BE part of our Miracle in 2025
As 2024 comes to an end, your generosity can bring us closer to a breakthrough in gene therapy for INAD.
Every dollar raised fuels the research needed to further develop our Gene Therapy Program. Together, we can take the next crucial steps toward hope and healing for our children and their families.
Take advantage of Year-End Giving benefits and Donate today to be part of our Miracle.
Making a Miracle Happen in 2025
From Generosity to Gene Therapy -
a Miracle can happen with INADcure!!
In collaboration with the INADcure Foundation, Jackson Laboratories' earlier research showed incredible promise in delivering the PLA2G6 gene to the brain using an AAV9 vector, leading to improved survival in treated mice.
Now, this new study will build on that success by testing a wider range of doses to assess safety and efficacy. Jackson Labs will track survival, body weight, and neurological function, analyze brain, spinal cord, and liver samples, and provide regular updates on progress.
This vital research costs $200K to complete. By funding this next step, we move closer to a clinical trial that could transform the future for children with INAD.
Jackson Laboratory & INADcure Foundation Partnership
A family’s mission: ‘Deep down, we need to know we’ve done everything we can’
