"We are building a leading lentiviral-based cell and gene therapy company using our versatile dual technology platform and in-house manufacturing capabilities to advance development of potentially transformative therapies for patients and families seeking relief from devastating genetic diseases."
Matthew Gantz, President and Chief Executive Officer
Courtesy of debra—The Dystrophic Epidermolysis Bullosa Research Association of America and the Oliveira family. Used with permission.
Courtesy of the Pratt family. Used with permission
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 MoreFunding expected to provide sufficient capital for completion of Phase 3 trial for ex vivo product candidate for recessive dystrophic epidermolysis bullosa
Read MoreCompany adds in vivo capabilities to existing ex vivo approach for development of novel gene therapies
Read MoreRDEB is a devastating, rare genetic blistering disease with no FDA-approved treatments currently available
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