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AntiSense Oligonucleotides (ASOs), for the treatment of Wolfram syndrome by La Jolla Labs

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 Wolfram-related disorder.
  • The head of research at LJL presented a review of the potential use of ASO therapy for WS based on disease biology and different ASO strategies.
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WFS1 Gene Therapy to correct chronic inflammation due to Wolframin loss of function mutations

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 mice with Wolfram syndrome, researchers will study whether inflammation is reduced and whether the progression of Wolfram syndrome symptoms can be slowed by addressing/reducing inflammation.
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Clinical evaluation of Idebenone as a Potential Treatment for Wolfram Syndrome

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 stage and will last a minimum of 12-24 months
  • Data from the past several years, current data, and data going forward will be evaluated.
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Gene Therapy Treatment of Wolfram Syndrome

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 gene therapy to correct symptoms of Wolfram syndrome in a WFS1 knock-out model.
  • New steps in the project include ensuring that overexpression of WFS1 in cells will not impair this correction and validating the already reported promising effects of gene therapy in a knock-in mouse model that more precisely mirrors human WS.
  • This complete data set will help us to determine the efficacy of gene therapy for preserving vision in WS
  • This work will provide validation and efficacy data to move gene therapy closer to development for WS.
  • Corrected WFS1 will be delivered with a retina-specific vector, directly to the retina with direct injection to the eye in a WS knock-in (more similar to humans than a knock-out) mouse model.
  • Corrected WFS1 will be delivered systemically with a different vector, also in a knock-in mouse model, to determine if multiple organs (including pancreas and retina) can be treated in WS with a single treatment.
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From the Desk of Dr. Sarah Gladstone

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. Then there are moments when things finally come together: the data start to make sense, key questions are answered, and important milestones are achieved. We are now in a time of data coming together. What follows is a look back at the progress made in 2025, and an exciting look ahead at the possibilities now opening up for the year to come. Treating Wolfram syndrome is not a one-size-fits-all approach. The condition itself is not the same for everyone. Each person with Wolfram syndrome has a unique combination of symptoms based on their specific genetic variation and individual biology. Because of this, treatment may differ from person to person depending on age, genetic findings, and which symptoms are most prominent. Even though treatments may differ, the overall treatment strategy follows a clear and consistent path. The goals remain the same:

  • First, to slow the progression of cell stress and dysfunction;
  • Second, to prevent ongoing cell death;
  • Ultimately, to replace damaged cells with new, healthy cells.

Based on the expressed needs of the Wolfram syndrome community, current treatment efforts focus on the central nervous system (including the brain, retina, vision, and hearing), the pancreas (diabetes), and bowel and bladder function, while also addressing other associated symptoms. It has been an incredibly busy and fulfilling year…. and decade! We are profoundly thankful to our donors and supporters—you are the reason this work happens. Your generosity fuels every step forward, and we are so grateful to have you as part of this community. Because of you, we are moving forward with energy, hope, and momentum. The year ahead is shaping up to be an exciting one, and we can’t wait to celebrate and share our progress with you in 2026!

— Dr.Sarah Gladstone

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Dr. Fumihiko Urano Wolfram Updates

Dr. Fumihiko Urano Washington University School of Medicine, USA

January 31, 2026

Dr. Fumihiko Urano

Dear Friends,

I hope the new year has started well for you and your family. Thank you, as always, for being part of the Wolfram syndrome community. Your trust, patience, and partnership mean a great deal to us. Everything we do in the clinic, the clinical trial unit, and the laboratory is driven by a single purpose: to improve the lives of individuals and families living with Wolfram syndrome. Our shared goal is CURE4WOLFRAM, and every visit, study, and experiment moves us one step closer. I would like to share where we are now, what we are learning, and how these efforts are coming together as we move into 2026.

New Drugs and Supplements

We are actively developing a systematic platform to identify medications and supplements that may benefit individuals with Wolfram syndrome. Using patient derived induced pluripotent stem cells, we generate brain cells in the laboratory that closely reflect the biology of Wolfram syndrome. These cells allow us to directly test existing drugs, supplements, and new compounds to see whether they improve cell survival, reduce stress responses, support mitochondrial function, or restore healthier cellular balance. Our highest priorities include antioxidants, sigma 1 receptor agonists, NAD activators, idebenone, GLP-1 receptor agonists, and other compounds that target endoplasmic reticulum stress and mitochondrial dysfunction. We also have a long list of additional candidates based on scientific rationale and emerging evidence. This platform allows us to evaluate potential therapies before moving toward clinical studies. We plan to expand this effort and will continue to share updates as we learn more.

Read more

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