Recognizing the Ohio Bioscience Industry on Rare Disease Day
How do you define rare? For upwards of 350 million people worldwide, rare is the term used to describe an illness or condition they live with every day. In the United States, upwards of 30 million individuals are living with one of more than 7,000 identified rare and orphan diseases. Of those 7,000-plus known diseases, only about 500 currently have some identified form of treatment.
On February 28, patients, families, caregivers, and researchers around the world will come together for Rare Disease Day — an international day of recognition dedicated to raising awareness about rare diseases and the impact they have on patients and their families.
Though the numbers seem grim, a good deal of progress has been made. Cystic Fibrosis, for example, is a rare disease that affects 30,000 people in the US. Thanks to increased incentives for rare disease research and drug discovery provided by the Orphan Drug Act, there are now currently more than 24 approved treatments for the disease.
But for every success story and new treatment that is approved for someone living with a rare disease, there is another story of a patient with no treatment options available. The Ohio bioscience industry is working hard to change that.
The state of Ohio is home to more than 3,300 bioscience companies, top-ranked health systems, and research universities—many of which are focused on developing innovative new treatments for rare disease patients. BioOhio, the state’s membership organization focused on supporting Ohio’s bioscience community, reports yearly growth within the industry in their annual publication, The Ohio Bioscience Growth Report.
In Cleveland, companies like Milo Biotechnology and Abeona Therapeutics, both launched based on technologies from Nationwide Children’s Hospital in Columbus, are developing therapies for a range of rare diseases, including various forms of muscular dystrophy, Epidermolysis Bullosa, Sanfilippo Syndrome, and Batten Disease. These companies and many other biotech start-ups have worked closely with BioEnterprise, a venture development organization that provides services to launch successful biotech companies in the region. In addition, BioMotiv, a biotech accelerator, and The Harrington Project, centered at University Hospitals in Cleveland, have unique platforms to identify promising technologies nationally and bring together pharmaceutical expertise, aligned capital, and industry partners to accelerate these breakthrough discoveries from academic institutions into therapeutics for patients.
Nationwide Children’s and The Ohio State University School of Medicine, both in Columbus, are the sources of numerous innovative therapeutic advances for rare disease, especially novel genetic therapies. Myonexus, another spinout from Nationwide Children’s that is supported by regional accelerators Rev1 Ventures and CincyTech, recently partnered with Sarepta Therapeutics to conduct clinical trials for patients with Duchenne muscular dystrophy.
AveXis, which was recently acquired by Novartis, is also developing a promising technology based out of Nationwide Children’s. This unique gene therapy treatment and novel manufacturing process to treat spinal muscular atrophy Type 1, among other opportunities, is showing encouraging success during clinical trials.
From the university side, Lumos Pharma is commercializing a drug candidate developed out of the University of Cincinnati for the treatment of Creatine Transporter Deficiency Syndrome. Researchers at Ohio University developed Somavert, a human growth hormone blocker, which was launched commercially by Pfizer in 2011.
We are proud to be a part of Ohio’s growing bioscience industry. And on this Rare Disease Day, we recognize the families who fiercely advocate for new research and treatments for their loved ones. We recognize the caregivers who provide care and comfort to patients and families who are looking for answers. We recognize the scientists and researchers who have dedicated their careers to developing treatments and cures that could change lives. Thank you for the difference you make to millions of rare patients around the world.
Baiju Shah, CEO, BioMotiv
John F. Lewis, Jr., President & CEO, BioOhio