According to a story from BioPortfolio, Rare Disease Day, which took place on February 28th, 2019, serves as a time of reflection on the progress that has been made in developing treatments for rare diseases. Rare diseases affect around 30 million people in the US, a fact which highlights that while the number of patients with a single rare disease may be pretty low, the impact when all of the diseases are put together is significant.
Developing Therapies for Rare Disease
Bioscience companies based in Ohio have begun to get involved in the field of developing new treatments for these rare diseases.The city of Cleveland is home to Abeona Therapeutics and Milo Biotechnology. Both of these companies are planning on developing therapies in a number of rare indications, such as Batten disease, Sanfilippo syndrome, muscular dystrophy, and epidermolysis bullosa. Companies like these are also helped by organizations such as BioMotiv and BioEnterprise, two organizations which can help biotechnology companies get on their feet.
Technology derived from research facilitated by the Nationwide Children’s Hospital in Columbus was instrumental in getting these companies started, and this hospital, alongside the Ohio State University School of Medicine, have also made valuable contributions in the rare disease space. Another company called Myonexus, which is also another derivative from Nationwide, has also entered a partnership with Sarepta Therapeutics in order to test a new experimental therapy for Duchenne muscular dystrophy. A company called AveXis, which was recently scooped up by the drug company Novartis, is in the midst of putting together a promising gene therapy for type 1 spinal muscular atrophy and the results from trials so far have been encouraging.
There have also been advances coming from the University of Cincinnati. Researchers at the school conducted critical research and early development for an experimental therapy intended to treat creatine transporter deficiency syndrome. This drug will be groomed for the commercial market by Lumos Pharma.
There are over 7,000 identified diseases that are considered rare. Unfortunately, only about 500 of them have any approved therapies. There is clearly much work to be done in improving treatment for rare disease patients, but it is clear that biotech companies based in Ohio are doing their part to develop innovative therapies that can change the lives of these patients for the better.