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We are a genome editing company developing genetic medicines for the fight against rare pediatric diseases. GeneRide™ is a promoterless and nuclease-free approach to gene insertion which may improve the safety profile of AAV based gene therapies With GeneRide™, the therapeutic gene is site-specifically integrated into the genome allowing durable gene expression in dividing cells and mature tissues. Furthermore, therapeutic gene expression is regulated by the targeted locus so production can be limited to specific cell types.
Equillium is a clinical-stage biotechnology company leveraging deep understanding of immunobiology to pioneer new products with a mission to dramatically improve the lives of patients with severe autoimmune and inflammatory diseases who continue to suffer with high unmet medical needs.
Exonics Therapeutics was launched in February 2017 to advance the research of our scientific founder, Dr. Eric Olson and his laboratory at the University of Texas Southwestern Medical Center (UTSW) to develop treatments for patients with neuromuscular diseases. Dr. Olson is one of the world’s leading experts in the study of muscle cells and the application of gene editing to treat these types of diseases. In particular, Dr. Olson’s laboratory has used adeno-associated virus (AAV) to deliver a CRISPR/Cas9 technology that can identify and repair exon mutations to restore the production of dystrophin, a protein that helps stabilize and protect muscle fibers. Dystrophin is the protein missing in boys with Duchenne. The loss of dystrophin causes Duchenne.
Selecta Biosciences, Inc. is a clinical-stage biopharmaceutical company that is seeking to unlock the full potential of biologic therapies by avoiding unwanted immune responses. Selecta`s tolerogenic Synthetic Vaccine Particles (SVP™) technology platform is designed to enable a range of novel biologics for rare and serious diseases that require new treatment options. The company`s current proprietary pipeline includes SVP-enabled enzyme, oncology and gene therapies. SEL-212, the company`s lead candidate in Phase 2, is being developed to treat severe gout patients and resolve their debilitating symptoms, including flares and gouty arthritis. Selecta`s clinical oncology candidate, LMB-100, is in a Phase 1 program targeting pancreatic cancer and mesothelioma. Its two proprietary gene therapy product candidates are being developed for rare inborn errors of metabolism and have the potential to enable repeat administration. The use of SVP is also being explored in the development of vaccines and treatments for allergies and autoimmune diseases. Selecta is based in Watertown, Massachusetts.
Puerto Rico Labor and Human Resources is a New York, NY-based company in the Healthcare, Pharmaceuticals, and Biotech sector.