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There are approximately 7,000 rare disorders worldwide. Each day more are being discovered. 95% of rare diseases have not one single FDA approved treatment. Glycomine develops therapeutics for diseases which have no treatment options.
Nitrome Biosciences has discovered and identified a new class of enzymes.
Autobahn Therapeutics is focused on improving life health for people affected by CNS disorders by driving the regenerative power of the human body. Altering the progressive and debilitating course of these conditions by returning the brain to a healthier state will transcend the benefits offered by existing therapies and allow people to live the futures they deserve.
Translate Bio is a leading mRNA therapeutics company developing a new class of potentially transformative medicines to treat diseases caused by protein or gene dysfunction. Our proprietary mRNA therapeutic platform (MRTTM) is designed to develop product candidates that deliver mRNA carrying instructions to produce intracellular, transmembrane and secreted proteins for therapeutic benefit. We believe that our MRTTM platform is applicable to a broad range of diseases caused by insufficient protein production or where production of proteins can modify disease, including diseases that affect the lung, liver, eye, central nervous system, lymphatic system and circulatory system. Our two lead programs are being developed as treatments for cystic fibrosis (CF) and ornithine transcarbamylase (OTC) deficiency.
Our founding belief is that the future of medicine will rely on artificial intelligence, because biology is too complex for humans to understand. Today, the molecular world of the cell can be experimentally interrogated like never before. The resulting datasets provide an unprecedented opportunity to build artificial intelligence systems that are biologically accurate and that support the detection of disease and the development of molecular interventions. Deep Genomics is building a biologically accurate data- and AI-driven platform that supports geneticists, molecular biologists and chemists in the development of therapies. Over the next two years, Deep Genomics will use its platform to unlock new classes of antisense oligonucleotide therapies that were previously inaccessible or out of reach, and advance them for clinical evaluation. In project Saturn, the platform will be used to search across a vast space of over 69 billion molecules with the goal of generating a library of 1000 compounds that can be used to manipulate cell biology and design therapies.