Queen Mary trials new approach to drug development for rare diseases

Fixing the discovery bottleneck

Developing disease modifying therapies for rare diseases is extremely challenging. It’s clinically difficult due to the small cohort sizes, and it’s difficult to raise investment as the market may not be big enough to cover the costs. Better collaboration between academia, industry and philanthropy could address this.

Based at the Centre for Cell Biology and Cutaneous Research at Queen Mary’s Blizard Institute, this new collaboration involves working with DEBRA Research to create the infrastructure for commercial testing of promising EB drug candidates.

Queen Mary research teams led by Dr. Emanuel Rognoni and Dr. Matthew Caley will provide capabilities for testing in relevant preclinical models as a service to other researchers across academia, biotech and pharma.

This is a major step forward because access to high-quality preclinical models is one of the major bottlenecks in EB drug development.

The partnership will initially focus on preclinical research, laying the groundwork for future therapeutic innovation and potentially new therapy approaches.

Turning research excellence into real world impact

Both lead researchers have extensive experience in regenerative approaches and cellular mechanisms relevant to skin disorders.

Dr. Rognoni and his team are investigating how different fibroblast types – critical cells in the connective tissue – contribute to development, healing, ageing, and disease.

 Dr. Caley’s research focuses on the basement membrane zone, a structural interface essential for skin stability and repair, and its role in wound healing, ageing, cancer, and EB.

Not only will this initiative accelerate the path to effective therapies, transforming the lives of people living with EB, it also represents a creative, scalable new cooperation model between academia, philanthropy, and industry for advancing rare disease research – a model that could be replicated across other conditions to benefit millions of patients.

Contact our Business Development team to learn more about partnering with Queen Mary.