Industry Overview

An Expansive and Growing Industry

As of year-end 2016, ARM estimates there are 775+ companies with a regenerative medicine and/or advanced therapies focus, ranging from divisions of multinational corporations to smaller organizations focused solely on the sector.

The products they are developing include several hundred cell-based therapies, biologics, tissue-engineered cells and materials and implantable devices. Additional products use cells as drug discovery or toxicity testing tools as well as clinical tools, bioprocessing tools and platforms that include equipment, consumables, reagents and storage systems.

The field also incorporates a variety of service companies specializing in clinical trial management, manufacturing, engineering and financing among others.

Regenerative Medicine Technologies Include a Variety of Therapeutic Approaches

The regenerative medicine and advanced therapies sector is truly the next frontier for patients, developing revolutionary, durable treatments and potential cures for some of humankind’s most devastating diseases – many currently untreatable via conventional treatments – through the use of transformative scientific discoveries and technologies. These products are intended to augment, repair, replace or regenerate organs, tissues, cells, genes and metabolic processes in the body.

This sector holds the promise of addressing many currently unmet medical needs, and represents a major paradigm shift in medicine – treating the root causes of disease and disorders to stop or even reverse progression.

GENE THERAPY AND GENOME EDITING

Gene therapy seeks to modify, replace, inactivate or introduce genes into a patient’s body with the goal of durably treating, preventing or even curing disease.

Gene therapy techniques can also be used to genetically modify a patient’s cells outside of the body, which are then re-introduced to deliver modified or corrected cells into the body, an approach known as gene-modified cell therapy.

Gene therapy may be performed in vivo, in which a gene is transferred to cells inside the patient’s body, or ex vivo, in which a gene is delivered to cells outside of the body, which are then transferred back into the body.

Genome editing is a practice in which DNA is inserted, replaced or removed using “molecular scissors” or artificially engineered gene constructs, in order to potentially cure genetic diseases and/or disorders. Types of genome editing technology include CRISPR/Cas, TALEN, zinc finger nucleases and more.

CELL THERAPY

Cell therapy is the administration of viable, non-genetically modified cells into a patient’s body to grow, replace or repair damaged tissue for the treatment of a disease. Cells can be administered allogeneically, in which the patient receives cells from a donor, or autologously, in which the patient receives cells from his or her own body.

TISSUE ENGINEERING AND BIOMATERIALS

Tissue engineering combines scaffolds, cells and biologically active molecules into functional tissues to restore, maintain or improve damaged tissues. Biomaterials are medical devices designed to interact with living systems, providing physical structures and support for engineered tissues.