Genisphere Pursues Neurotherapeutics Under Collaboration Agreement With Inserm

Genisphere LLC has signed a collaborative research agreement with The Institut national de la santé et de la recherche médicale (Inserm, The French National Institute of Health & Medical Research) to study targeted nanotherapeutics for neurological disease. Inserm scientists Dr. Pierre Gressens and Dr. Bobbi Fleiss will explore a variety of therapeutic molecules with the 3DNA drug delivery platform provided by Genisphere.

The research project aims to understand the mechanisms of brain development under normal and pathological conditions, in order to decrease poor neurological outcomes associated with abnormal development or injury to the developing brain. Neuroprotective and therapeutic strategies will be evaluated by using the 3DNA nanocarrier to deliver various drug molecules, siRNA, microRNA, peptides or proteins to brain cells in vivo.

Dr. Bobbi Fleiss explained, “We have tested a variety of strategies for delivery of therapeutics to the brain with limited success, but 3DNA has shown surprising promise and that it can target and penetrate specific cell types in the brain. Further, two unique payloads, a peptide and a microRNA, have both reduced neuroinflammation in our in vivo models of preterm brain injury.” Dr. Pierre Gressens commented, “Our goal is to hit multiple points of pathological pathways to treat genetic and environmental disorders of the developing brain.”

Bob Getts, Genisphere’s Chief Science Officer, said “Targeting the brain is a high priority for Genisphere. Bobbi and Pierre are true experts in the field of perinatal neuroscience. Now that our 4-year relationship has led to this agreement, we predict future work will provide real advances in neurodegenerative indications such as cerebral palsy and multiple sclerosis among others.”

Genisphere LLC is the provider of the 3DNA platform for targeted drug delivery. 3DNA is a nanoscale, multivalent scaffold made from proprietary, synthetic DNA formed in a flexible, branched structure. 3DNA nanocarriers are engineered and cross-linked to form a stable architecture while maintaining the biocompatibility of the nucleic-acid building blocks, and demonstrate efficacy and safety with a variety of drug cargos across multiple indications. Genisphere’s technology is IP-protected and fully customizable to deliver small molecules, biologics, and nucleic acids with precise specificity enabled by multivalent targeting via antibodies, peptides and other molecular entities. Genisphere leverages a collaborative model to advance its 3DNA drug delivery platform, and seeks additional partnerships with biotechnology and pharmaceutical companies to improve efficacy and reduce toxicity. Genisphere is also advancing its own lead compounds based on 3DNA nanotechnology. For more information, visit http://genisphere.com.