Innovation in Motion: GRIN2B Patent Marks Key Advancement

Professor Shai Berlin, a lead scientist with the 2BCured research team, announced the issuance of a patent for a novel gene therapy aimed at treating GRIN2B-Related Neurodevelopmental Disorder.

The patent involves use of a recombinant adeno-associated virus (AAV) vector capable of delivering a full-length, healthy GRIN2B gene across the blood-brain barrier to restore proper NMDA receptor function in neurons. 

According to Dr. Tali Garin-Shkolnik, Research Coordinator at 2BCured, this achievement marks the first time the complete GRIN2B gene has been successfully packaged into an AAV and delivered to the brain in preclinical models. Until now, GRIN2B was thought to be too large to fit inside the AAV typically used for gene therapy—making this a significant breakthrough.

“Early results show successful gene expression in neurons following intravenous administration,” said Dr. Garin-Shkolnik. “Importantly, ongoing safety assessments in animal models and brain organoids have shown no evidence of toxicity or neuronal death.”

This patent marks an important advancement in preclinical development. The team notes that additional work remains—including expanded safety testing, refining dosage, and scaling up manufacturing. Collaborative partnerships with biotech firms and support from the patient community will be essential in advancing this research toward future therapeutic use.

Prof. Berlin added: “While this is just one step in the journey toward a viable treatment, it is a meaningful one. We are grateful for the support from families, clinicians, and our research network.”

Key highlights from the patent include:

• Delivery via AAV vectors specifically engineered to cross the blood-brain barrier.
• Neuron-specific promoter usage for targeted gene expression.
• Application in treating a spectrum of NMDA receptor-related conditions beyond GRIN2B, including GRIN1, GRIN2A, and GRIN2D-associated disorders.

The full patent can be found here.

For more information contact us at info@2bcured.org.