#USA #San Diego #Vanda Pharmaceuticals #CMT2S #ASO Therapy

Vanda Pharmaceuticals to Present Innovative Research at AAN 2025

Vanda Pharmaceuticals Inc., traded on Nasdaq as VNDA, has recently announced its participation in the upcoming American Academy of Neurology (AAN) Annual Meeting. This year's conference will be hosted in San Diego, California, from April 5 through April 9, 2025. Vanda is set to highlight its latest advancements in treatment for neurodegenerative diseases, specifically focusing on Charcot-Marie-Tooth disease type 2S (CMT2S).

Key Presentation Details

The notable presentation, scheduled for April 9, 2025, is titled "Translating IGHMBP2 Variants with a CMT2S Patient-specific Organ-on-a-chip Model Personalized Medicine ASO-based Therapeutic Rescue." This poster presentation will be delivered by Dr. Sandra Paulina Smieszek, who serves as the Head of Genetics at Vanda. The presentation will focus on VCA-894A, a novel therapeutic that has been customized for a specific genetic mutation related to CMT2S.

Breakthrough Therapy for CMT2S

The significance of VCA-894A lies in its targeted approach to tackle a specific genetic mutation in patients with CMT2S. The data shared in Dr. Smieszek's presentation will cover the results from using Hesperos' Human-on-a-Chip® neuromuscular junction model, which utilizes patient-derived cells. This innovative technology demonstrated notable improvements in neuromuscular function post-treatment, including reduced muscle fatigue and enhanced synaptic transmission between motor neurons and skeletal muscle.

This biopsic research methodology not only signifies a leap in precision medicine but also emphasizes the relevance and applicability of human models in evaluating treatment effects. VCA-894A has received orphan designation by the FDA and is expected to be administered soon to the specific patient it was developed for, representing a tailored and personalized approach to managing genetic disorders.

Unpublished Data and Future Directions

Additionally, Vanda's presentation will reveal novel unpublished data concerning conduction velocity, which will contribute to a deeper understanding of the therapeutic benefits of VCA-894A. The recent developments build upon Vanda's previously published work on ASO-based personalized treatment strategies for CMT2S, which can be explored in their recent publication: "Potential ASO-based personalized treatment for Charcot-Marie-Tooth disease type 2S" available under DOI 10.1016/j.omtn.2025.102479.

Vanda aims to leverage these findings to pave the way for more effective treatments addressing genetic diseases, emphasizing the urgent need met through such personalized therapeutic approaches in the field of neurology.

Learn More

For those interested in more detailed information about the annual meeting or Vanda Pharmaceuticals, additional resources can be found on Vanda's official website and the AAN’s website. The implications of Vanda’s findings resonate well beyond CMT2S, promising advancements in treating complex neurological conditions.

As the AAN Annual Meeting approaches, the anticipation builds not only around Vanda's innovative study but also around the potential it holds for reshaping treatments and improving patient outcomes in neurology. Stay tuned for updates as Vanda Pharmaceuticals continues to lead the way in biopharmaceutical innovation.

For inquiries, please contact Kevin Moran, Senior Vice President, Chief Financial Officer, and Treasurer at Vanda Pharmaceuticals, or refer to their dedicated communications team.