The Rare Disease Lab
Built for Families and Foundations.
Let’s begin your journey towards a treatment today!
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We Cure Rare Diseases
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At Everlume we circumvent the slow, traditional process with precision medicine.
Using a personalized approach, we deliver swift, custom therapeutics for rare diseases impacting one out of seven families. With our novel cure plan, we’re committed to giving our customers a transparent path as we work to cure the disease.
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The Problem
Traditional research and pharmaceutical routes are slow and expensive. Our north star for Everlume is to free parents from this frustrating cycle and lead them to real solutions. Why? Because, we’ve been those parents.
If you are a parent of child with a rare disease you might have experienced…
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Spending significant time money without seeing progress
Feeling overwhelmed by the complexity and lack of clear guidance
Endless waiting for test results and treatment options
Losing hope as your child’s condition worsens while waiting for answers
The Approach
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Direct to Patient
We took the with our precision approach to rare diseases, we give parents and children a better way to heal and a clear path for testing and treatment. This means, parents and families don’t need to be scientists, business owners, researchers and project managers.
Modality agnostic approach
No que policy
Ownership of data
Built for speed
How It’s Done
Step One
Toolbox Development
Cure Plan
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It all starts with the Everlume Cure Plan. Everlume not only uses its proprietary AI platform, but we also work with the worlds leading scientists on your disease to create a robust plan on how to best attack the disease. We takes a multi-modality approach meaning that we let the disease dictate the best path to a cure whether it be a Gene Therapy, ASO, Small Molecule, or something else. When this step is missed it can lead to wasted efforts, and when it comes to many rare diseases, time is life.
Cell Lines
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iPSCs enable screening of potential drug candidates, accelerating drug discovery for rare diseases. By observing how different compounds affect patient-derived cells, our researchers can identify novel therapeutics and repurpose existing drugs for rare disease treatments. Studying iPSCs derived from patients with rare diseases can help identify key targets and pathways involved in the disease.
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Animal Models
Everlume is working towards a world where animal models are unnecessary, but currently animals models are still required by the FDA, and an important part of drug discovery. Animal models ensure that the created therapeutic not only does the intended job when delivered to a whole biological system not just cell lines. These series of experiments also ensure the drug is not toxic or have off targeting effects.
Step Two
Rapid Proof Concept
Assay development: Everlume designs a series of experiments that allows us to determine if the created therapeutic is effective and how effective it is.
Gene therapy: Similar to ASO’s a gene therapy adds a functional gene in coding a functional protein.
ASO design, screening, validation: ASOs are DNA OR RNA molecules that are able to modulate the gene that we will design and then screen in cell lines
Small molecule and repurpose drug screening: Many times mitigating symptoms through a repurposed drug can radically change a patients life
Step Three
FDA Approval & Distribution
For many of our patients they will receive the therapeutic during an N=1 FDA trial. This is a special mechanism the FDA has created for Rare Diseases. If the drug is commercializable then we will take it through full FDA approval and work to get it covered by insurance.
The Solution
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With Everlume Bio, you don’t get generic. Our patients get immediate access to customized treatment options built for them.
You get a dedicated team passionate about recovery and transparency that every parent and child deserves.
Our team has developed over 100 potential therapeutics for patients, including antibodies, gene therapies, ASOs, and small molecules. Impressively, 17 of these have progressed to Clinical Trials.
Success Stories
It’s Time to Take Action
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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.
Or schedule a call with our team to meet us today.
Join Our Community
We are on a mission to eliminate rare disease by 2035. We would love for you to join us.