BelSU Engineering the Next Generation of Brain‑Saving Gene Medicines

Scientists at Belgorod National Research University (BelSU) have built a validated experimental platform that brings gene therapies for stroke, traumatic brain injury and neurodegenerative diseases closer to the clinic.

Led by Associate Professor Olesya Shcheblykina of the Department of Pharmacology and Clinical Pharmacology, the team has already achieved three key milestones: a reproducible animal model of severe haemorrhagic stroke that mirrors human pathology, a patented technique for delivering drugs into the cisterna magna of small rodents, and a laboratory‑scale adeno‑associated viral (AAV) vector construct ready for preclinical testing. The AAV vectors carry a gene that instructs brain cells to produce neurotrophic factors – proteins that safeguard neurons and stimulate repair.

“We have a reliable platform that causes a severe haemorrhagic stroke very similar to the clinical picture in humans. It lets us objectively measure how well a future gene therapy helps the brain recover, using a standardised battery of behavioural tests to track neurological deficits over time,” Shcheblykina explains.

The long‑term project, scheduled through 2033, is run in partnership with Genotarget LLC (Artgen Group). The company designs and optimises the gene‑therapy constructs, while the BelSU team evaluates their safety and efficacy in the validated models. Together they are building a bridge from fundamental research to pharmaceutical development and, ultimately, clinical trials.

With the platform now established, the next phase moves to full‑scale preclinical studies of specific AAV‑based constructs. Researchers will refine dosing, confirm safety, and assemble a data package suitable for a potential transition into human trials.