FRAXA Drug Validation Initiative (FRAXA-DVI) Provides Preclinical Testing of Potential Fragile X Treatments
Patricia Cogram, PhD
Principal Investigator
IEB, Fundacion Ciencia y Vida
Santiago, Chile
Robert Deacon, PhD
Co-Principal Investigator
University of Oxford
Oxford, United Kingdom
Paulina Carulo, MD
Collaborator
FLENI Institute
Buenos Aires, Argentina
2026 Grant Funding: $80,000
Continuously Funded by FRAXA Since 2011
Summary
FRAXA-DVI is a cost-effective, streamlined preclinical platform at the Fundacion Ciencia y Vida in Santiago, Chile for assessing the potential of investigational new drugs and repurposed compounds to treat Fragile X. It is the cornerstone of FRAXA’s community-based drug development program to accelerate the discovery of effective Fragile X syndrome (FXS) treatments.
Spearheaded by FRAXA Research Foundation in 2011 and funded every year since, including $80,000 in renewed support for 2026, FRAXA-DVI is one of FRAXA’s longest-running and most impactful research initiatives. It has evaluated hundreds of potential treatments, enabling major clinical development programs, including some now in Phase 3 clinical trials.
Virtually every investigational new drug in development for Fragile X has been tested at FRAXA-DVI.
Working with FRAXA-DVI
At FRAXA-DVI, scientists run dozens of experiments on compounds, evaluating their potential to correct Fragile X-related deficits. Some drugs tested here were discovered through FRAXA-funded research, while others originated from pharmaceutical pipelines for different conditions and show promise for Fragile X.
The team is led by Dr. Patricia Cogram, Associate Professor of Genetics at Fundacion Ciencia y Vida, Santiago, Chile, whose pioneering work has made FRAXA-DVI a critical resource in the global Fragile X research pipeline. Dr. Robert Deacon has over forty years of experience in rodent behaviour as the head of the rodent behavior unit at Oxford University. Collaborator Dr. Paulina Carulo leads a Fragile X clinic in Argentina, and this direct connection with individuals and families affected by Fragile X provides crucial insights.
See FRAXA-DVI in action in our feature story Inside the FRAXA Drug Validation Initiative, which shows how the program evaluates potential Fragile X treatments and helps identify the most promising paths forward.
Interested in collaborating or supporting our work?
Contact Patricia Cogram at FRAXA-DVI or Michael Tranfaglia at FRAXA to explore how we can advance Fragile X treatments together.
We have run hundreds and hundreds of compounds through FRAXA-DVI over the years. That experience has helped us identify which pathways are most important in Fragile X. The changes aren’t just in behavior, but also in the brain’s connectivity, giving us a clearer picture of which treatments are most promising.
Patricia Cogram, PhD
Director, FRAXA-DVI
The FRAXA-DVI Process: How We Test Potential Treatments
The FRAXA-DVI process takes just six weeks, offering a streamlined way to evaluate potential Fragile X treatments in a well-established preclinical model and helping identify which compounds deserve deeper investigation.
Identifying Promising Compounds
Candidate drugs come from academic researchers, pharmaceutical companies, and FRAXA-funded projects.
Preparing Fragile X Mouse Models
Testing is conducted on FMR1 knockout mice, the gold-standard preclinical model for Fragile X.
Establishing a Baseline
Before treatment, mice undergo behavioral testing to assess their initial cognitive and physiological state.
Administering the Drug
The candidate compound is given to the mice over a specific timeframe, with careful monitoring of:
- Daily responses
- Side effects
- Weight changes
Behavioral, Blood & Brain Analysis
Statistical evaluation determines whether the compound positively impacts Fragile X symptoms:
- Behavioral tests to measure learning, memory, and aberrant behaviors
- Biomarker studies
- Post-mortem brain analysis for protein and gene expression changes
Translating Research to Action
A comprehensive report is compiled and reviewed by FRAXA in collaboration with pharmaceutical and academic scientists, to determine next steps:
- Should the drug progress toward clinical trials?
- Is additional testing needed?
Updates from FRAXA-DVI
GEXVal Completes FRAXA-DVI, Receives FDA Orphan Drug Designation, and Prepares for Phase 2a Trial
Orphan Drug Designation positions GXV-001 for next steps after FRAXA-DVI completion as GEXVal prepares a Phase 2a Fragile X trial in Europe.
Nospharma Announces Partnership with FRAXA to Test NOS-01 in Pre-Clinical Models of Fragile X
NOS-01 enters the FRAXA Drug Validation Initiative (FRAXA-DVI) for Fragile X mouse-model testing, generating data to guide development decisions.
BK Channel Openers: From FRAXA Seed Funding to Big Pharma Investment and Trials
See how FRAXA’s early grants propelled Fragile X research from lab discoveries to industry momentum, with partners advancing therapies into trials.
BK Channel Openers: A New Hope for Fragile X Treatment – Insights from Kaerus Bioscience CEO Robert Ring
Kaerus Bioscience’s BK channel openers for Fragile X syndrome are advancing through Phase 1 trials, offering hope for new treatments with FRAXA’s continued support.
Marvel Biosciences Partners with FRAXA to Test MB204 for Fragile X Syndrome
Marvel Biosciences and FRAXA Research Foundation are collaborating to test MB204, a promising new treatment for Fragile X which targets adenosine receptors.
Fragile X Treatment Target Emerges from Neurolixis & FRAXA Collaboration
FRAXA pharma partner Neurolixis has filed patents for treatment of Fragile X with a highly selective new drug, NLX-101, which targets the serotonin receptor 5-HT1A.