Asklepios BioPharmaceutical has created a non-profit organization to develop cell and gene therapies for orphan diseases by increasing the number of scientific collaborations.
The company formed the Chapel Hill Project to have its technology accessible to researchers for use in orphan drug development without the paying up-front costs.
Orphan (rare) diseases are life-threatening or chronically debilitating diseases affecting fewer than 200,000 people in the United States. Due to the small market size of orphan drugs, pharmaceutical companies do not generally invest significant funds toward their research and development. As a result, most sufferers of orphan diseases lack adequate treatments, the company said in a news release.
Officials for Asklepios BioPharmaceutical said their main goal for the Chapel Hill Project is to develop medication for diseases with no viable treatment options. By providing access to technology, scientific and clinical trials expertise and funding, the organization will aid in translating science from the laboratory to the clinic.
An advisory board consisting of key clinicians and scientists will advise researchers, helping them turn scientific research into cost-effective, viable treatment options. The Chapel Hill Project will also act as a depository for intellectual property that can be used for the development of therapeutics for orphan diseases.
"We want to connect scientists and clinicians to expedite the translation of gene and cell based therapeutics for orphan diseases into the clinic," said Jude Samulski, the company's chief scientific officer. "We encourage large pharmaceutical companies to provide researchers with access to their technology and assist with advancement into clinical trials either through the Chapel Hill project or through their own structures."