Synpromics Announces Gene Therapy Research Partnership with Solid Biosciences
News Sep 20, 2017
Sciad Newswire / Synpromics, announces 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 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.
“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,” David Venables, CEO of Synpromics, commented. “The collaboration between the scientific teams will enable the effective and rapid in vivo evaluation of the candidate promoters as compared to industry standards.”
“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, Head of Exploratory R&D 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.”
Duchenne muscular dystrophy (DMD) is a genetic muscle-wasting disease that is progressive, irreversible and ultimately fatal. DMD affects approximately one in every 3,500 to 5,000 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.
This research collaboration will help advance Solid’s programme to develop optimal product candidates for the company’s AAV muscle-directed gene therapy research programme.
This article has been republished from materials provided by Synpromics. Note: material may have been edited for length and content. For further information, please contact the cited source.
First Successful Gene Therapy Trial Reported for People with Hemophilia ANews
Patients with hemophilia A who received a single infusion of an investigational gene therapy showed improved levels of the essential blood clotting protein FVIII, with 11 of 13 achieving normal or near-normal FVIII levels.READ MORE
Some Immune Cells May Help Tumors Instead of Destroying ThemNews
New data shows that neutrophils promote tumor progression and can actually hamper the work of immunotherapy in lung cancer.READ MORE
Cancer Gene Plays Key Role in Cystic Fibrosis Lung InfectionsNews
Recent findings explain why people with cystic fibrosis are particularly prone to respiratory infections—and suggest a new approach to treatment.READ MORE