Principle Investigator: Chengwen Li, M.D./PhD

“Gene therapy with adeno-associated virus (AAV) vector has been successfully applied in numerous clinical trials with 6 AAV based drugs approved by the FDA recently. However, more than 50% of patients have antibodies against AAV and been excluded from this effective treatment. We have developed a biomolecule with high efficiency to block AAV antibodies in mice. In this study, we will study the efficacy and safety of this biomolecule in non-human primates. If successful, this biomolecule could be immediately transited into the clinical applications to benefit more patients receiving AAV gene therapy.”

Principle Investigator: Gerlinde Wernig, M.D.

“Pulmonary fibrosis (PF) and architectural remodeling of tissues disrupt lung function with often fatal consequences. Currently, there are no successful treatments for reversing lung fibrosis. Our previous research revealed effective anti-fibrotic therapeutic strategies to obtain reversion of interstitial lung disease in long COVID patients with PF. Our anti-fibrotic therapeutic strategy is to resolve lung fibrosis in our pre-clinical models at the transcriptional level with significant therapeutic efficacy and minimal or no toxicity. Our goal is to further our approach in non-human primate models for further evaluation of the toxicity and efficacy of our studies.”

Principle Investigator: Leah Byrne, PhD

“Oculocutaneous albinism (OCA) is a group of disorders that causes reduced pigmentation in the eyes, hair, and skin, resulting in severe visual impairment. Currently, there are no effective treatments for OCA vision loss, making it a significant unmet clinical need. Gene therapy is a promising approach for treating OCA, but we lack animal models with eyes similar to humans in which to develop and test these therapies. Recently, several adult rhesus macaques at CNPRC were identified as having features similar to humans with OCA, including reduced pigmentation in the hair and eye. In this proposal, we will screen NHPs at CNPRC to identify carriers of OCA-causing mutations. We will also perform preliminary testing of OCA gene therapy in primates. Overall, this research may lead to the development of a potential gene therapy treatment for OCA and improve the quality of life for those affected by this disorder.”