Principal Investigator Dr.Benjamin Delprat, Montpellier, France With help from the WS patient community and a special shout-out to our dear friend Nufar, a list of 30 widely available and potentially therapeutic compounds was compiled to be evaluated for the potential treatment of WS. We are completing negotiations to test these compounds individually and in combination […]
Principal Investigators Dr. Lucas Fernandez Brillet, and GemaEsteban Bueno, MD, Barcelona, Spain Broad categories of neurodegenerative findings in WS have been proposed and established through prior work done by WS researchers. This project, proposed by Dr. Lucas Brillet, aims to determine in more detail the areas of the brain that are affected in WS, and […]
Principal Investigator Dr. Mariana Igoillo-Esteve, Co-Investigator Ane Olazagoitia-Garmendia, Brussels, Belgium New potential WFS1 interactors were discovered by Dr. Igoillo-Esteve in 2025. This work aims to validate the new interactome data obtained in 2025, uncover the molecular pathways in which they are involved, and characterize their roles in cellular function, ER stress, and survival. Following this […]
Principal Investigator Dr. Samagya Banskota, Boston, USA This project will attempt to make gene editing available to all WS patients who have a variant that involves a single base pair change (missense variant). Over 60% of people with WS with documented genotypes may benefit from this strategy. This approach is important because a single attempt […]
The Snow Foundation has partnered with Odylia Therapeutics to bridge the gap between preclinical/bench research and clinical trials. Odylia provides expert guidance and advice to take potential treatments from the lab to clinical trial and accelerate treatment development. We will continue to work together to forge a path forward for treatments like gene editing and […]
La Jolla Labs, USA Members of The Snow Foundation met with representatives from La Jolla Labs to investigate the role of RNA directed gene therapy, specifically AntiSense Oligonucleotides (ASOs), for the treatment of Wolfram syndrome. La Jolla Labs undertook an investigation to determine the potential for ASO application, specifically for the treatment of autosomal dominant […]
Principal Investigators- Drs. Raniero Chimienti and Giulio Frontino, Milan, Italy This work is done in collaboration with Telethon Foundation, Italy This project is designed to test whether immune cells that have been genetically corrected can help improve the inflammatory symptoms of Wolfram syndrome in a mouse model. By transplanting genetically corrected WS immune cells into […]
Extending into 2026 Principal Investigator-Felipe Chicani, MD, Sao Paolo, Brazil This patient comparison study will evaluate WS patients who have been treated with idebenone compared to an untreated control group to determine if idebenone can help slow the progression of WS symptoms, with a focus on vision loss. This project is currently in the planning […]
Principal investigator Dr. Cécile Delettre-Cribaillet, INM, Montpelier, France Extending into 2026: This work will be done in conjunction with a grant from the Be a Tiger Foundation This is an ongoing project evaluating and validating the potential for WFS1 wildtype gene transfer as atreatment for Wolfram syndrome. Preclinical data have already shown the ability of […]
Chief Medical and Scientific Officer From the Desk of the Dr. Sarah Gladstone, CMO/CSO As we enter 2026 and look back on 2025, it’s remarkable how much progress has been made in such a short time.Scientific research can be challenging—there are periods when progress feels slow and frustrating, and answers seem just out of reach. […]