Our Platform Technologies
Each of our platforms is based on science in-licensed from world-leading researchers. These platforms have the potential to prevent, treat, and/or reverse multiple aging-related diseases, each with critical unmet medical needs.
Chaperone-Mediated Autophagy Platform
Chaperone-mediated autophagy (CMA) is a process by which unwanted proteins are degraded in cells. CMA activity declines during aging due to a reduced expression of a protein called LAMP2A, which leads to the accumulation of insoluble protein aggregates that disrupt cellular function.
We have identified novel small molecule oral compounds that increase the expression of multiple proteins in the CMA pathway, including LAMP2A, and thereby increase CMA activity. Our CMA activator compounds have been shown to have efficacy in preclinical models of frontotemporal dementia, Alzheimer’s disease, and aged hematopoietic stem cell dysfunction.
Mechanism of Action
Status:
Initiate IND-enabling program in 2022
Mitochondrial Uncoupler Platform
As we age, our metabolic rates decline. Mitochondrial uncouplers increase metabolic rates, decrease oxidative stress, and extend lifespan in preclinical species.
Earlier generations of mitochondrial uncouplers such as 2,4-Dinitrophenol (DNP) were effective at inducing weight loss but had unacceptable toxicity. We have developed next generation oral mitochondrial uncouplers that have mitigated these safety risks. Safety and efficacy demonstrate weight loss in obesity models and reduction of fatty liver in NASH models.
Mechanism of Action
Status:
Initiate Phase 1 study in 2022
Epigenetic Reprogramming PlatformThe epigenome consists of chemical modifications of DNA and DNA binding proteins that turn genes on and off. The epigenome drifts with age, leading to dysregulated gene expression.
Our epigenetic reprogramming platform reprograms the epigenome of older animals to resemble the epigenome of younger animals via expression of three Yamanaka factors [OCT4, SOX2, and KLF4 (OSK)]. Intravitreal injection of our OSK therapy increased nerve regeneration following crush injury of the optic nerve, restored vision in a mouse glaucoma model, and significantly improved vision in naturally aged mice. No adverse safety findings were seen in mice 15 months after systemic delivery of the OSK therapy.
Mechanism of Action
