Pfizer and CYTOO develop DMD target discovery platform

The research collaboration will aim to modify CYTOO’s existing MyoScreen platform to enable its potential use as a Duchenne muscular dystrophy target discovery platform

CYTOO has entered into a research and option agreement with Pfizer to develop a target discovery platform for Duchenne Muscular Dystrophy (DMD), through modifying CYTOO’s existing MyoScreen platform.

DMD is a rare and life-threatening genetic disorder that affects children approximately 1 in 3500 to 5000 boys and families. It is caused by mutations in the dystrophin gene that results in progressive muscle degeneration and weakness.

By the early teens, most individuals with DMD have lost the ability to walk unassisted and their heart and respiratory muscles have also weakened. Individuals with DMD usually die from cardiomyopathy and respiratory failure in their second decade of life.

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.

Under the terms of the agreement, Pfizer and CYTOO will work together to develop and validate a target discovery platform using a DMD patient muscle-derived MyoScreen platform. The goal of the collaboration is to attempt to establish a robust in vitro system that may be used for a high throughput target identification screen.

Should such a system be 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, VP, 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 CEO, 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.”

The financial terms of the agreement were not disclosed.

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