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Adverum Biotechnologies (Nasdaq: ADVM) is a clinical-stage gene therapy company targeting unmet medical needs for serious ocular and rare diseases. Adverum is evaluating its novel gene therapy candidate, ADVM-022, as a one-time, intravitreal injection for the treatment of its lead indication, wet age-related macular degeneration.
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.
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.
At AviadoBio our mission is to transform the lives of people living with neurodegenerative disorders by developing and delivering transformative gene therapies for diseases including frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). The Company`s technology is based on pioneering research from King`s College London and the UK Dementia Research Institute.
Bactrin International is a Belgrade, MT-based company in the Healthcare, Pharmaceuticals, and Biotech sector.