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Korro Bio is an RNA editing company focused on the discovery and development of a new class of precision genetic medicines for both rare and common diseases. The company`s proprietary and modular platform, OPERA™ (Oligonucleotide promoted editing of RNA), builds on a deep understanding of ADAR biology, and combines data-driven design, oligo discovery and chemistry with clinically validated delivery vehicles, to achieve highly selective RNA editing. This unique technology enables the development of RNA editing therapeutics that deliver the functional benefits of gene therapy with a reversible, transient, titratable and specific treatment regimen, offering the potential to propel genetic medicine beyond rare genetic diseases into larger patient populations with common diseases. Korro is based in Cambridge, Mass.
Quad Five Materials Bio is a Ryegate, MT-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Spheryx, Inc. is a privately held analytical services and instruments company providing Total Holographic CharacterizationTM of colloidal materials. Spheryx’s breakthrough proprietary technology uses holographic video microscopy to characterize each particle in colloidal dispersions and multi-component colloidal mixtures, thereby offering unprecedented insights into these materials’ characteristics. Applications include research and development, quality assurance and manufacturing process control across a broad spectrum of industries, where characterization of colloids can enhance innovation, improve safety and reduce costs.
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.
Taysha Gene Therapies is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we hope to rapidly translate our treatments from bench to bedside. We have combined our team`s proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to quickly and efficiently build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for new cures—to dramatically improve patients` lives.