Regenerative biotherapeutics: Pivoting toward a new strategy for fighting disease

ROCHESTER, Minn. — Mayo Clinic has made significant strides biomanufacturing early-stage therapeutics. The focus is on new medicines derived from the human body, called biotherapeutics, which are shaping the future of medicine.

"Our commitment to delivering new cures for unmet conditions is behind Mayo's pivot to biomanufacturing," says Julie Allickson, Ph.D., the Michael S. and Mary Sue Shannon Family Director of Mayo Clinic's Center for Regenerative Biotherapeutics and the Otto Bremer Trust Director, Biomanufacturing and Product Development, Center for Regenerative Biotherapeutics.

Regenerative biotherapeutics is an emerging field in which biologics from human sources — cells, blood, enzymes, tissues, genes or genetically engineered cells — are used in medicines. The hope is that biologics will provide targeted healing with fewer side effects.

"Mayo Clinic is investing in biotherapeutics to provide new options aimed at treating diseases that have not responded to standard, small molecule drugs," Dr. Allickson says. "Biotherapeutics derived from human sources show promise for treating conditions that, so far, have eluded medical science, particularly for rare and complex disorders."

Chimeric antigen receptor therapy (CAR-T therapy) is an example of a biotherapeutic that is transforming care for some blood cancers. CAR-T therapy uses genetically engineered T cells to recognize and destroy cancer cells. It has shown therapeutic value in some B-cell leukemias and lymphomas and multiple myeloma.

Mayo Clinic is building collaborations among physicians, scientists, industry collaborators and facilities to advance new biotherapeutics.

"Industry alliances are essential for moving promising discoveries beyond phase two clinical trials," Dr. Allickson says. "At Mayo Clinic, we are working side-by-side with industry collaborators to manufacture validated therapies. It's a fairly new model for an academic medical center, and we're very excited about the new biotherapeutic options this could bring to patients."

Strategic focus is on potential treatments for chronic conditions such as diabetes, kidney disease or cancer.

"Our goal is to accelerate validated therapies that enhance quality of life for the patient," Dr. Allickson says. "For example, we are assessing CAR-T discoveries that can target solid tumors. If we determine we can manufacture products for early phase clinical trials, we will seek industry collaborators to commercialize them for the benefit of patients around the world."

Mayo Clinic is developing biomanufacturing facilities at its sites in Arizona, Florida and Minnesota. The strategy is built on four pillars of biomanufacturing early-stage therapeutics:

• Process development – the bridge from the lab to clinical manufacturing.
• Quality control – ensuring regulatory standards for safety in patients are met.
• Quality assurance – monitoring and evaluation to ensure consistent quality.
• Advanced biomanufacturing – manufacturing processes that align with Food and Drug Administration standards for commercial therapies.

Any revenue Mayo Clinic realizes from licensing its discoveries are reinvested in Mayo's nonprofit research and education initiatives.

Dr. Allickson is available for interviews on early stage biomanufacturing of biotherapeutics and what that could mean for patients.

###

About Mayo Clinic 
Mayo Clinic is a nonprofit organization committed to innovation in clinical practice, education and research, and providing compassion, expertise and answers to everyone who needs healing. Visit the Mayo Clinic News Network for additional Mayo Clinic news.