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AVROBIO`s vision is to bring personalized gene therapy to the world. We aim to prevent, halt or reverse disease throughout the body with a single dose of gene therapy designed to drive durable expression of therapeutic protein, even in hard-to-reach tissues and organs including brain, muscle and bone. Our ex vivo lentiviral gene therapy pipeline includes clinical programs in Fabry disease, Gaucher disease type 1 and cystinosis, as well as preclinical programs in Hunter syndrome, Gaucher disease type 3 and Pompe disease. AVROBIO is powered by our industry leading plato® gene therapy platform, our foundation designed to deliver gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario.
Applied Molecular Therapeutics (AMT), a public biopharmaceutical company based in South San Francisco, California, is committed to developing novel, oral biological therapeutics to treat severe autoimmune, metabolic and inflammatory diseases. AMT is leveraging its propriety technology platforms to create gastrointestinal (GI)-select therapeutics that harness naturally occurring transport and targeting mechanisms to cross the protective barrier of the intestinal epithelium (IE). Once across the IE, the therapeutics gain privileged access to the immune cell-rich environment of the GI tissue as well as the hepatic portal system and downstream systemic circulation. With this privileged access, AMT`s goal is to develop transformative new oral therapeutic treatment options that offer patients greater efficacy and tolerability than available with today`s therapeutics. AMT has a robust pipeline of oral biologic product opportunities, including AMT-101 which is currently in Phase 1b clinical development for the treatment of adults with ulcerative colitis (UC). AMT-101 is a novel, gut-selective, investigational oral biologic fusion protein of interleukin 10 (IL-10), an anti-inflammatory cytokine. Derived from AMT`s proprietary platforms, AMT-101 is engineered to cross the selective barrier of the IE and enhance localized IL-10 directly within the immune-cell rich environment of GI tissue where up to 75 percent of immune cells reside. This direct targeting of the immune system creates the potential for AMT-101 to address, at its point of origin, the immune dysregulation that gives rise to UC and other inflammatory bowel diseases, and restore immune homeostasis with minimal systemic exposure and potentially fewer adverse events compared to systemic administration of IL-10. AMT has amassed a broad global IP portfolio surrounding its multiple technology platforms and therapeutic programs.
Vizgen is unlocking the power of spatial biomolecule profiling to bring new insight into biological systems and disease.
Merck KGAA is a Durham, NC-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
ChromaCode is a molecular diagnostics company with a bioinformatics focus and pedigree from Illumina, Google[x], Luminex, and Caltech.