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Portage is a unique entity in the world of biotechnology, catalyzing R&D to produce more quality clinical programs and maximize potential returns.
Our mission is to create a new generation of smarter medicines that outmaneuver complex diseases in ways previously inconceivable. To accomplish this mission, we are building a synthetic biology platform that could enable us to program next-generation cell and gene therapies with what we refer to as “gene circuits”. These gene circuits, which are created from novel and proprietary combinations of DNA sequences, are intended to reprogram cells with biological logic to sense inputs, compute decisions and respond to their cellular environments. We are designing gene circuits to improve the “intelligence” of cell and gene therapies in order to enhance their therapeutic effectiveness against a broad range of diseases that conventional medicines do not readily address.
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.
Cleave Biosciences is a biopharmaceutical company pioneering the development of novel protein homeostasis inhibitors for the treatment of cancer.
Arkuda Therapeutics is a biotechnology company applying insights into lysosomal biology to drive the development of medicines to change the trajectory of neurodegenerative disease. Arkuda`s lead program ARKD-104 aims to correct progranulin deficiency and lysosomal dysfunction in patients with GRN-related frontotemporal dementia (FTD-GRN), a genetically-defined subtype of frontotemporal dementia caused by an autosomal dominant mutation in the progranulin (GRN) gene. The company is further exploring the therapeutic potential of its molecules in other neurodegenerative diseases where genetic links to dysfunction in progranulin biology have been established, including Alzheimer`s Disease and Parkinson`s Disease. Arkuda is backed by leading investors including Atlas Venture, Cormorant Asset Management, Pfizer Ventures, funds managed by Tekla Capital Management LLC, Mission BioCapital, and Eli Lilly and Company.