Connecting Patient Advocacy and Industry for Gene Therapy Development

“Through early collaboration, the patients can give their sense of urgency and can answer some pretty hard questions. Through this, the pharmaceutical companies can really humanize the diseases. They're in the human business and these partnerships humanize the business.”

As more companies explore gene and cell therapies for a variety of diseases, including rare diseases like Charcot-Marie-Tooth (CMT) disease, it has become apparent that collaboration between industry, academics, and patient advocacy groups is paramount to successfully develop and evaluate these therapies.

A discussion, entitled “Partnering for Progress”, explored this topic during the 2022 BIO International Convention, held June 13-16 in San Diego, California. Among panelists was Susan Ruediger, founder and chief mission officer, CMT Research Foundation, who herself has CMT.

CGTLive spoke with Ruediger to learn more about the unique perspectives that each group has to offer in the therapy development process. She also discussed the value of meetings like BIO for bringing together these groups and ideas.

REFERENCE

Schmidt H, Ruediger S, et al. Partnering for progress: Collaborating with patient organizations to advance therapy development and maximize patient benefit. Presented at: 2022 BIO International Convention, June 13-16, San Diego, CA.