Insights into our Strategy : A Purposeful Approach to Disruptive Innovation

A Purposeful Approach to Disruptive Innovation   

Authored by Sumit Aggarwal

The fulfilling pursuit of therapeutic innovation for seriously debilitating or life-threatening diseases can be a long and challenging road, which is why it is important to continually provide updates as we work toward our ambition.

I had the pleasure of presenting the ZIkani’s TURBO-ZM strategy of rationally targeting human the ribosome– a powerful asset that is being leveraged to identify new candidates for a range of rare and nonsense mutation diseases as well as cancers at the virtual Summer Private Company Showcase, which was hosted by Solebury Trout, Piper Sandler and White & Case.

The TURBO-ZM platform allows a unique approach to drug discovery. Instead of searching for a needle in a haystack from large combinatorial chemical libraries to discover unique NMEs, we are taking well established macrocyclic chemistry, its known interaction with the ribosome and breaking known biological activities into distinct modules to enable directed drug discovery by using the TURBO-ZM platform chemistry. This allows the modules to be recombined in a manner that could not have been done in the past to achieve novel activities on the human ribosome while retaining those modules essential for favorable safety and PK, thus bringing three critical components to accelerate drug development.

With TURBO-ZM and our team’s rich expertise in ribosome science, we are poised to apply this rare thinking to deliver rare solutions and create a preeminent platform to develop multiple targeted small molecule ribosome modulators including read through agents to build an extensive pipeline in rare disease and oncology.

The forum this week was a valuable opportunity to highlight the momentum we are generating. During the presentation, I mentioned that we expect to have our lead candidate identified later this year, with an IND as early as the end of next year. You’re welcome to watch the replay of my brief presentation here and we’ll post more updates as we progress on our journey to deliver disruptive innovation with the power of the ribosome.

We are driven by a shared passion to support people around the world with rare diseases or nonsense mutation diseases who have few options today.  There are critically important solutions to these diseases that can be found within the ribosome and we are working persistently to uncover these insights and generate therapeutics that can improve lives.