Dr. Ross Fredenburg to oversee a new initiative focused on identifying new therapies to correct mutant NPC1 protein folding and trafficking.

In 2022, the APMRF received a  $1 million gift from Steve and Maureen Klinsky , along with additional donations from the annual Parseghian Classic, to launch a new program focusing on identifying new therapies for Niemann Pick Type C patients.

On November 18th, a volunteer group of NPC scientists and clinicians, along with industry experts, gathered in Chicago for a special workshop to learn about protein folding and outlined optimal approaches to identify state of the art therapies.

The significant efforts needed to identify advanced therapies will rely on experts in developing assays, carrying out small molecule screens, and complex biology to determine the effectiveness.  Collaboration amongst NPC scientists and working with Contract Research Organizations to carry out the program will determine the overall impact.

After searching for an individual to assist in overseeing the new Klinsky and Sherry Initiative, we are pleased to announce Dr. Ross Fredenburg has agreed to partner with APMRF and NPC scientists on this new initiative. Fredenburg has over 15 years of experience driving preclinical drug development programs in biotechnology and pharmaceutical companies and was recently a founding member and Vice President of Biology of Amathus Therapeutics. His expertise in biochemistry and assay development is ideal for this effort and he has extensive experience identifying and managing external partners to accelerate preclinical research.