Ikshana Therapeutics is developing breakthrough treatments for age-related diseases, starting with a focus on restoring vision for people affected by dry age-related macular degeneration (AMD), a leading cause of vision loss in older adults with no effective cure.
Our innovative approach works by reactivating your body's natural cellular cleaning and renewal processes. Think of it as awakening your cells' built-in repair system that naturally declines with age. By restoring this vital renewal process, we aim to help your body heal itself.
Our groundbreaking science could improve the lives of millions of people affected by age-related conditions. We're beginning with vision restoration, but our approach has the potential to address multiple challenges of aging, offering hope for conditions that currently have limited treatment options.
Product: Cryba1 (encodes for βA3/A1-crystallin) gene therapy, which is an epigenetic regulator
Strong efficacy demonstrated in multiple mouse models of dry AMD and iPS cells with the complement factor H variant
The drug exhibits robust safety in both young and old monkeys based on comprehensive non-human primate data
Regulating the mTORC1 (mechanistic target of rapamycin, complex 1) signaling axis in lysosomes by activating βA3/A1-crystallin
Targeting the mTORC1 pathway prolongs the lifespan in model organisms (National Institute of Aging study).
Direct inhibition by rapamycin or new generation rapalogs causes intolerable side effects. Human clinical trials, including AMD, have failed.
βA3/A1-crystallin is a nascent protein found in the lysosomal lumen and nucleus of RPE cells, and is an epigenetic modulator in the RPE cells as well as V-ATPase and mTORC1 activity
Tested for efficacy in mice and safety in non-human primates
The solution is a gene therapy of Cryba1 (encodes for bA3/A1-crystallin)
Strong efficacy was demonstrated in multiple mouse models of dry AMD and human stem cells with the complement factor H variant
Shows safety in young and old non-human primates
Targeted Patient Population
Patients suffering from debilitating dry AMD
At an early stage
And late stage
