Periodic Reporting for period 1 - FLDcure (A potent Micro-RNA therapeutic for nonalcoholic fatty liver disease (NAFLD))
Periodo di rendicontazione: 2015-01-01 al 2016-06-30
MicroRNA (miRNA) family members enable simultaneous modulation of numerous target mRNAs and thereby control entire physiological pathways. We have discovered that engineered overexpression of miR-132 in body tissues triggers rapid and massive accumulation of fat in the liver as well as elevated triglycerides and circulating fatty acids, classic symptoms of Nonalcoholic fatty liver disease (NAFLD). Furthermore, we proved that reducing miR-132 levels in peripheral tissues by injection of Locked Nucleic Acids (LNA)-protected antisense oligonucleotides to miR-132 (AntiMiR-132) showed rapid therapeutic efficacy in reducing the fatty liver accumulation and other NAFLD biomarkers in a mouse model of NAFLD.
This unexpected discovery encouraged us to pursue the development and testing of LNA AntiMiR-132 as a novel and potent therapeutic for the treatment of NAFLD, a widespread medical condition that affects as many as one-third of adults and an increasing number of children in developed countries. The disease begins with the aberrant accumulation of triglycerides (fat) in the liver, can progress to nonalcoholic steatohepatitis (NASH) and subsequently to cirrhosis and liver cancer. We are now in the process of developing microRNA based therapeutic for the treatment of NAFLD. The aims of FLD cure was to enable us to continue with preclinical testing, solidifying intellectual property protection, pursuing negiotiations with industry and recruiting outside funding for the translational research. All of these goals have now been achieved. We have received research and development funding from a healthcare venture capital company, conducted further research both in house and in collaboration with a contract research organization (CRO), filed patents for IP protection in collaboration with a leading IP law firm and have continued negotiations with several large Pharma companies. We hope that continued preclinical and translational research will allow AM132 to become a potent pharmaceutical treatment for NAFLD.
This unexpected discovery encouraged us to pursue the development and testing of LNA AntiMiR-132 as a novel and potent therapeutic for the treatment of NAFLD, a widespread medical condition that affects as many as one-third of adults and an increasing number of children in developed countries. The disease begins with the aberrant accumulation of triglycerides (fat) in the liver, can progress to nonalcoholic steatohepatitis (NASH) and subsequently to cirrhosis and liver cancer. We are now in the process of developing microRNA based therapeutic for the treatment of NAFLD. The aims of FLD cure was to enable us to continue with preclinical testing, solidifying intellectual property protection, pursuing negiotiations with industry and recruiting outside funding for the translational research. All of these goals have now been achieved. We have received research and development funding from a healthcare venture capital company, conducted further research both in house and in collaboration with a contract research organization (CRO), filed patents for IP protection in collaboration with a leading IP law firm and have continued negotiations with several large Pharma companies. We hope that continued preclinical and translational research will allow AM132 to become a potent pharmaceutical treatment for NAFLD.