RARE Daily

LSU Health New Orleans and Takeda Collaborate to Advance Research for Rare Neurological Disorders

December 10, 2021

LSU Health New Orleans said it entered into an exclusive option and license agreement with Takeda Pharmaceutical that gives exclusive rights to develop a technology invented at the university with the aim of discovering therapeutics to treat rare neurological diseases caused by DNA repeat expansion.

Photo: Ed Grabczyk, associate professor of genetics at LSU Health New Orleans School of Medicine

In DNA repeat expansion disorders, a segment of repeated DNA expands within a gene to cause disease, and these repeats can number in the dozens or even hundreds. The repeated DNA sequence itself may continue to increase in size over time, which can impact the severity of the disorder, leading to earlier onset or more rapid disease progression. There are more than 30 progressive neurological disorders caused by DNA repeat expansion.

“Imagine that you have a ticking time bomb within your body that will go off in the future and destroy you,” explains Ed Grabczyk, associate professor of genetics at LSU Health New Orleans School of Medicine, who invented the technology. “That is essentially the situation faced by people who inherit a gene carrying a DNA repeat expansion. Our novel therapeutic approach targets a central mechanism that is shared by all repeat expansion diseases.”

Grabczyk’s research focuses on a novel therapeutic approach to DNA repeat expansion disorders. He received a grant from LSU’s proof-of-concept fund to validate his research and as a result of that work, received a Harrington Rare Disease Scholar Award from the Harrington Discovery Institute at University Hospitals in Cleveland, Ohio, which provided additional research funding and translational support.

In April 2018, Grabczyk and LSU Health New Orleans began a collaboration with Takeda as part of a broader Collaborative Grant Agreement with Harrington Discovery Institute. The exclusive option and license agreement will allow Takeda to develop further this important field of research.

Author: Rare Daily Staff

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