Stoke Therapeutics Inc (NASDAQ:STOK) recently announced the publishing of data on its innovative method of treating severe genetic health conditions through upregulating protein expression.
The company revealed that the data on the innovative approach to genetic illnesses was recently published in Nature Communications. The data published in the journal supports Stokes Therapeutics’ innovative and proprietary mechanism that can be used to accurately regulate protein expression through Targeted Augmentation of Nuclear Gene Output (TANGO) antisense oligonucleotides (ASOs).
“Stoke was founded on the idea that we could use unique insights in RNA biology to do something that has never been done before,” stated Stokes Therapeutics Vice President and co-founder, Isabel Aznarez.
Aznarez noted that the approach that Stoke Therapeutics is using is different from the traditional gene therapy approaches. It focuses on increasing protein output from healthy cells rather than editing or replacing faulty genes. She also noted that preliminary data shows that it is possible to boost protein expression to full capacity from different healthy genes. The company hopes that this approach will pave the way to an entirely new way of addressing severe genetic conditions.
Stoke Therapeutics picked four gene targets that will allow researchers to dive deeper into the mechanisms involved. The gene targets are of different types and are rich in protein function, non-productive splicing events, and gene size. Stoke Therapeutics hopes that the approach will help address more genetic diseases.
So far, scientists have discovered that a single gene mutation can cause over 10,000 genetic diseases. Existing gene therapy approaches only cover about 5% of all the genetic diseases. This means that there is still a lot to be done addressing genetic illnesses, and it might call for investigating innovative new approaches.
Stoke Therapeutics revealed in the recently published data that researchers analyzed RNA sequencing datasets using proprietary bioinformatics. They discovered that a multitude of non-productive alternative-splicing events causes mRNA degradation, which leads to reduced protein production. These findings will play an important role in developing therapies that promote more protein production in cells, thus preventing or curing genetic illnesses.