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Aims to develop a target discovery platform for Duchenne Muscular Dystrophy
September 6, 2018
By: Kristin Brooks
Managing Editor, Contract Pharma
CYTOO and Pfizer Inc. have entered a research collaboration to modify CYTOO’s existing MyoScreen platform to enable its potential use as a Duchenne muscular dystrophy (DMD) target discovery platform. Financial terms were not disclosed. DMD is a rare and life-threatening genetic disorder caused by mutations in the dystrophin gene that results in progressive muscle degeneration and weakness. CYTOO has developed a muscle-on-a-plate platform using patients’ primary cells, called MyoScreen. MyoScreen is an in vitro system in which skeletal muscle cells mimic the morphology, contractile and metabolic functions of human muscle in vivo and therefore allows analyses of the molecular mechanisms involved in such functions in health and disease. Pfizer and CYTOO will work together to develop and validate such a target discovery platform using a DMD patient muscle-derived MyoScreen platform. The collaboration aims to establish a robust in vitro system that may be used for a high throughput target identification screen. If a system is developed, Pfizer will have an option to acquire a license for the use of the resulting platform for DMD target identification efforts. Dr. John Murphy, vice president, Biology, Pfizer’s Rare Disease Research Unit, said, “Although the genetic cause of DMD has been known for years, little is known about the molecular functions that are affected in DMD muscles. Pfizer is committed to early stage DMD research and target identification.” Luc Selig, CYTOO’s chief executive officer, said, “Muscle dystrophies affect children severely, and we still don’t know what happens in muscle. This is why we developed MyoScreen: to have a laboratory model of patient-derived muscle, to study and modulate muscle functions and to identify drug candidates that are specific to muscle function. Teaming with Pfizer on DMD means they have shown a great dedication to this project, and we are extremely motivated to succeed.”
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