JONESBORO, Ark. (KAIT) – Sylamore Bio, an Arkansas Delta biotech company, received a significant fundraising boost to aid its groundbreaking research to combat a rare and fatal neurodegenerative disease.
Their innovative approach aims to overcome long-term challenges and paves the way for treatments for a rare condition, and potentially other neurological disorders.
Sylamore Bio focuses on developing therapies for diseases affecting tissues that are difficult to treat, such as those in the central nervous system.
One of the diseases they are working to treat is GM1 gangliosidosis, a rare neurodegenerative disease that is fatal and primarily found in infants.
Dr. Walter Acosta, chief scientific officer with Sylamore Bio, and Dr. Tianhong Yang, both Arkansas State University alumni, were recently awarded a highly competitive $3 million Phase II Small Business Innovation Research grant from the National Institutes of Health’s National Institute of Neurological Disorders and Stroke.
The grant will allow them to accelerate their research to find a lifesaving therapeutic for GM1 gangliosidosis. There is currently no cure for the disease.
“This will be a lifesaving drug for these patient population,” Acosta said.
The reason there is no cure yet lies in a significant medical obstacle.
“The blood-brain barrier is formed by specialized cells in the vessels in your brain,” Acosta said. “So, basically, anything that isn’t in the blood stream, isn’t capable to reach the brain tissue because only specialized molecules can cross that blood-brain barrier.”
The company plans to address this through their SyLEC platform, which is a delivery system that aims to cross the blood-brain barrier.
“That is specifically designed to safely and effectively transport therapeutic agents across the blood brain barrier, allowing us to deliver a potential drug directly to the affected cells in the central nervous system,” Acosta said.
The grant will fund crucial development phases.
“With this, we will be picking our lead candidate, optimizing the drug so we can move to patients in the next five years,” Acosta said. “It is basically to fund the preclinical testing of this drug.”
If the delivery system drug works as intended, it could be used to treat other diseases.
“That can be translated not just to rare pediatric conditions, but also any neurological disorder that requires treatment, such as Alzheimer’s, Parkinson’s, or dementia,” Acosta said.
Dr. Acosta added this grant marks a critical step towards developing a much-needed treatment for devastating neurological conditions.
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