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WASHINGTON, April 7, 2025 /PRNewswire/ -- Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced participation at the American Academy of Neurology (AAN) Annual Meeting, to be held in San Diego, California from April 5 through April 9, 2025.
The following will be presented:
Presentation Title: "Translating IGHMBP2 Variants with a CMT2S Patient-specific Organ-on-a-chip Model: Personalized Medicine ASO-based Therapeutic Rescue"
Poster Presentation Session: P-12
Poster Number: 11-017
Presenter: Dr. Sandra Paulina Smieszek, Head of Genetics
This poster describes the data around a novel breakthrough therapeutic (VCA-894A) customized to a specific genetic mutation of a patient with Charcot-Marie-Tooth disease type 2S (CMT2S). The effects of VCA-894A were confirmed in Hesperos' Human-on-a-Chip® neuromuscular junction model with patient-derived cells where VCA-894A demonstrated significant improvements in neuromuscular function, including reduced muscle fatigue and improved synaptic transmission between motor neurons and skeletal muscle myotubes. The use of a microphysiological system to evaluate the effects of a genetically tailored treatment represents a significant advancement in precision medicine and the use of human relevant methods of evaluation.
VCA-894A has been granted an orphan designation by the FDA and it is expected to soon be administered to the specific patient for whom it was developed. The experimental platform described in this poster has the potential to unlock the development of treatments that can address significantly unmet medical needs based on genetic understandings and precision medicine.
Vanda has previously published an article titled "Potential ASO-based personalized treatment for Charcot-Marie-Tooth disease type 2S." In addition to the data published in the article, this poster will present some novel unpublished data on conduction velocity.
For access to the full article, visit DOI: 10.1016/j.omtn.2025.102479.
For more information on potential ASO-based personalized treatment for CMT2S, please see Vanda's prior announcement, available here.
For more information on the AAN Annual Meeting, please refer to www.aan.com.
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