Delving into the Latest Updates on Canine AAV8-MTM1 vector with Synapse

Overview

Basic Info

Drug Type

AAV based gene therapy

Synonyms

Target

MTM1

Action-

Mechanism

Myotubularin expression stimulants

Therapeutic Areas

Nervous System DiseasesSkin and Musculoskeletal DiseasesOther Diseases

Active Indication

Congenital Structural Myopathy

Inactive Indication-

Originator Organization

University of Washington

Active Organization

University of Washington

Inactive Organization-

License Organization-

Drug Highest PhasePreclinical

First Approval Date-

Regulation-

Author: Childers, Martin K. ; Williams, J. Koudy ; Rider, Branden E. ; Mitchell, Erin ; Grange, Robert W. ; Danièle, Nathalie ; Vignaud, Alban ; Jamet, Thibaud ; Rivière, Christel ; Furth, Mark E. ; Lockard, Mandy ; Mack, David L. ; Moullier, Philippe ; Doering, Jonathan A. ; Soker, Thomas ; Guan, Xuan ; Poulard, Karine ; Hammer, Caroline ; Van Wittenberghe, Laetitia ; Buj-Bello, Anna ; Joubert, Romain ; Mavilio, Fulvio ; Beggs, Alan H. ; Goddard, Melissa ; Moal, Christelle ; Barber, Jane ; Masurier, Carole ; Lawlor, Michael W. ; Martin, Samia

Intravenous injection of an adeno-associated viral vector expressing the myotubularin ( MTM1 ) gene improves survival and rescues skeletal muscle function in mice and dogs affected by myotubular myopathy.

100 Deals associated with Canine AAV8-MTM1 vector

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