UCL is collaborating with Synpromics on a gene therapy for young-onset Parkinson's disease. The pact will see UCL work with Synpromics on synthetic gene promoters that enable highly specific gene therapies.
Synpromics is bringing its expertise in synthetic gene promoters to the collaboration. These are DNA sequences that activate transcription, driving the synthesis of a protein associated with their target genes. Some drug developers piggyback on nature by deploying promoters found in the body. But Synpromics eschews naturally occurring promoters in favor of novel, purpose-designed sequences tailored to different gene expression profiles.
The goal is to deliver higher levels of gene expression and fewer off-target effects than is possible using natural promoters. Synpromics CSO Michael Roberts, Ph.D., thinks such a favorable profile is particularly important when targeting Parkinson’s.
“Tight gene control is an absolute requirement,” Roberts said in a statement.
UCL, formerly known as University College London, shares Synpromics’ belief in synthetic gene promoters. The two organizations will spend the next two years in an equal collaboration to create novel gene promoters. If the promoters can control gene expression in subpopulations of neurons, as the collaborators hope, UCL will use the switches to develop Parkinson’s gene therapies.
Multiple research groups are working on programs that fall under the umbrella of Parkinson’s gene therapies. Early efforts entered the clinic years ago. A more recent entrant, Voyager Therapeutics, posted early clinical data on its attempt to improve and prolong the efficacy of levodopa earlier this year.
UCL’s planned program is a different spin on the broad idea of Parkinson’s gene therapies. And the U.K. academic institution has a track record that suggests it may emerge from the Synpromics alliance with the makings of a candidate and a company. In recent years, UCL’s commitment to gene therapy research has contributed to the emergence of a clutch of startups including Athena Vision, Freeline Therapeutics and Orchard Therapeutics.