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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.
Protagonist Therapeutics is a biotechnology company pursuing the discovery and development of target oral peptides as well differentiated alternatives to antibodies, and also as new chemical entities (NCEs) against those targets and life threatening diseases for which suitable small molecule and/or biologic options are not available. Peptides typically suffer from limitations of poor proteolytic stability and therefore find scarce therapeutic utility that is largely limited to ‘injectable drugs’. Protagonist’s technology platform is aimed at overcoming these restrictions and expanding the scope of peptide therapeutics to address unmet needs. Specific emphasis is placed on identifying ‘orally stable’ scaffolds and/or engineering oral stability characteristics onto them. The platform has been optimized over the years and involves synergistic integration of rational drug design, diversity oriented computational tools, phage display libraries, recombinant peptide expression, ex vivo oral stability methods, and peptide/medicinal chemistry techniques. This activity has led to the identification of ‘privileged scaffolds’ with favorable oral stability characteristics. Furthermore, the technology platform is well suited both for de novo discovery against a target and optimization around a given chemical starting point.
Lysogene is a biopharmaceutical gene therapy company with lead programs in neurodegenerative lysosomal storage disorders that affect children. Founded in 2009 by Karen Aiach, whose own child was affected by a neurodegenerative disease, Lysogene is rooted in a deep and compassionate understanding of the impact these diseases have on patients and families. Karen`s personal experience fuels her determination to deliver real solutions that will improve patient outcomes and enhance quality of life for both patients and caregivers. Over the past 10 years, Lysogene has acquired deep experience in developing gene therapies—from early discovery through the clinical, registration and review phases. Our strong science and execution track record has been validated by our partnership with Sarepta Therapeutics and the support of top-tier investors, with nearly $100m raised since inception.
Denali Therapeutics Inc. (“Denali”) is a biotechnology company focused on the discovery and development of therapies for patients with neurodegenerative diseases, including Alzheimer’s disease, Parkinson’s disease, ALS and others. Denali was founded by scientists, industry experts and investors who share the vision that recent scientific insights into the genetic causes and biological processes underlying neurodegenerative disease, together with new translational medicine tools, offer an unprecedented opportunity to discover and develop effective medicines. Denali is rigorously pursuing a science-driven approach to translational medicine and clinical development. Founding investors include Fidelity Biosciences, ARCH Venture Partners, Flagship Ventures and the Alaska Permanent Fund (represented by Crestline Investors).
Novartis Consumer Health Inc is a Big Rapids, MI-based company in the Healthcare, Pharmaceuticals, and Biotech sector.