A New Collaboration Will Support Research of Prader-Willi Syndrome and Other Rare Diseases

Vanderbilt University has just announced a new collaborative agreement with Soleno Therapeutics in aim to develop KATP channel activators as a therapeutic for rare diseases. This therapy has already been shown to provide benefit for patients with Prader-Willi syndrome (PWS) within a Phase 3 trial.

The Project

This project will be lead by Dr. Craig Lindley and Dr. Jerod Denton from the Warren Center of Neuroscience Drug Discovery.

They will be investigating the therapeutic potential for this therapy in various rare conditions.

Since KATP are present within several different kinds of tissues, they can be activated as a therapeutic option for many types of conditions such as PWS and hyperinsulinemic hypoglycemia.

The team hopes to develop both in vitro and in vivo tools with which to characterize KATP activators from different classes of chemistry. The ultimate goal is to advance these candidates to the clinic.

They feel as though the expertise they currently have will help to accelerate this process. The identification and characterization will be expedited, therefore leading to accelerated development of new activators. These activators should hopefully be both more potent, more specific, and safer than current options.

The world of research is constantly evolving, and when something is discovered that has the potential to help many conditions, our efforts should be two fold. The potential of this therapy to aid many rare diseases is exciting, and this collaborative effort should help to ensure research moves forward seamlessly.

Whenever we share knowledge and resources, we are more successful than we could be on our own. This success is vital for the patients who live day to day waiting for it.

You can read more about this collaboration and the research in this avenue here.

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