According to the partnership terms, Solid Biosciences will gain access to muscle-selective promoter candidates developed by Synpromics, which are based on the company’s synthetic promoter design technology.
Synthetic promoters are small genetic sequences that do not exist naturally. They are engineered to regulate the activity of genes and control the production of the proteins that are encoded by those genes. Synpromics’ technology allows the development of tailored synthetic promoters that can trigger the expression of a specific gene.
“We are excited to leverage our PromPT technology platform to generate promoter candidates closely matching the precise criteria and specific product requirements for Solid Biosciences’ muscle gene medicine program in Duchenne,” David Venables, CEO of Synpromics, said in a press release. “The collaboration between the scientific teams will enable the effective and rapid in vivo evaluation of the candidate promoters as compared to industry standards.”
The collaboration with Synpromics will help Solid Biosciences advance its muscle-directed gene therapy research by improving the specificity and potency of its investigational gene therapy candidates for DMD.
“Synpromics’ cutting-edge technology has the potential to enhance our gene therapy development efforts, helping us to fulfil our promise to bring meaningful therapies to all patients with this devastating disease,” said Joel Schneider, PhD, chief technology officer and head of exploratory research and development at Solid Biosciences.
“This type of collaboration is key for building our next-generation gene therapy portfolio as we focus on enhancing fundamental aspects of our gene delivery and protein expression capabilities,” Schneider said.
Solid Biosciences is not only focused on developing new options to overcome the underlying mechanism of DMD, but also to find new strategies to manage the disease manifestations.
In preclinical studies, the company’s lead drug candidate SGT-001 has demonstrated the potential to correct DMD gene deficiency in canine models of DMD. Solid Biosciences is planning to initiate a Phase 1/2 human trial at clinical sites in the United States, and potentially in some centers abroad. This trial will assess the efficacy, safety, and tolerability of SGT-001.
DMD is a rare genetic disease that causes muscle weakness. It starts in childhood, usually between ages 3 and 5, and may be noticed when a child has difficulty standing up, climbing, or running. The disease affects about one in every 3,500-5,000 boys worldwide. DMD is caused by an absence of dystrophin, a protein that helps keep muscle cells intact.