A researcher pipetting into microtubes

Biomanufacturing capabilities

Mayo Clinic’s strategy in regenerative biotherapeutics emphasizes an enhanced capability for biomanufacturing, with technology platforms supporting the development of new therapeutics known as biotherapies. Biotherapies are different from the medicines that have traditionally been used to treat symptoms and manage disease. They are new types of treatment that are made from samples from living people, such as blood, cells, enzymes and genes. Biotherapies have the potential for targeted healing with few side effects.

The Center for Regenerative Biotherapeutics’ biomanufacturing capabilities enable healthcare teams to deliver next-generation treatments and cures for patients. The center is focused on late-stage discovery, clinical translation and manufacturing for phase 1 clinical trials.

The center’s capabilities are centered around a comprehensive manufacturing ecosystem that includes process and analytic development, Current Good Manufacturing Practice (cGMP) manufacturing, quality control, quality assurance, and regulatory support. Its state-of-the-art research and biomanufacturing facilities allow for field-leading capabilities in process development, manufacturing and quality standards.

Focus areas

The Center for Regenerative Biotherapeutics has three domains of activity aimed at bringing new regenerative therapies to patients:

  • Cellular therapies.
  • Gene and viral therapies.
  • Biomaterials and tissue engineering.

Cellular therapies

Cellular therapies are living cells that are used to treat or prevent diseases. They can restore, repair or replace damaged or dysfunctional tissues in the body. Cellular therapies hold great promise for the treatment of diseases and conditions including:

  • Autoimmune diseases.
  • Blood cancers.
  • Bone or joint damage.
  • Degenerative diseases.
  • Dermatitis.
  • Genetic diseases.
  • Inflammatory bowel disease.
  • Lupus.
  • Musculoskeletal damage.
  • Solid tumor cancers.
  • Tissue damage.

Gene and viral therapies

Gene and viral therapies introduce, modify or replace genetic material within a person’s cells to treat or prevent a disease. They aim to correct the underlying genetic causes of disease by targeting the specific genes involved. These treatments have potential to be used with genetic conditions including inherited diseases, certain types of cancer and some viral infections.

Some of the center’s key breakthroughs in gene and viral therapies include:

  • B cell activating factor-receptor (BAFF-R) chimeric antigen receptor (CAR)-T cell therapy for B cell cancers that have returned and no longer respond to standard treatment.
  • CAR-T cell therapies for solid tumors and immune disorders.
  • A dendritic cell-based vaccine for advanced ovarian cancer. Dendritic cells have shown potential for triggering the immune system to fight cancer.
  • Thyroid-stimulating hormone receptor CAR-T cell therapy targeting thyroid cancer.
  • Mesenchymal stem cell CAR-T cell therapy targeting graft-versus-host disease.

Biomaterials and tissue engineering

Biomaterials are living tissues that scientists design and grow outside the body. Doctors can then use the tissue products to replace damaged or diseased tissues in a patient. Biomaterials and engineered tissues could improve options for transplant or be used to treat difficult-to-heal wounds. For example, Mayo Clinic teams are working on creating new tissues to treat patients with:

  • Glottic insufficiency.
  • Liver disease.
  • Trachea and larynx injuries or disease.

The GENESIS initiative: Revolutionizing organ transplantation

The Center for Regenerative Biotherapeutics supports Mayo Clinic’s transplant research, which could lead to new lifesaving therapies for patients who currently have limited options. The GENESIS initiative aims to overcome barriers such as a shortage of donor organs in the following ways:

  • Organogenesis — developing methods to create organs from stem cells.
  • 3D printed organs — using advanced printing technologies to create organs.
  • Tissue engineering for organ regeneration and rejuvenation — creating biological substitutes to restore or improve organ function.
  • Immunomodulation — designing therapies to control immune responses and reduce rejection of transplanted organs.