Pharma Focus Asia

CYTOO and AskBio Enter Research Agreement to Screen Gene Therapy Candidates for Rare Muscle Disorder

Tuesday, March 03, 2020

CYTOO, a leading drug discovery company on muscle disorders, today announced that it has entered into a research collaboration aimed at selecting a gene therapy candidate for a rare muscle disorder with Asklepios BioPharmaceutical, Inc. (AskBio), a clinical-stage, fully integrated adeno-associated virus (AAV) gene therapy company. Under the terms of the agreement, AskBio and CYTOO will work together to develop an AAV-based screening platform derived from patient cells. The goal of the collaboration is to screen and select a preclinical candidate capable of restoring a healthy phenotype from patient cells cultured in vitro.

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 physiology, contractile and metabolic functions of human muscle in vivo and allow infection by AAV-based gene therapy vectors targeting muscle.

Dr. Philippe Moullier, Chief Scientific Officer, AskBio Europe, said, “As a leader in the gene therapy space, the ability to quickly and efficiently screen potential therapeutic candidates will be invaluable. The expertise provided by CYTOO and the MyoScreen platform will potentially give us a better understanding of how those candidates perform in patient cells and improve efficiency throughout the R&D process.”  

Luc Selig, CYTOO’s CEO, added, “Gene therapy for muscle disorders is becoming a reality for patients and their families, and we are proud that AskBio has chosen our expertise to investigate a potential new treatment. We have developed MyoScreen as a laboratory model of patient-derived muscle that can be used to screen gene therapy candidates and QC clinical and commercial batches.”

The financial terms of the agreement were not disclosed. 

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