What if we could
cure fatal rare
Rare Monogenic Disease
Many rare diseases are caused by a single mutation in a gene, which leads to a build up of a misfolded, non-functional protein. These proteins go on to do serious damage to the cell and therefore cause often fatal disease. And very often they inhibit the ability of the cell to undergo autophagy which itself leads to more damage.
So if we boost autophagy we can remove the misfolded, cytotoxic protein, restore the cell’s ability to undergo autophagy, and in some cases we find that we can also restore the function of the mutated protein. This means restoring autophagy is not just helping an aspect of the disease but is truly curative.
In Samsara we are studying specific rare genetic liver diseases where we believe autophagy can be completely curative.
This could be life-changing for many patients suffering with severe diseases which are currently poorly treated.
It’s a busy time for Samsara Therapeutics as we move into our new purpose-built labs and offices in the Wood Centre for Innovation in Oxford.
Our strategy in Samsara is to treat diseases where there is a high unmet need. But we also want to find out how our molecules work to affect healthspan and longevity.
Over the last twenty years or so, the phenomenon of autophagy has become a hot topic in the world of biology.