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Enzyvant is also advancing the development of RVT-802, an investigational tissue-based biologic therapy for the potential treatment of primary immune deficiency associated with complete DiGeorge Syndrome. RVT-802 has been granted orphan drug designation, Breakthrough Therapy designation, Regenerative Medicine Advanced Therapy designation, and pediatric rare disease designation by the U.S. Food and Drug Administration. Enzyvant anticipates a potential BLA filing for RVT-802 in the first half of 2018. Enzyvant plans to develop treatments for additional rare diseases with high unmet need.
Founded by Walter Lorenz more than thirty years ago, Biomet Microfixation manufactures a complete range of craniomaxillofacial products, including LactoSorb, the first completely resorbable fixation system available in the U.S. Other products include
Scribe is focused on the engineering, delivery, and development of next-generation CRISPR molecules to rewrite and repair the underlying cause of genetic disorders.
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.