Uncommon Cures
Clinical research organization focused on designing and running patient‑centric clinical trials for rare and orphan diseases. Services emphasize rare‑disease-specific trial design, consolidation of site and CRO functions, home‑based and pediatric trial operations, and partnerships to expand geographic reach and regulatory pathways.
Industries
N/A
Nr. of Employees
small (1-50)
Uncommon Cures
Chevy Chase, MD; Washington, DC (contact page)
Products
Clinical trial: mRNA‑based therapy for OTC deficiency
A site‑run clinical study evaluating an investigational mRNA therapeutic for ornithine transcarbamylase (OTC) deficiency, delivered via lipid nanoparticle technology; includes multiple infusion visits and follow‑up safety assessments.
Pediatric clinical study for Friedreich’s ataxia
A pediatric study evaluating an investigational therapeutic for Friedreich’s ataxia with remote screening, short on‑site stays and at‑home nurse follow‑up.
Clinical trial: mRNA‑based therapy for OTC deficiency
A site‑run clinical study evaluating an investigational mRNA therapeutic for ornithine transcarbamylase (OTC) deficiency, delivered via lipid nanoparticle technology; includes multiple infusion visits and follow‑up safety assessments.
Pediatric clinical study for Friedreich’s ataxia
A pediatric study evaluating an investigational therapeutic for Friedreich’s ataxia with remote screening, short on‑site stays and at‑home nurse follow‑up.
Services
Execution and management of clinical trials focused on structured data collection for investigational therapies.
Management of clinical trials that use external complex facilities and partner site networks.
End‑to‑end trial conduct using company‑operated clinical sites without reliance on external facilities.
Design and execute Phase IV or post‑approval safety and effectiveness studies.
Specialized protocol development tailored for small populations, including alternative statistical approaches and patient‑centric endpoints.
Participant screening, eligibility assessment, and enrollment coordination including remote pre‑screening workflows.
Execution and management of clinical trials focused on structured data collection for investigational therapies.
Management of clinical trials that use external complex facilities and partner site networks.
End‑to‑end trial conduct using company‑operated clinical sites without reliance on external facilities.
Design and execute Phase IV or post‑approval safety and effectiveness studies.
Specialized protocol development tailored for small populations, including alternative statistical approaches and patient‑centric endpoints.
Participant screening, eligibility assessment, and enrollment coordination including remote pre‑screening workflows.
Expertise Areas
- Rare disease clinical trial management
- Patient‑centric site design and operations
- Adaptive and N‑of‑1 trial methodologies
- Pediatric clinical research
Key Technologies
- Adaptive trial design
- Master protocol frameworks
- N‑of‑1 (single‑patient) trial methods
- mRNA therapeutic evaluation
News & Updates
Discussion of an integrated CRO/site model to reduce trial timelines and costs for rare diseases, plus advocacy partnerships and novel trial designs.
Completed Series A financing and began operations as a rare disease clinical research organization.
Named a Chief Medical Officer with experience in rare disease clinical practice and trials.
Discussion of an integrated CRO/site model to reduce trial timelines and costs for rare diseases, plus advocacy partnerships and novel trial designs.
Completed Series A financing and began operations as a rare disease clinical research organization.
Named a Chief Medical Officer with experience in rare disease clinical practice and trials.