July 14, 2025 | Jon Kelvey - A team of researchers from the University of Maryland School of Medicine (UMSOM) recently won a $250,000 prize from the National Institutes of Health (NIH) to improve technologies for delivering genome editing tools to targeted cells in the body. They were one of four winners in a competition after successfully demonstrating a new technique to deliver genome editing biotechnology across the blood-brain barrier to brain cells in preclinical models.
The team included Pavlos Anastasiadis, PhD, Alexandros Poulopoulos, PhD, Jung Soo Suk, PhD, and Graeme Woodworth, MD, who called themselves the Crisaptics Trans-BBB Genome Editing Team. They used a combination of engineered nanoparticles, microbubbles, and focused ultrasound to open the blood-brain barrier and deliver advanced CRISPR agents to edit genes in narrowly targeted clusters of brain cells in an in vivo animal model.
The work earned them a top winning spot and the monetary prize from the NIH Targeted Genome Editor Delivery (TARGETED) Challenge, which the team plans to use to further their ongoing research into their technologies.
“All of us on the team were thrilled and honored to be among the challenge winners taking this critical step on the path to develop genome therapeutics for the brain,” said Dr. Poulopoulos, an Associate Professor of Pharmacology and Physiology. “We’re excited to continue developing this technology, which could one day help treat diseases that have proven extremely difficult to manage, including Huntington’s disease, genetic epilepsies, and glioblastoma.”
The NIH launched the TARGETED Challenge in 2023 to foster innovation in delivering genome editing tools and the delivery of those tools across the blood-brain barrier to target central nervous system tissues. Developing the right genome editing tools with the precision to target tissues could allow researchers to cure rare genetic disorders by editing the malformed genes that cause them. For example, researchers like Dr. Poulolpoulos are trying to edit an error in the IT15 gene in neurons in the brain’s basal ganglia, which could alleviate the progressive cognitive and motor dysfunctions of Huntington’s disease.
Taking place in three phases, the TARGETED Challenge offers a total of $6 million in prizes awarded to a narrowing field of competitors. The Crisaptics Trans-BBB team was one of four teams that each won a $250,000 prize in Phase 2 of the challenge. In Phase 3, the NIH will select six teams and test their cross-blood-brain barrier delivery and editing technologies in large animal preclinical studies before awarding a final winning team a $650,000 prize.
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