Finding Hope for Frizzle (FRRS1L) and Apertura Gene Therapy Announce License Agreement for the Development of Frizzle Gene Therapy Using TfR1 CapX
WHY THIS MATTERS
Finding Hope for Frizzle (FRRS1L), a parent-led organization, is excited to announce its partnership with Apertura Gene therapy to license their TfR1 CapX technology for Frizzle gene replacement therapy. The organization is funding and driving treatment forward for Frizzle disease and has a goal to begin a clinical trial in September of this year! This partnership has the opportunity to move science forward not just for Frizzle disease but for all neuro and rare diseases pursuing gene therapy.
Although Frizzle is a rare neurodevelopmental disease, 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.
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FRRS1L/TfR1 CapX
Recorded Webinar
Finding Hope for Frizzle has prepared a recorded webinar to share more information about the organizaton, Frizzle disease, supporting partners, and why we have partnered with Apertura Gene therapy to license their TfR1 CapX technology for our Frizzle gene therapy. Access the webinar to hear more from:
Chrissy Green, President of Finding Hope for Frizzle
Dr. Ben Deverman, Senior Director of Vector Engineering at the Broad Institute of MIT and Harvard, as well as the scientific founder of Apertura Gene Therapy and developer of the TfR1 CapX technology
Andy Holt, Chief Commercial Officer of Viralgen Vector Core
Please fill out the form here to access to the Finding Hope for Frizzle TfR1 CapX webinar.
For questions or difficulties accessing the webinar, please email: contact@FRRS1L.org.
Apertura Gene Therapy
Learn more about the TfR1 CapX techonology that Frizzle will be using for its FRRS1L/TfR1 CapX gene therapy.
TfR1 CapX
Frizzle (FRRS1L) Gene Therapy will be using the Apertura TfR1 CapX technology to deliver the FRRS1L gene to the brain. Apertura Gene Therapy has engineered a central nervous system (CNS)-targeting AAV capsid that binds to the human Transferrin Receptor 1 (TfR1). Apertura’s TfR1 CapX can cross the blood-brain barrier (BBB) and deliver therapeutic cargo throughout the central nervous system (CNS) via systemic administration