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Generation Bio is innovating genetic medicines to provide durable, redosable treatments for people living with rare and prevalent diseases. The company`s non-viral platform incorporates a novel DNA construct called closed-ended DNA, or ceDNA; a unique cell-targeted lipid nanoparticle delivery system, or ctLNP; and a highly scalable capsid-free manufacturing process that uses its proprietary cell-free rapid enzymatic synthesis, or RES, to produce ceDNA. The platform is designed to enable multi-year durability from a single dose, to deliver large genetic payloads, including multiple genes, to specific tissues, and to allow titration and redosing to adjust or extend expression levels in each patient. RES has the potential to expand Generation Bio`s manufacturing scale to hundreds of millions of doses to support their mission to extend the reach of genetic medicine to more people, living with more diseases, around the world.
Our genetically-engineered, universal iPSCs-derived immune effector cell products (NK, T, Dendritic cells, and macrophage) are designed to specifically target hematologic and solid tumor cancers. Our commitment to developing off-the-shelf cell therapies will expand patient access and provides an unparalleled opportunity to advance the course of cancer care.
Proginitor Cell Therapy is a Mountain View, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Insmed is a global biopharmaceutical company focused on the needs of patients with rare diseases. Headquartered in Bridgewater, NJ, Insmed is a dynamic and rapidly growing company with a strong pipeline of drug candidates. At Insmed, we are on a mission to transform the lives of patients battling serious rare diseases. We are developing novel, targeted therapies to help serve the critical unmet needs of these patients. In concert with developing therapeutic solutions, we are committed to improving the entire patient experience. “Insmed is a team of talented and motivated professionals who are dedicated to making a difference in the lives of patients with rare diseases, said Will Lewis, president and CEO at Insmed.
Nohla Therapeutics is a cellular therapy company driven to provide leading off the shelf therapies for patients with critical diseases. Nohla’s first product is a universal donor cell therapy targeting hematopoietic recovery and chemotherapy induced neutropenia, and is being extended to treat a range of other medical indications. This and other products in the pipeline are based on a unique umbilical cord blood expansion platform developed at the Fred Hutchinson Cancer Research Center