalable and redosable medicines. The platform uses closed-ended DNA (ceDNA) encased in cell-targeted lipid nanoparticles. ceDNA has 3x the carrying capacity of AAV capsids and has the potential to offer durable expression. The medicines designed by Generation Bio are efficiently manufactured - ceDNA can be consistently produced with high purity using their cell-free rapid enzymatic synthesis approach. Generation Bio is building a portfolio initially targeting diseases of the liver and retina before expanding into skeletal muscle and the CNS.