RareLabs is a precision medicine creation lab for patients, families, and foundations facing a rare disease with no approved treatment. We build a living, personalized model of the disease from the patient's own cells, then use it to test different genetic technologies, and potentially thousands of existing treatment possibilities in parallel. The goal is simple: find a credible path forward as quickly as the science allows.
Scientific promise
We provide answers to patient questions that the traditional medical system can't or won't answer. We run a science-first, patient-focused drug development laboratory with urgency, discipline, and clarity to help patients move forward in their journey.
We can't ensure every answer for all patients, but what we do provide is a disciplined scientific process that is transparent about what the science shows.
If your doctor has said “there's nothing approved for this,” or a diagnostic odyssey has gone on for years with no clear answer, you are exactly who RareLabs was built for.
Patients and families living with a rare disease genetic diagnosis and no available therapy.
Patients still searching for a genetic answer — the so-called “under diagnosed,” or those with a non-genetic diagnosis like autism.
Physician-scientists and foundations looking for a serious lab partner.
Advocacy groups running N-of-1 and N-of-few programs for their communities.
You don't have to wait for the system to catch up. We meet you where you are and start from there.
You own the data. You own the IP. RareLabs builds the scientific roadmap, tools, and treatment hypotheses that make action possible.
Most rare diseases have no drug, no model, and no roadmap. RareLabs builds the missing scientific roadmap, tools — and builds treatments when the science shows it’s possible.
Step 01
From a small blood or skin sample, we create induced pluripotent stem cells (iPSCs) — a Nobel Prize-winning technology that lets us grow the exact cell type affected by the disease from the patient’s own biology.
Brain cells. Heart cells. Liver cells. Whatever your disease needs.
We also create a CRISPR-corrected "cured" version of those cells, so we have a healthy baseline to compare against.
We generate your personal Amenability Study through our RINAE AI engine and our team of scientists to create a deep understanding of the disease, as well as a roadmap for developing potential treatments.
Step 02
With your toolbox and plan ready, we run treatment discovery in our modality agnostic lab, which may include one or more of:
A repurposed drug screen, using high-speed automation to evaluate existing therapies.
A custom ASO campaign, an RNA medicine designed and tested for your specific mutation.
A gene therapy program, which can edit, replace, or supplement the defective gene.
We often run these experiments in parallel. Every week spent running one experiment at a time is a week lost.
Step 03
You get clear, plain-language updates. Your physicians stay in the loop. Collaborators, foundations, and funders get what they need to move the work forward.
We help turn the science into a story others can understand and act on.
Traditional rare disease R&D runs in series: one experiment, one answer, then the next. For a patient whose disease is progressing, serial doesn't work. RareLabs runs in parallel. We test repurposed drugs, ASO medicines, and gene therapy approaches against the same disease model at the same time. If something is going to work, we want to know sooner, not later.
Across eight patient programs, RareLabs has already created or identified promising treatment candidates where no approved option existed. Some candidates came from drugs already approved by the FDA. Others were designed from scratch for a single patient's mutation.
We don't promise an answer in every case. We do promise an honest scientific process run with urgency, discipline, and transparency.
RareLabs is a division of AlphaRose Therapeutics, a public benefit corporation, focused on scaling ultra rare genetic medicines for suffering patients, utilizing its proprietary AI, ASO platform, and translational expertise.
Every RareLabs patient and family receives a working program: scientific assets, clear communication, and a dedicated point of contact from intake through final report.
A personal iPSC cell line, biobanked for future use.
A CRISPR-corrected isogenic control line.
Amenability Study — your in-depth roadmap of the disease and potential therapeutic strategies.
A disease-specific assay designed around how your mutation actually behaves.
A repurposed drug screen focused on existing treatment possibilities.
Preclinical proof-of-concept ASO treatment (RNA therapy, multiple chemistries available).
Gene therapy (multiple vector types available).
Other modalities (on a case-by-case basis).
Monthly progress updates in plain language.
A dedicated project manager from intake to final report.
Timeline
Approximately 12–15 months
First step
Start with a conversation
When a patient or family reaches out, we begin by understanding the case, reviewing what is already known, and outlining the most sensible next step.
We can generate new treatments and IP for your genetic disease of choice fast and efficiently. Because time is life.
Patient-derived iPSC disease models for genetic indications.
Isogenic CRISPR-corrected controls for cleaner data.
Real-world data from our patient and family network.
ASO treatment programs.
Gene therapy treatment programs.
Repurposed drug screens for new formulation pathways.
If you're building a program in a rare or ultra-rare indication and need a partner who can move with urgency, we should talk.
Partner with usFor patients and families living with a rare disease, waiting is never neutral. Disease progresses. Childhoods pass. Adult lives narrow. We exist so “there's nothing we can do” is never treated as the end of the conversation.
You don't have to wait. Let's start building the right path for your treatment.
Patient-first. Plainly scientific. Honest about what the science can support.
When there is no treatment, we build the science needed to create one — rigorously, in parallel, and with the urgency patients and families deserve.
Whether you are a patient, family, physician-scientist, foundation, or biopharma partner, RareLabs can begin with a focused conversation about rare disease treatment development, clinical trial readiness, compassionate use strategy, and patient access decisions.
Direct contact path
Email [email protected] to start the conversation.
For the most direct handoff, contact RareLabs at [email protected]. If you prefer to stay on the site, you can also use the secure intake form below and the request will still be captured for follow-up.