Every gene requires a naturally occurring combination of DNA known as a promoter to code and produce a protein. Synpromics and UCL will develop synthetic promoters that can overcome nervous system gene mutations. Synthetic promoters are composed of DNA combinations not seen in nature.
The partners want to develop promoters that control the various kinds of nerve cell genes’ protein production. They will use Synpromics’ patented promoter-development platform in their work.
Promoters are basically switches that control genes’ production of proteins. For the most part, scientists are unable to use natural promoters to create neurological disease therapies.
Because synthetic promoters have DNA sequences not found in nature, they have the ability to regulate genes’ activity, including protein production.
The gene switches that UCL will create will help it create a gene therapy for people who develop Parkinson’s when they’re fairly young.
Between 10 and 20 percent of Parkinson’s patients are diagnosed before the age of 50. And half of this early-onset group are diagnosed before 40.
“Tightly controlling the therapeutic gene is an essential element in the development of any successful gene therapy, and Synpromics’ technology offers the best means to achieve that control,” Michael Roberts, the company’s founder and chief scientific officer, said in a press release.
“This collaboration will allow the company to develop a gene therapy approach for a largely unmet clinical need, where tight gene control is an absolute requirement,” he said. “It also gives us the opportunity to work with UCL, one of the few world-leading institutions actively developing novel gene-based therapies.”
The collaboration will last two years, with work equally split between Synpromics and UCL.
“We are delighted to be working with the leaders in gene control, Synpromics, to develop gene therapy for young-onset Parkinson’s disease,” said Simon Waddington of UCL’s Institute for Women’s Health. “Parkinson’s is the second most common untreatable progressive brain disease, and novel therapeutic approaches are required. This collaboration allows us to develop tailor-made gene therapy vectors for untreatable brain disorders.”