Invest in a child’s life
Help bring ultra-rare genetic disease research to life.
The state of gene therapy is moving forward at a rapid pace. As an effective means for delivering corrected genes to targeted tissues and cells, AAV gene therapy is transforming medicine and turning hope into reality. Cultivating the help of industry sponsors, the research community and caring individuals, we can ensure ultra-rare children get the same access to research and innovative science as indications with larger patient populations.
Founding trustees accelerate programs aimed at eradicating devastating genetic diseases. Through a one million dollar commitment, these industry and philanthropic leaders help us change the ultra-rare disease gene therapy landscape.
Providing an annual gift of $150,000 – $500,000, Rare Child Champions will directly support life-altering gene therapy treatments with the potential to save child’s life, no matter how rare the disorder.
When a child is eligible for clinical access to a life-saving therapeutic, a $25,000 gene flight sponsor erases physical and financial barriers for children and their families.
Directed research sponsorships advance gene therapy candidates with immediate promise and support for children to access treatment. Giving levels are $25,000 – $100,000.
Family support program
We provide the resources and assistance to alleviate the burden associated with caring for ultra-rare children. Our family support programs include:
- Access to the most current ultra-rare genetic disease research
- Clinical trial notification and eligibility assistance
- Travel and lodging assistance for trial participation or to see medical specialists
- Disease-related medical and advocacy resource
Become a Gene Flight Sponsor
If your child needs care from a medical specialist or is participating in a CCF clinical trial, the Gene Flight program provides equitable access to all children. We want to make sure as many children as possible can benefit from the best possible care and to participate in clinical research without any limiting factors.
Every donation to help a child with an ultra-rare genetic condition makes an immediate difference.