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GeNeuro Presents Precision Medicine Approach for ALS at Global Symposium

NextEdge Admin

07 Dec 2024

2 min 27 sec

GeNeuro announced significant progress in ALS treatment during the 35th International Symposium on ALS/MND in Montreal, Canada (Dec. 6-8, 2024). The findings, presented by Dr. Darshan Pandya of NIH/NINDS, spotlight GNK-301, a humanized monoclonal antibody targeting HERV-K ENV—a neurotoxic protein linked to neuronal death and blood-brain barrier (BBB) dysfunction in ALS.

Developed with NIH/NINDS, ERBC (France), and the University of Oxford, GNK-301 offers a dual diagnostic and therapeutic approach. In preclinical studies, GNK-301 detected HERV-K ENV in cerebrospinal fluid (CSF) and neutralized its toxic effects. In neuron cultures and mouse models, the antibody prevented neuronal death and restored BBB integrity, addressing critical ALS hallmarks.

Importantly, GNK-301 accumulated specifically in brain regions with HERV-K ENV, enhancing precision. It outperformed natural autoantibodies found in some ALS patients by offering higher affinity and stronger neutralizing effects. GNK-301 also leveraged BBB dysfunction caused by HERV-K ENV to access affected brain regions effectively.

The integrated approach includes an immunoassay to identify HERV-K ENV-positive ALS patients, followed by targeted treatment with GNK-301. This strategy holds promise to transform sporadic ALS management, offering a personalized therapy for patients with HERV-K ENV-associated pathology.

Medical-grade GNK-301 production is underway to support clinical trials. “These groundbreaking results lay the foundation for a new precision medicine paradigm in ALS treatment,” said Hervé Perron, GeNeuro’s CSO.

The findings reinforce the potential of targeting HERV-K ENV in ALS and highlight the importance of early detection and intervention. This innovative approach could significantly improve outcomes for ALS patients.