February 28, 2023
GEXVal Inc. is pleased to announce that we have initiated a collaboration project with FRAXA*-DVI** (FRAXA-Drug Validation Initiative) to investigate the efficacy of GXV-001 in a loss of function animal model for Fragile X syndrome (FXS***).
“We are excited to share that GXV-001 is highly evaluated by FRAXA and recommended to test the drug using various validated platforms at FRAXA-DVI. Very promising results in all behavioral indexes have been obtained in repeated-dosing study, indicating the potential of GXV-001 to target broad spectrum of symptoms found in patients with FXS”, said Dr Kohei Shimada, the Chief Scientific Officer of GEXVal Inc.
‘The preliminary data of GXV-001 from Fmr1 knock-out mice are intriguing. We are always encouraged by novel drug candidates like GXV-001 that is safe and improves the symptoms.’ said Dr Patricia Cogram, Associate Professor of Genetics at Institute of Ecology and Biodiversity (IEB), University of Chile, and the Principal Investigator of FRAXA-DVI.
Dr Michael Tranfaglia, Medical Director and Co-founder of FRAXA also commented ‘I am delighted to see another promising drug candidate for FXS. Look forward to the further investigation to strengthen the therapeutic profile of GXV-001.’
GEXVal Inc. is striving to address unmet medical needs in rare diseases and deliver innovative medicine to patients and their families who are awaiting better treatment options.
*About FRAXA Research Foundation
FRAXA Research Foundation is a national nonprofit organization founded in 1994. FRAXA's mission is to find effective treatments and ultimately a cure for Fragile X syndrome. FRAXA funds research at top universities around the world and partners with biomedical and pharmaceutical companies to bridge the gap between research discoveries and actual treatments. Treatments for Fragile X are likely to help people affected by autism, Alzheimer’s, and other brain disorders. For more information please visit https://www.fraxa.org.
The FRAXA Drug Validation Initiative (FRAXA-DVI) provides speedy, cost-effective, objective preclinical testing of potential Fragile X treatments. FRAXA-DVI uses in-vitro systems, behavior batteries, and gene expression and peripheral biomarker platforms to validate investigational new drugs and repurposed available compounds in Fragile X syndrome (FXS). FRAXA Research Foundation has funded this resource every year since 2011.
***About Fragile X Syndrome (FXS)
Fragile X syndrome (FXS) is the most common inherited cause of autism and intellectual disabilities. Fragile X syndrome occurs when a single gene on the X chromosome shuts down. This gene makes a protein needed for normal brain development. In FXS it does not work properly, the protein is not made, and the brain does not develop as it should. For more information on Fragile X syndrome visit https://www.fraxa.org/fragile-x-syndrome/.
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