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AGTC is a clinical-stage biotechnology company that uses its proprietary gene therapy platform to develop products designed to transform the lives of patients with severe diseases, with an initial focus in ophthalmology. AGTC`s lead product candidates are designed to treat inherited orphan diseases of the eye, caused by mutations in single genes that significantly affect visual function and currently lack effective medical treatments. AGTC`s product pipeline includes six named ophthalmology development programs across five targets (X-linked retinoschisis (XLRS), X-linked retinitis pigmentosa (XLRP), achromatopsia, wet age-related macular degeneration and blue cone monochromacy), two non-ophthalmology programs (alpha-1 antitrypsin deficiency and adrenoleukodystrophy) and AGTC is continuing to develop early research studies in additional indications. The company is also exploring genetic defects in cells in the inner ear that lead to deafness and expects to advance several product candidates into development within the next few years. AGTC employs a highly targeted approach to selecting and designing its product candidates, choosing to develop therapies for indications having high unmet medical need, clinical feasibility and commercial potential. AGTC has a significant intellectual property portfolio and extensive expertise in the design of gene therapy products including capsids, promoters and expression cassettes, as well as, expertise in the formulation, manufacture and physical delivery of gene therapy products
NeuroVasc Technologies is a developer of mechanical thrombectomy solutions for revascularization of the brain.
Nurix Therapeutics discovers drugs that harness the body`s natural process to control protein levels. Our drugs control ubiquitin E3 ligases, the key enzymes responsible for protein breakdown in human cells, as a unique therapeutic approach to treat a broad range of diseases. Our focus is on developing drugs to treat cancer including novel, small molecule immuno-oncology agents.
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.
We are bringing an entirely new approach to the treatment of autoimmune disease by engineering Tregs as targeted therapies that stop immune-mediated destruction, restore homeostasis – a state of harmony – and promote repair in the affected tissues. In addition to our lead program in progressive MS, Abata has early programs in Type 1 Diabetes (T1D) and inclusion body myositis (IBM). We bring together industry experts and deeply engaged pioneers in Treg biology, T cell receptor and antigen discovery, disease pathogenesis, and molecular and imaging biomarkers. We are bold in our mission and purpose and aim for nothing less than a transformative impact on people`s lives.