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Type 1 diabetes (T1D), formerly known as juvenile diabetes, is a chronic, life-threatening disease that affects millions of people worldwide. In the United States, 30,000 new cases are estimated every year with half of those cases diagnosed in young children. Type 1 diabetes is an autoimmune disease in which the patient`s immune system goes awry and attacks and destroys the pancreatic beta cells. Beta cells are responsible for regulating blood sugar (glucose) levels by producing precise amounts of the essential hormone insulin. The discovery of injectable insulin in the 1920s changed T1D from a uniformly fatal disease with a life expectancy of months to one that could be carefully managed for decades through multiple daily blood glucose measurements and insulin injections. However, insulin injections are not a cure and patients face a lifetime of difficult disease management and serious complications including kidney failure, blindness and nerve damage. Despite nearly a century passing since the discovery of insulin, insulin injection remains the only treatment available to patients. Semma Therapeutics was founded to develop transformative therapies for patients who currently depend on insulin injections. Recent work in the laboratory of Professor Douglas Melton led to the discovery of a method to generate billions of functional, insulin-producing beta cells in the laboratory. These cells develop in islet-like clusters grown from stem cells. Initial preclinical work in animal models of diabetes has shown that transplantation of these cells are sufficient to control blood glucose levels. This breakthrough technology has been exclusively licensed to Semma Therapeutics for the development of a cell-based therapy for diabetes. Ongoing research at Semma Therapeutics is focused on combining these proprietary cells with a state-of-the-art cell delivery and immune protection strategy that can protect these cells from the patient`s immune system and allow the beta cells to function as they do in non-diabetic individuals. Implantation of the beta cell-filled device has the potential to provide a true replacement for the missing beta cells in a diabetic patient and would not require patient immunosuppression. Semma Therapeutics is working to bring this new therapeutic option to the clinic and improve the lives of patients with diabetes.
Sierra Oncology is a clinical stage drug development company advancing targeted therapeutics for the treatment of patients with unmet medical needs in hematology and oncology. Our lead drug candidate, momelotinib, is a potent, selective and orally-bioavailable JAK1, JAK2 & ACVR1 inhibitor with a differentiated therapeutic profile in myelofibrosis encompassing a range of meaningful anemia benefits, including eliminating or reducing the need for frequent blood transfusions, as well as achieving substantive spleen and constitutional symptom control. We are also advancing SRA737 and SRA141. SRA737, is a potent, highly selective, orally bioavailable small molecule inhibitor of Checkpoint kinase 1 (Chk1). We are pursuing an innovative development plan for SRA737, which is currently being evaluated in two Phase 1/2 clinical trials in patients with advanced cancer. SRA737-01 is intended to evaluate SRA737`s potential to induce synthetic lethality as monotherapy, while SRA737-02 is intended to evaluate the combination of SRA737 potentiated by subtherapeutic, low dose gemcitabine. Concurrently, we are conducting preclinical research evaluating SRA737 in combination with other DDR-targeted agents, including PARP inhibitors, as well as with immuno-oncology therapeutics, that may guide a potential next wave of clinical development for our asset, possibly further broadening its therapeutic utility. SRA141, a potent, selective, orally bioavailable small molecule inhibitor of cell division cycle 7 kinase (Cdc7). Cdc7 is a key regulator of DNA replication and is involved in the DDR network, making it a compelling emerging target for potential treatment across a broad range of tumor types. An Investigational New Drug Application (IND) submission to the U.S. Food and Drug Administration (FDA) is being prepared in order to commence clinical trials with this drug candidate.
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