Santhera Signs a Pre-Clinical Research Agreement with Biozentrum to Advance Gene Therapy for Congenital Muscular Dystrophy

Santhera Reports Submission of Marketing Authorization Application to the EMA for Puldysa (Idebenone) in Duchenne Muscular Dystrophy


  • Santhera enters into a collaboration with Biozentrum, University of Basel to explore the feasibility of the gene therapy by standard viral vectors for LAMA2 MD or MDC1A, co-financed by Innosuisse of ~$1.19M
  • The focus of the collaboration is to foster Santhera’s neuromuscular pipeline and to utilize Biozentrum’s gene therapy in its Omigapil for improving its benefits in LAMA2 deficient MD
  • Biozentrum’s gene therapy utilizes two linker proteins derived from agrin, laminin and nidogen leading to a restoration of muscle fiber, recovery of muscle force and size and increases body weight. Omigapil is a deprenyl-analog, exclusively licensed from Novartis by Santhera for CMD

Click here to read full press release/ article | Ref: Santhera | Image: Pinterest