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Stoke Therapeutics is pioneering a new way to treat the underlying causes of severe #geneticdiseases by precisely upregulating protein expression. We develop antisense oligonucleotide medicines that increase gene expression to treat genetic epilepsies and other severe monogenic diseases, including genetic conditions affecting the central nervous system, eye, liver and kidney. At Stoke, we believe our proprietary technology platform, Targeted Augmentation of Nuclear Gene Output, or #TANGO, will allow us to deliver disease-modifying therapies in a highly precise, durable and controlled manner. We have identified approximately 2,900 genetic diseases which we believe may be amenable to TANGO. At Stoke, we are united around a common goal: to improve the lives of people living with severe genetic diseases. Stoke is located in Bedford, MA with offices in Cambridge, MA.
Gennao Bio is a privately held genetic medicines company developing first-in-class targeted nucleic acid therapeutics utilizing a proprietary gene monoclonal antibody (GMAB) platform technology.
Gotham Therapeutics has assembled an exceptional team and network to establish a novel drug class targeting RNA-modifying proteins.
Aids Vaccine Advocacy Coalition is a New York, NY-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
REGENXBIO is a leading biotechnology company focused on the development, commercialization and licensing of recombinant adeno‐associated virus (AAV) gene therapy. REGENXBIO`s NAV® Technology Platform, a proprietary AAV gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO`s mission is to transform the lives of patients suffering from severe diseases with significant unmet medical need by developing and commercializing in vivo gene therapy products based on REGENXBIO`s NAV Technology Platform. REGENXBIO seeks to accomplish this mission through a combination of internal development efforts and third‐party NAV Technology Platform licensees.