Drishti Discoveries

Autosomal dominant diseases are caused by heterozygous mutations that are either dominant negative or gain of function. Most are currently untreatable, largely due to technical difficulties in removing the dominant effects of the mutation while restoring the normal functions lost due to the mutation.

A solution to this challenge is the removal of faulty protein by modulating gene expression from the mutated gene copy. Drishti Discoveries is utilising an shRNA- based gene modulation technology that is highly efficient and specific in knocking down gene expression. Using the vectorised shRNA sequences embedded into a proprietary miRNA scaffold, Drishti will knockdown the expression of the faulty gene and simultaneously replace its function by introducing an optimised exogenous functional copy of the gene (described as ‘silence and replace’).

The technology can be also used to knockdown multiple genes at once to achieve a more complete silencing of a pathway. This mutation-independent approach to knockdown target gene expression has the potential to achieve ‘one and done’ outcomes for the treatment of autosomal dominant diseases. Their first therapeutic targets is Amyotrophic Lateral Sclerosis (ALS) followed by a pipeline of additional autosomal dominant disorders.