Effectively Enabling Rare Diseases To Reach Patients

By Dr. Rebecca Abram, Director, Strategic Technical Marketing, and Dr. Joanna Norman, Director of PD Operations, Viral Gene Therapy Operations, U.K.

Rare diseases, a widespread, unmet medical challenge affecting over 400 million people globally, are no longer an insurmountable hurdle. While there are over 7,000 rare diseases affecting patients, recent advancements offer a glimmer of hope, including five groundbreaking approvals for cell, gene, and RNA therapies. The increasing number of fast-track approvals and orphan drug designations from regulatory authorities signifies a commitment to accelerating the development of rare disease treatments.

However, to bring a rare disease therapy to clinic, developers will need a robust understanding of how to manage timeline, budget, critical quality attributes, and scale-up. In many cases, inexperienced sponsors are facing clinical holds from the FDA as a result of insufficient chemistry, manufacturing, and controls information. To alleviate this, developers could consider partnering early with an experienced CDMO that offers flexibility and capacity at multiple scales, regulatory knowledge, and high-throughput analytical processing technologies. Download the full article to learn more about what a manufacturing partner can offer your rare disease therapy.