Customer Success
It is an immense privilege to be entrusted with the task of helping to find cures for those effected by a rare diseases. Below are just a few examples of the work we are accomplishing for our customers and patients.
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Their Story
A family reached out to us about a year ago, after their daughter was diagnosed with Hereditary Spastic Paraplegia caused by a mutation in the SPG11 gene. Within the first year we had a pre clinical gene therapy that showed it halted the disease in its tracks.
Treatment
The current therapeutic we are working on is a gene therapy delivered by a Lenti Virus. We have cell line data showing the gene therapy works as expected. We are in the process of completing a mouse trial and then will start clinical trials with in the next 12 months
Time to Cure
9 months
In their words
"Everlume is great to work with, they moved faster then we ever thought possible"
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Their Story
Rose seemed to be developing normally, perhaps a bit slower than others - but nothing that would cause alarm. She took longer to learn how to crawl, walk, and talk, but she was a happy, growing baby. Her first word was "outside."
Before long though, Rose's parents knew something was wrong.She missed more and more milestones as she grew up. Rose would fall, but she wouldn't put her hands out to catch herself. In 2019, they received an official diagnosis: her HNRNPH2 gene was mutated, and her body wasn't producing the proteins necessary for her to develop properly.
Rose lost the few words she could say, and continues to lose her ability to communicate to this day. Her motor skills are impaired, and she struggles to feel safe and comfortable in overstimulating situations.Neurologists told Casey there was nothing they could do. There is no treatment for HNRNPH2.
Treatment
Everlume Bio designed and created 5 Antisense Oligos (ASOs) that showed efficacy in both Rose's cell lines and a humanized mouse model. Casey who was one of the Everlume Co-Founders has spun out another company working to commercialize the drug and ultimately get it in his daughter.
Time to Cure
24 months
In their words
"Working with Everlume has been an amazing experience. They don't waste time and get right into the lab working to build cures not just for Rosies, but for many children with life threatening conditions."
-Casey
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Their Story
Our Chief Science Officer, Rodney, has a close friend whose child died of DIPG, a rare form of childhood brain cancer with no known cure. The disease onsets at approximately 4 -6 years old. Children with this this cancer die with in 1-2 years.
Treatment
Everlume has developed an Antisense Oligo (ASO) that is injected into the brain and has been shown to kill the cancer in cell lines. We are currently testing our results in mice and plan on opening up a clinical trial in the next 12-18 months.
Time to Cure
12 months
In their words
"This cancer is one of the worst and I want to kill the bastard"
-Rodney
Time is life—every second counts in curing rare diseases.
Let’s take the first step together. We are on a mission to eliminate rare disease by 2035. We would love for you to join us.