Passage Bio has just recently announced that Krabbe disease will be the focus of their third clinical trial program.
Krabbe Disease
Krabbe disease is a rare, inherited, neurodegenerative lysosomal storage disease. It is also known as globoid cell leukodystrophy. The condition destroys the myelin of nerve cells in the brain and the body’s nervous system, causing progressive damage.
The most common form of the disease is infantile Krabbe disease. Unfortunately, it is also the most severe. Infantile Krabbe disease usually manifests before 6 months. From there, progression is rapid and the median survival is only two years.
There are currently no treatments for the disease besides symptomatic supportive care. However, Passage Bio is excited by their new research program for this condition.
The Program
Passage Bio has been highly encouraged by their preclinical data for Krabbe disease and plan to initiate a clinical trial in 2020.
This investigation will be a global, multi-center, open-label, dose escalation study. It will investigate GTP-206, which is being developed in collaboration with the Gene Therapy Program at the University of Pennsylvania. The trial will evaluate the efficacy, tolerability, and safety of the therapy for infantile Krabbe disease.
The Gala
In addition to this new research endeavor by Passage Bio, the company also announced their support of KrabbeConnect’s recent Million Dreams Fundraising Gala held on May 31st.
KrabbeConnect is a foundation which provides information, resources, and support for patients and families affected by Krabbe disease. Additionally, they work to accelerate research for the condition by fostering collaboration and communication between patients, advocacy groups, industry, researchers, and government. This organization believes in a patient-centered model for care and aims to help ensure the patient voice is being heard by researchers in order to optimize outcomes. To learn more about KrabbeConnect, you can visit their website or contact them directly.
Their first ever gala was held in Minneapolis, Minnesota. It’s goal was to not only raise awareness, but provide a space for researchers, patients, and families to come together and engage with one another in person. The event featured food, entertainment, a silent auction, and more. All proceeds from the gala went to support KrabbeConnect’s mission.
Both Passage Bio and KrabbeConnect are excited for their future collaboration and supporting one another in their unified goal: to aid those suffering with this debilitating disease.
You can read more about this gala as well as the upcoming clinical trial for Krabbe disease here.
