Researchers at UNC Chapel Hill Find New Potential Treatment for AS

Ben Philpot, PhD and his lab at UNC Chapel Hill have identified a small molecule that could be a potential gene therapy treatment for AS.

The ASF first funded Dr. Philpot’s pilot study to partner with Pfizer to evaluate over 2,800 small molecules to determine if any could potently turn on paternal UBE3A in mice.

According to a UNC press release, (S)-PHA533533, a small molecule compound, was found to have clinical potential. It was also determined that (S)-PHA533533 also “has excellent bioavailability in the developing brain, meaning it travels to its target with ease and sticks around.”

The next step will be to refine the chemistry of the compound to ensure it would be safe and effective for a clinical setting.

Today, the findings of the research have been published in Nature.