Early 2024, Pranam Chatterjee, assistant professor of biomedical engineering at Duke University, received a donation of $50,000 from the foundation EndAxD.

With the support from EndAxD, Chatterjee plans to develop a platform that can target and degrade the mutated GFAP. Working at the intersection of generative artificial intelligence (AI) and experimental bioengineering, Chatterjee and his team have recently developed AI algorithms that can design binding peptides to previously undruggable target proteins.

For example, his lab’s most recent algorithm, PepMLM, generates binding peptides that can precisely and accurately bind using only a small target protein sequence. This work has allowed them to create new therapeutic tools, called “ubiquibodies,” that can bind to and degrade these target proteins.

“Our goal for the first year of the project is to identify, design and optimize ubiquibodies that can specifically and efficiently degrade GFAP proteins. It’s a hard protein to target, so if we can prove that our approach will work, that would be a significant step forward. GFAP proteins are a potential target in a lot of neurodegenerative diseases, so there is a lot of motivation for us to figure out how we can break it down.“ - Pranam Chatterjee, Assistant Professor of Biomedical Engineering

Chatterjee and his team are looking forward to using their unique combination of computational and wet-lab tools to develop potential new therapies for this devastating disease.

“Not only is EndAxD giving us financial support to study Alexander disease, but they are also giving us access to specific cell lines, animal models and a community of researchers that will help us go after this disease,” said Chatterjee. “I’m grateful to EndAxD for providing us with the support to take this on.”

“We are grateful to Duke University, Dr. Chatterjee, and his team for the opportunity to partner on this groundbreaking research” said Laura Ledbetter, co-founder of End AxD. “ Our hope is this investment will bring us closer to a treatment and eventual cure of Alexander disease”.

Since then, Dr Chatterjee has received an NIH grant to further his research and made public computational results for GFAP binders.