Rare Disease Consultancy

Overview

CortexBio delivers specialised consulting services for organisations developing therapies for rare and ultra-rare diseases, providing strategic guidance throughout the development journey. Our approach combines scientific expertise with practical experience navigating the unique challenges of rare disease drug development.

Our Approach

We offer comprehensive support tailored to the specific needs of rare disease therapeutic development. Our services include:

·      Rare Disease Strategy Development: Create comprehensive approaches that address scientific, clinical, regulatory, and commercial considerations unique to rare diseases

·      Orphan Drug Designation Support: Navigate regulatory pathways and prepare compelling applications for orphan drug designation in multiple jurisdictions.

·      Natural History Study Design: Develop robust natural history studies that characterise disease progression and inform clinical development.

·      Rare Disease Registry Strategy: Design and implement disease registries that generate valuable long-term insights

Benefits

·      Accelerated development timelines through optimised strategies

·      Enhanced regulatory success through targeted approaches

·      Improved patient identification and clinical trial recruitment

·      Stronger value propositions for rare disease therapies

Why CortexBio

Our integrated view of rare diseases and ageing provides unique insights into disease mechanisms and therapeutic approaches. Dr. Martini-Drew's extensive experience in rare disease development at companies like Genzyme and Astellas brings practical knowledge of successful development pathways and regulatory strategies.