ARM Patient Resources & Advocacy

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.

 Download "About Regenerative Medicine" one-pager

 

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.

Glossary of Terms

Allogeneic cell transplant – A procedure in which a patient receives cells from a donor.

Autologous cell transplant – A procedure in which a patient receives cells from his or her own body.

DNA (deoxyribonucleic acid) – A molecule found in the cell’s nucleus that contains an organism’s genetic information.

Ex vivo gene therapy / genome editing – A practice in which a gene is transferred to, or modified in, cells outside of the body. The modified cells are then transferred into the
patient’s body.

Gene – A short section of DNA that forms the basic physical and functional unit of heredity.

Genome – An organism’s complete set of DNA, comprising all genes. Each genome is like a blueprint and contains all the information needed to build and maintain that organism.

Induced pluripotent stem cells (iPSCs) – Adult cells that have been genetically reprogrammed into an embryonic-like pluripotent state that enables the development of an unlimited source of any type of human cells needed for therapeutic purposes.

In vivo gene therapy / genome editing – A practice in which a gene is transferred to, or modified in, cells inside the patient’s body.

Pluripotent stem cell – A type of “master cell” that is capable of becoming one of many different cell types in an organism.

Additional Resources 

American Society of Gene & Cell Therapy 
California Institute for Regenerative Medicine
European Society of Gene & Cell Therapy 
International Society for Cellular Therapy 
International Society for Stem Cell Research
Tissue Engineering International & Regenerative Medicine Society