Let's Change Lives & Change the World - TOGETHER! Finding Hope for Frizzle Gene Therapy Announcement скачать в хорошем качестве

Let's Change Lives & Change the World - TOGETHER! Finding Hope for Frizzle Gene Therapy Announcement

Finding Hope for Frizzle (FRRS1L), a parent-led organization, has partnered with Apertura Gene therapy to license their TfR1 CapX technology for its Frizzle gene replacement therapy. As a parent-led organization, that has control of its treatment program, we know our kids and patients can only get dosed once with a gene therapy, and we need it to be the best technology possible that can deliver our Frizzle gene to the whole brain. We also need it to be a route of administration that is less invasive and more easily accessible for our global patient population. TfR1 CapX does exactly that: it is dosed intravenously and can deliver genes throughout the whole brain. It is our goal to be first in human with Frizzle/TfR1 CapX by this September 2026. We have our plan and timeline set and partners are in place; however we still need funding to get this treatment to the finish line and in patients. Although Frizzle is a rare neurodevelopmental disease, a Developmental and Epileptic Encephalopathy (DEE), advancing this treatment into the clinical setting has implications far beyond a single disease. Progress in this space will not only support smaller rare disease communities, but also contribute valuable insights that may accelerate breakthroughs in more prevalent neurodegenerative conditions such as Parkinson’s and Alzheimer’s disease. We are seeking leaders and champions across these communities who are willing to step forward in support, recognizing that this effort has the potential to advance treatment development across the entire neurological disease and gene therapy development landscape. Join the organization on this journey of hope as they change lives and change the world. Learn how you can help at: www.FRRS1L.org ‪@broadinstitute‬