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
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
Clinical trial — data collection study
Execution and management of clinical trials focused on structured data collection for investigational therapies.
Clinical trial with external facility management
Management of clinical trials that use external complex facilities and partner site networks.
In‑house clinical trial operations (no external facilities)
End‑to‑end trial conduct using company‑operated clinical sites without reliance on external facilities.
Post‑marketing study management
Design and execute Phase IV or post‑approval safety and effectiveness studies.
Rare disease study and protocol design
Specialized protocol development tailored for small populations, including alternative statistical approaches and patient‑centric endpoints.
Subject screening and enrollment services
Participant screening, eligibility assessment, and enrollment coordination including remote pre‑screening workflows.
Clinical trial — data collection study
Execution and management of clinical trials focused on structured data collection for investigational therapies.
Clinical trial with external facility management
Management of clinical trials that use external complex facilities and partner site networks.
In‑house clinical trial operations (no external facilities)
End‑to‑end trial conduct using company‑operated clinical sites without reliance on external facilities.
Post‑marketing study management
Design and execute Phase IV or post‑approval safety and effectiveness studies.
Rare disease study and protocol design
Specialized protocol development tailored for small populations, including alternative statistical approaches and patient‑centric endpoints.
Subject screening and enrollment services
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