Trial updates and gene therapy

Thank you for your continued support and encouragement. I would like to update you about our progress on the therapeutic development for Wolfram syndrome.

Our clinical trial of dantrolene sodium for adult and pediatric patients with Wolfram syndrome has been going well. Based on the results of dantrolene trial, I feel confident that counteracting ER stress (a type of cell stress causing Wolfram syndrome ) is beneficial for patients with Wolfram syndrome. However, we need a better drug that can efficiently counteract ER stress. To achieve this goal, I have been working on two new drugs targeting ER stress. Preclinical data (i.e., data using cell and animal models) and the safety profile of both new drugs look very good. Thus, I am planning a multi-center trial using one of these drugs. My goal is to make this happen in the next 12-24 months. I will keep you updated on this.

Another priority for me is to develop gene therapy for vision impairment in Wolfram syndrome. We have created the adeno-associated virus (AAV: a safe virus utilized for gene therapy) that can deliver normal Wolfram gene (WFS1) and a regeneration factor, MANF, into retinal cells. We got an encouraging result several weeks ago. One of our new types of viruses could introduce MANF into retinal ganglion cells efficiently in a mouse model of Wolfram syndrome. I would like to accelerate the pace of this project.

As always, please feel free to contact me with any questions or concerns ( I would like to know what you think and how you feel. Thank you again for your continued support.

With gratitude,
Fumi Urano