3DNA Precision Medicine Platform

3DNA is a nanoscale, multivalent scaffold made from proprietary, synthetic DNA formed in a flexible, branched structure. This unique nanocarrier allows for precise, targeted delivery of therapeutic cargo to intracellular targets in a rapidly scalable, cost-effective manner. The 3DNA nanocarriers are engineered and cross-linked to form a stable architecture while maintaining the biocompatibility of the nucleic acid building blocks. The modular design of 3DNA enables tunability to optimize the ratio of targeting agents and therapeutic cargo, which improves precision of targeting and uptake of the payload without creating immunogenic effects.

3DNA Precision Medicines for the Treatment of Rare Diseases

Microglia are the brain’s resident immune cells. They regulate many processes in the brain that are crucial for tissue development, maintenance of the neural environment, response to injury and promotion of repair. Microglia function and, more specifically, regulation of key genes and pathways have been linked to multiple neurodegenerative disorders. Therefore, modulating these genes could offer important options for addressing many elusive neurodegenerative diseases. Recently published nonclinical in vivo results in Brain using a model of preterm brain injury demonstrate a novel 3DNA therapy from Genisphere that targets microglia with a Wnt/β-catenin pathway agonist preventing pro-inflammatory microglia activation, reducing damage to the brain, and providing short- and long-term behavioral benefits. Genisphere is advancing a discovery program in microglia-targeted 3DNA therapeutics for rare neuroinflammatory diseases such as Niemann-Pick disease Type C1 and Amyotrophic Lateral Sclerosis.

Maximizing the value of the 3DNA Precision Medicine Platform via Partnerships

Genisphere and its collaborators in both academia and industry have demonstrated efficacy and safety utilizing the 3DNA platform to deliver a variety of targeted drug cargos including plasmid DNA and siRNA across multiple indications in nonclinical models. Data generated from studies in ovarian cancer, pancreatic cancer, colon cancer, and rheumatoid arthritis demonstrate high biological response at low doses of cargo, the ability to deliver siRNA-targeted therapies beyond the liver, and significant improvements in the delivery of whole gene therapy, multivalent antibodies, small molecules and vaccines. Genisphere will advance the progress of the 3DNA Precision Medicine Platform in these areas via external partnerships, generating non-dilutive funding that will support the development of its internal rare disease pipeline.