Our mission is to accelerate customized genetic treatments for millions of children affected by severe genetic diseases.
Our daughter, Lydia, was born with a genetic disease that will cause severe disabilities. There are millions affected by the long-tail of genetic diseases. We can save Lydia and others like her with personalized (N-of-1) genetic treatments.
Lydia was born a year ago, and started having seizures soon after birth. After 3 weeks in the hospital, we discovered a spontaneous mutation in a critical gene called KCNQ2 that controls brain function. She will be severely disabled. Mentally she will never progress beyond a few months of age. Physically she will never crawl, walk or talk. We are devastated but we now understand that we can still change her trajectory. Lydia’s diagnosis was very early; her brain is still plastic.
Lydia is not alone. There are millions affected by one-off genetic mutations. This a classic long-tail problem, individually each is rare, but collectively there are many. There is a technology called Antisense Oligonucleotides (ASO) that is extremely effective at silencing these mutations at the source, but we need a platform driven approach to customize a drug for each patient.
We have started work on a customized ASO which will be delivered to Lydia this year and set up Lydian Accelerator to help accelerate this platform for others.