Finding Hope for Frizzle (FRRS1L) and Apertura Gene Therapy Announce License Agreement for the Development of Frizzle Gene Therapy Using TfR1 CapX
Watch the announcement in the video below.
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.
Join us on this journey of HOPE as we seek to CHANGE LIVES and CHANGE THE WORLD! Donate Now!
LEARN MORE ABOUT FRRS1L/TfR1 CapX
Watch a 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.
2026 the Year for Frizzle Treatment
But only with your support!
Our goal is to begin treating Frizzle patients by September of this year with a one-time gene replacement therapy.
We have a clear plan, committed partners, and a defined timeline. What remains is raising the funds needed to bring this treatment across the finish line and into patients.
We must raise $4 million to cover both the manufacturing of the drug and the costs of treating patients in a clinical trial.
Every donation goes directly toward changing lives and changing the world!
Pre-clinical Data shows Restoration of Function
Our pre-clinical research on FRRS1L mouse models showed the recovery of function through gene replacement therapy. The gene replacement therapy given to FRRS1L diseased mice worked to bring function back to FRRS1L diseased mice brains and bodies! This is life changing news! We want recover for Frizzle patients and are working to get treatment developed this year. Would you join us in making treatment a reality for sick Frizzle patients this year?
Donate today to help us fund treatment development for Frizzle patients!
QUICK LINKS
DONORS and PARTNERS
We are looking for donor partners and champions to help us get this treatment to the finish line and in patients by September this year! Learn how you can help here.
PHYSICIANS and RESEARCHERS
Researchers, doctors, and medical professionals looking for more information about Frizzle disease, published research, and treatment information, learn more here.
FRIZZLE (FRRS1L) FAMILIES
Are you a parent or caregiver of a Frizzle (FRRS1L) patient? Learn more here. Learn more about our special community and register your Frizzle child or children today here!
Treatment by September 2026 is possible with your support!
Our race to treatment has begun. We have a goal to begin treating Frizzle patients by Sept. 2026, but it will only happen with your support! Donate today to help fund the manufacturing of the drug that will be a one time injection for Frizzle patients!
All donations are tax deductible, and go directly to fund the development of FRRS1L gene therapy treatment. Your gift will help ALL children with FRRS1L. That is older children who have lost hope, younger children who haven’t yet regressed, and unborn children who will inherit this disorder. This research also moves forward the science that may result in cures for other genetic diseases.
It wasn't always like this for children with FRRS1L gene disorder.
Before the disorder took full effect, parents knew and saw their children to be busy toddlers. FRRS1L is a critical brain encoding gene that affects all cells in the brain. Children with this gene disorder develop, albeit delayed, until age two when they begin having seizural activity that causes regression and total loss of function.
FUNDRAISING UPDATE
Current Funding Need: $4 million
Goal 1: COMPLETED
GOAL 2: COMPLETED
GOAL 3: COMPLETED
Goal 4:
Fundraising Goal: $3 million to fund the manufacturing of the drug/treatment
Current Status of Goal: $1 million raised of $3 million goal
Goal 5:
$2 million for Clinical Trials to treat Frizzle children
Check out our Fundraising Plan for more details.
Within weeks children lose their ability to move, speak, eat, and control their body.
We see these children looking at a toy they once held. We know they want to play with it, but they physically can’t. They are trapped.
Learn about their Hope for treatment and how you can help!
Every day is a fight for these kids, but you can help them.
Watch the trailer and learn more about our One Dose Documentary. Follow along on our journey to develop lifesaving treatment for Frizzle patients around the world!