WESTON, Mass. & CARLSBAD, Calif.--(BUSINESS WIRE)-- Biogen Idec (BIIB) and Isis Pharmaceuticals, Inc. (ISIS) today announced that they have entered into an exclusive, worldwide option and collaboration agreement under which the companies will develop and commercialize a novel antisense drug for the treatment of myotonic dystrophy type 1 (DM1), which is also known as Steinert disease. DM1, the most common form of muscular dystrophy in adults, is a genetic neuromuscular disease characterized by progressive muscle atrophy, weakness and disabling muscle spasms. It is caused by a genetic defect in the dystrophia myotonica-protein kinase (DMPK) gene in which a sequence of three nucleotides repeats extensively, creating an abnormally long toxic RNA, which accumulates in the cell and prevents the production of proteins needed for normal cellular function. Isis DM1 antisense program is being developed to correct the underlying genetic defect that causes DM1. Isis will receive an upfront payment of $12 million and is responsible for the discovery of a lead antisense drug candidate targeting DMPK for the treatment of DM1. Isis is eligible to receive up to $59 million in milestone payments associated with the clinical development of the DMPK-targeting drug prior to licensing. Biogen Idec has the option to … Continue reading →

By Chris Reidy, Globe Staff Biogen Idec Inc., a global biotechnology company headquartered in Weston, said Friday that it is entering another collaboration with Isis Pharmaceuticals Inc., with this collaboration focusing on an experimental drug for myotonic dystrophy, a debilitating neuromuscular disease. In January, the two companies announced plans to work together on a potential treatment for spinal muscular atrophy. Fridays agreement calls for California-based Isis to receive an upfront payment of $12 million, the two companies said in a press release. Under the agreement, Isis is responsible for the discovery of a lead antisense drug candidate for the treatment of myotonic dystrophy type 1, which is also known as Steinert disease. Isis could receive up to another $200 million in a license fee and regulatory milestone payments as well as double-digit royalties on sales of the drug. Isis will be responsible for global development of the drug through the completion of Phase 2 clinical trials, with Biogen Idec providing advice on the clinical trial design and regulatory strategy, the companies said in a press release. If Biogen Idec exercises its option under the agreement, it will assume global development, regulatory, and commercialization responsibilities. Biogen Idec said the collaboration fits … Continue reading →

Editor's Choice Main Category: Muscular Dystrophy / ALS Also Included In: Transplants / Organ Donations Article Date: 29 Jun 2012 - 11:00 PDT Current ratings for: Stem Cells From Muscular Dystrophy Patients Transplanted Into Mice A new study published in Science Translational Medicine reveals that researchers have, for the first time, managed to turn fibroblast cells, i.e. common cells within connective tissue, from muscular dystrophy patients into stem cells and subsequently changed these cells into muscle precursor cells. After modifying the muscle precursor cells genetically, the researchers transplanted them into mice. In future, this new technique could be used in order to treat patients with the rare condition of limb-girdle muscular dystrophy, which primarily affects the shoulders and hips, and maybe other types of muscular dystrophies. The method was initially developed in Milan at the San Raffaele Scientific Institute and was completed at UCL. Muscular dystrophy is a genetic disorder, which typically affects skeletal muscles. The condition leads to severely impaired mobility and can, in severe cases result in respiratory and cardiac dysfunction. At present, there is no effective treatment for the condition. A number of new potential therapies, including cell therapy, are entering clinical trials. The scientists of this … Continue reading →