Aptagen will develop delivery aptabodies, which the institute will use to investigate gene therapies.

The ALS Therapy Development Institute (ALS TDI) signed an R&D agreement with Aptagen for drug delivery technology to boost discovery of new ALS therapeutics.

Aptagen will develop aptabodies designed to deliver a therapeutic payload to diseased cells. This technology will be used to expedite drug development in a leading mouse model of ALS.

Through its collaboration with the Muscular Dystrophy Association’s Augie’s Quest ALS initiative, the Institute will apply the aptabody technology in investigating the potential of gene therapy for treating ALS.

“The use of aptabody conjugates to target a therapeutic to a specific cell type is a powerful tool that removes some of the limitations of drug delivery that utilize engineered viruses,” according to Steven Perrin, Ph.D., CSO at ALS TDI. “Aptabody drug delivery may reduce some of the inefficient trial and error of developing small molecules that must cross the blood brain barrier to reach their respective targets in the central nervous system.”

An aptabody is comprised of a piece of RNA that acts as a molecular scaffold for attaching drug compounds. Because development of aptabody-based drugs does not require prior knowledge of a disease target, the technology reportedly can reduce the time and cost of drug development.

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