“[This] is a demonstration of how these novel technologies can be leveraged to develop a new treatment paradigm – moving away from the one-treatment-fits-all perspective and towards the development of personalized medicines,"
Mihael H. Polymeropoulos, M.D.,
President &CEO Vanda Pharmaceuticals
Vanda Pharmaceuticals recently put out a press release on a study published in Molecular Therapy Nucleic Acids.
Study In a Sentence: This study describes the development of an ASO-based therapeutic for a patient with a rare variant of Charcot-Marie-Tooth (CMT) disease and the use of Hesperos' Human-on-a-Chip® neuromuscular junction (NMJ) model with patient derived cells to demonstrate rescue of the NMJ function within the in vitro model.
Key Impact: This study highlights the promising potential of using Human-on-a-Chip technology for personalized therapeutics for rare diseases.
Highlights:
- Therapeutic VCA-894A was developed for a patient with Type 2S CMT
- Researchers observed significant improvements in neuromuscular function in the NMJ model following treatment
- Findings from this study pave the way for further research into personalized therapeutics for CMT