ABOUT
A collaborative initiative of OPHN1 Foundation.
ROCK Access Collaborative brings together rare-disease organizations, researchers, clinicians, and other stakeholders to evaluate shared RhoA–ROCK biology and, where scientifically justified, develop common scientific, safety, and regulatory infrastructure.
Why OPHN1 Foundation started this effort
OPHN1 is a Rho GTPase-activating protein involved in regulation of RhoA signaling, actin dynamics, synaptic function, and neurodevelopment. OPHN1 Foundation’s work on ROCK inhibition identified challenges that are difficult for any ultra-rare organization to address alone, including drug supply, pediatric safety, biomarker development, regulatory strategy, funding, and access to specialized expertise.
ROCK Access Collaborative was established to determine where those challenges—and potential solutions—can responsibly be shared across rare genetic disorders while preserving disease-specific scientific and development decisions.
Current work
The Collaborative is developing a mechanism-based framework for evaluating rare genetic disorders involving RhoA–ROCK signaling and identifying research needs that may be shared across multiple disease communities.
Current areas of work include translational evidence assessment, pediatric safety and pharmacology considerations, biomarker strategy, research standards, and regulatory and access pathways for ROCK-targeted therapies.
Principles
- Evidence before advocacy for a specific drug
- Transparent inclusion and prioritization criteria
- Scientific independence and conflict disclosure
- Patient and family priorities built into decision-making
- Disease-specific control of data and development decisions
- Responsible communications that distinguish hypothesis from proof