August 30, 2022 | ~Home Feature| Press Release
The cell and gene therapy company’s lead in vivo candidate uses lentiviral vector delivery to correct metabolic disorder in large animal model of hereditary tyrosinemia type-1
Read MoreMay 25, 2022 | ~Home Feature| Press Release
Funding expected to provide sufficient capital for completion of Phase 3 trial for ex vivo product candidate for recessive dystrophic epidermolysis bullosa
Read MoreJanuary 10, 2022 | ~Home Feature| Press Release
Company adds in vivo capabilities to existing ex vivo approach for development of novel gene therapies
Read MoreOctober 21, 2021 | ~Home Feature| Press Release
RDEB is a devastating, rare genetic blistering disease with no FDA-approved treatments currently available
Read MoreOctober 14, 2021 | Media Coverage
FDA Awards 11 Grants to Clinical Trials to Develop New Medical Products for Rare Disease Treatments
Read MoreSeptember 29, 2021 | Press Release
Research to focus on osteogenesis imperfecta and classical Ehlers-Danlos syndrome, debilitating disorders with no FDA-approved treatments
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