This research collaboration will help advance Solid Bioscience’s programme to develop optimal product candidates for the company’s adeno associated virus muscle-directed gene therapy research programme
Synpromics, a specialist in gene control, announced a new research partnership with Solid Biosciences. Under the terms of the agreement, Synpromics will provide Solid Biosciences access to a set of key muscle-selective promoter candidates to be used in the development of new treatment options for Duchenne muscular dystrophy (DMD).
The use of Synpromics’ synthetic promoters will enable the enhancement of Solid Biosciences’ investigational gene therapy candidates for the treatment of Duchenne muscular dystrophy (DMD). These muscle-selective promoters have been designed using Synpromics’ PromPT technology and bioinformatics expertise and will be evaluated by Solid Biosciences for optimal product specific gene control.
David Venables, CEO of Synpromics, said: “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 programme in Duchenne.
The collaboration between the scientific teams will enable the effective and rapid in vivo evaluation of the candidate promoters as compared to industry standards.”
Dr Joel Schneider, Chief Technology Officer and Head of Exploratory R&D at Solid Biosciences, said: “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. 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.”
DMD is a genetic muscle-wasting disease that is progressive, irreversible and ultimately fatal. DMD affects approximately one in every 3500-5000 live male births. Symptoms of DMD usually manifest between three to five years of age.
As the disease progresses, patients are typically wheelchair-bound by their early teens and succumb to respiratory or heart failure in early adulthood. There is no cure for DMD and for the vast majority of patients, there are no satisfactory treatments.