ALS News Today: Auttx gets ALS Network grant to develop new treatments for ALS

Biotech company aims to develop molecules that restore RNA processing

Biotech company Auttx has received a $125,000 grant to advance the development of new molecules that aim to restore normal RNA processing in people with amyotrophic lateral sclerosis (ALS), potentially leading to new treatments for ALS.

Messenger RNAs are intermediate molecules that are produced when genes are read to serve as templates for protein production. Many of these molecules are abnormally processed and matured in the presence of the abnormal TDP-43 protein clumps that mark ALS, which further contributes to disease progression.

The grant, from the ALS Network, will allow Auttx to screen a library of small molecules to expedite promising compounds that may reverse abnormal RNA processing.

The research will be led by Auttx’s co-founders Isabelle Draper, PhD, who serves as the company’s chief scientific officer, and Alan S. Kopin, MD, its CEO. Draper leads a laboratory at Tufts Medical Center and is focused on studying alterations in RNA processing in animal models. Kopin, a professor emeritus at Tufts University School of Medicine, has been involved in research examining the abnormal processing of the Stathmin-2 protein due to TDP-43 dysfunction.

“Our work is critical in helping expedite the development of targeted drug therapies that can reverse abnormal molecule processing,” Dr. Kopin said in an ALS Network press release.