Publications
Advancing Novel Treatments for Patients with Muscle Disease
Events / Presentations
American Society of Gene & Cell Therapy (ASGCT) 2024
Oral Presentation
Directed evolution of novel MyoAAV capsid variants enabling effective systemic muscle transduction while de-targeting the liver in non-human primates
Oral Presentation
Developing Highly Potent and Safe Muscle Gene Therapy Candidates by Combining Effective Liver De-targeted MyoAAV Capsids and Cell Type Specific Regulatory Elements
Poster Presentation
Developing a potent gene therapy candidate for facioscapulohumeral muscular dystrophy (FSHD) through high throughput AAV capsid and cargo engineering [495]
Poster Presentation
Highly effective expression of full-length Dysferlin protein in Bla/J mouse muscle after systemic MyoAAV administration [1489]
We are Focused on Delivering Life-Changing Therapies to Patients
We are applying technology platforms that address the key limitations of current gene therapies, including tissue-specific delivery and gene regulation.
