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,” Kopin said in an ALS Network press release.
Faster route to treatments for ALS
In ALS, certain proteins tend to form toxic clumps inside neurons and contribute to cell damage. A hallmark protein that’s dysfunctional in about 97% of all in ALS patients is TDP-43, which helps RNA inside the nucleus of cells to be appropriately processed.
When TDP-43 builds up outside the nucleus, it cannot perform its normal function. As a result, some RNA molecules that require TDP-43 to be properly matured become abnormal, leading to the production of abnormal protein or no functional protein at all.
Stathmin-2, a protein that’s essential for nerve cell growth and regeneration, is one of the proteins whose levels are reduced due to abnormal processing of RNA by TDP-43. Another protein important for nerve cell function, UNC13A, is also reduced, and researchers have shown that several other proteins are affected by the loss of TDP-43 function.
Kopin, Draper, and other researchers have discovered antisense oligonucleotides (ASOs), short RNA molecules that can bind to processed RNA molecules, that could rescue the abnormal processing and lead to the production of functional proteins. The small molecules Auttx intends to screen are those that could trigger the same repairing mechanism as ASOs, providing a faster route to new therapies for ALS.
The project was selected by an expert scientific advisory committee from the ALS Network, which funds projects with the potential to make significant advances in ALS treatment.
“The ALS Network’s research-funding model is an ambitious program driven by urgency and innovation,” said Sheri Strahl, ALS Network’s president and CEO. “We seek to propel promising science forward quickly with a relentless focus on improving and saving lives. Auttx’s project will move us closer to the discovery of new therapies for ALS and other motor neuron diseases.”
About the Author
