Generation Bio Co. is pioneering a new class of redosable in vivo genetic medicines through its innovative platforms: cell-targeted lipid nanoparticles (ctLNP) and immune-quiet DNA (iqDNA). The ctLNP delivery system is designed to selectively target specific tissues and cell types, enabling the delivery of genetic medicines directly within the body without the need for toxic preconditioning or ex vivo manipulation. This approach not only enhances the therapeutic reach but also allows for redosing, making treatments more accessible and adaptable to individual patient needs. The modular nature of the ctLNP system facilitates rapid identification of new targeting ligands, potentially expanding the range of treatable conditions.

The iqDNA platform complements the ctLNP technology by providing a durable and immune-quiet genetic cargo that can effectively replace or insert large genes. This design allows for sustained gene expression without triggering an immune response, which is crucial for long-term treatment efficacy. The ability to redose iqDNA means that therapies can be tailored over a patient's lifetime, addressing a variety of genetic disorders, including hematologic, oncological, and autoimmune diseases. Together, these platforms aim to revolutionize genetic medicine by offering scalable, cost-effective solutions that can reach a global patient population, ultimately transforming the landscape of treatment for both rare and prevalent diseases.

United States

Generation Bio Co., a genetic medicines company, develops gene therapies for the treatment of rare and prevalent diseases. The company is developing a portfolio of programs for rare and prevalent diseases of the liver and retina. It also focuses on the diseases of skeletal muscle, central nervous system, and oncology. The company was formerly known as Torus Therapeutics, Inc. and changed its name to Generation Bio Co. in November 2017. Generation Bio Co. was founded in 2016 and is headquartered in Cambridge, Massachusetts.

Generation Bio Co.

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