Yesterday, it would have taken 30 years and hundreds of millions of dollars to develop these treatments...
Today, we have the technology to develop treatments for many of these diseases in just a few years at a fraction of the cost.
Our goal is to develop genetic treatments quickly and sustainably, so more children like Rose have a chance to live a long, healthy life.
TCAR PHASE ONE
TCAR PHASE TWO
Creating a brighter future for children like Rose
Casey and his daughters, Rose and Weston, at the Everlum Bio Lab. Casey co-founded Everlum Bio in 2022.
DATA FOR OUR POTENTIAL NEW DRUG:
ANTISENSE OLIGONUCLEOTIDE TARGETING HNRNPH2 GENE EXPRESSION
We see in animal models that have an H2 mutation like Rose's, that many of their abilities can be rescued by knocking down the expression of the H2 gene. Once knocked down, H1 over expresses, bringing the much needed protein back to the brain. Our most recent screen in Rose's neurons have proved that the same thing happens in her cells once treated with our proprietary ASO design.
How a Treatment Today can Impact Generations to Come
Since the discovery of this HNRNPH2 mutation in 2016, ~130 individuals have been identified to suffer from it.
However, recent findings suggest there are closer to 10,000 people currently suffering worldwide, and 1:50,000 / 2800 children born every year with HNRNPH2.
A genetic treatment makes an impact forever.
With Your Help, a Treatment for Rose is on the Horizon.
What does that mean?
It means Rose could possibly make eye contact again, and connect with others.
It means she could play and interact with her Dad, her sister, and her Mom on a deeper level.
It means she could make new friends.
It could mean not being overwhelmed by the sounds of a grocery store, play area or an airplane. She could eat off her own plate instead of grabbing food from others.
A treatment could mean she can speak again, and let people know when she’s hungry, tired or sick - even if it's just a word or two.
A treatment could mean that Casey gets to hear his daughter say, "Daddy."