According to the National Institute of Neurological Disorder and Stroke, Huntington’s disease arises from genetically programmed degeneration of neurons in brain which may lead to emotional disturbance, uncontrolled movements or loss of intellectual faculties. This abnormality causes the loss of memory and perception. The increasing growth in the pipeline for Huntington’s disease is primarily attributed to the limited number of treatments available, as well as the technical advancements in medical field which are in turn boosting the research and development over gene related diseases. Various gene therapies and cell therapies have been developed, that have proven their efficacy in treatment of Huntington’s disease. The funding from various organization, which are involved in running programs related to spreading awareness regarding Huntington’s disease is another factor which has aided in the growth of pipeline of this disease. Therefore, the pipeline growth of Huntington’s disease is shooting up at a significant rate.
In August 2015, Ionis Pharmaceuticals, Inc. started a Phase I/II clinical trial on drug, ISIS 443139. This trial is a randomized, double-blinded, placebo-controlled study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of multiple ascending doses of intrathecally administered ISIS 443139 in patients with early symptoms of Huntington's Disease. The study is expected to be completed in November 2017. In June 2016, Azevan Pharmaceuticals Inc. started a Phase I/II clinical study on drug SRX246 to determine its safety, efficacy and activity in patients with Huntington’s disease. This trial is expected to be completed in June 2018. In June 2015, Ultragenyx Pharmaceutical Inc. started a Phase II clinical study to assess the activity of Triheptanoin as an Anaplerotic therapy in Huntington’s disease. This trial is expected to be completed in June 2019.
In February 2015, Voyager Therapeutics, Inc. and Genzyme Inc. had underwent a major strategic collaboration to discover, develop and commercialize novel gene therapies for severe CNS disorders. This collaboration would influence the long-standing commitment and scientific leadership of Genzyme Inc. in the field of adeno-associated virus (AAV) gene therapy and industry-leading AAV product engine of Voyager Therapeutics, Inc. to develop breakthrough therapies for patients suffering from severe CNS disorders. In February 2015, QR Pharma, Inc. signed a collaborative agreement with The Rockefeller University for a development of therapy against Huntington’s disease. This collaboration primarily focused on preclinical research involving QR Pharma, Inc.’s lead drug candidate, Posiphen. Additionally, the investigators at the Rockefeller also conducted experiments in stem cell culture models of Huntington’s disease to determine whether Posiphen lowers the levels of the toxic huntingtin protein in these cells or not. In September 2016, Teva Pharmaceutical Industries Ltd signed an agreement with Intel Corporation to develop a wearable technology platform to track the progression of disease in patients with Huntington's, a fatal degenerative disorder. Teva Pharmaceutical Industries Ltd with Intel Corporation deployed this technology as part of their ongoing mid-stage Huntington's study. In January 2011, Targeted Genetics Corporation and Sirna Therapeutics, Inc. entered into a collaborative agreement to develop novel therapies for the treatment of Huntington's Disease.
Some of the companies having a pipeline of Huntington’s disease therapeutics include, Ionis Pharmaceuticals, Inc., Azevan Pharmaceuticals Inc., Teva Pharmaceutical Industries Ltd., CHDI Foundation, Inc., GlaxoSmithKline plc, Janssen Research Development, LLC, Celgene Corporation, Novartis AG, F. Hoffmann-La Roche AG, Boehringer Ingelheim GmbH, Eli Lilly and Company.