Genprex Inc. has released preliminary in vivo proof-of-concept data for GPX-002, the company’s diabetes gene therapy drug candidate, in a nonhuman primate (NHP) model of type 2 diabetes and in a mouse model of type 2 diabetes. GPX-002 is being developed for the potential long-term control of both type 1 and type 2 diabetes.

In a recent publication in Molecular Therapy, researchers from Drexel University College of Medicine and UMass Chan Medical School presented a silence-and-replace gene therapy strategy aiming to address both the gain-of-toxicity and loss-of-function components of the disease hereditary spastic paraplegia (HSP).

SYNGAP1-related disorders (SRDs) are rare neurodevelopmental conditions characterized by a wide range of symptoms, including intellectual disability, epilepsy, motor deficits and increased risk-taking behavior.