The Alliance for Regenerative Medicine Releases Statement in Response to National Academies Report on Human Genome Editing - Seite 2

ARM believes that advances in the field of gene therapy, including somatic cell genome editing, have the potential to profoundly and positively impact the practice of medicine for currently incurable genetic diseases, such as muscular dystrophy, sickle cell disease (SCD), cystic fibrosis, hemophilia, adrenoleukodystrophy (ALD), Alpha-1 Antitrypsis Deficiency (AATD), and Transthyretin Amyloidosis (ATTR), as well as acquired diseases such as cancer, certain forms of heart disease, HIV, Hepatitis B virus, and other infectious diseases.

It is estimated that 30 million Americans, or 1 in every 10 people, are afflicted with one of the approximately 7,000 rare diseases. Two thirds of those affected are children. The National Organization for Rare Disorders (NORD) estimates that for 95 percent of these diseases no FDA-approved treatment currently exists,(1) and the few treatments that are available generally address the symptoms and not the underlying genetic cause of the disease. As a result, these treatments must be administered for the duration of a patient's life. In contrast, genome editing offers the very real potential to bring hope to rare disease patients through development of a broad range of new technologies to precisely target and modify the genetic material of a patient's cells. By removing, repairing, or replacing a defective gene or genes, these therapies hold the promise of potentially curing a broad range of diseases with a single treatment.

Similarly, in diseases such as cancer, HIV, and beta-thalessemia, genome editing is being employed to modify T cells and hematopoietic stem cells ex-vivo. The modified cells are then delivered to the patient to treat and potentially cure the underlying disease. These programs build upon early successes and several advanced programs based on somatic cell gene replacement therapies.

According to a recent white paper titled, "Therapeutic Gene Editing," published by the American Society of Gene & Cell Therapy (ASGCT), "the successful development of effective treatments based on genome editing could shift today's approach from a lifetime of symptom management for hereditary diseases to tomorrow's ideal of making a one-time curative repair or change to an individual's affected gene. The goal is a long lasting, perhaps life-long effect that minimizes or even eliminates disease."(2) Diseases involving multiple genes may also be treatable if the therapy can alter specific genes affecting the course of the disease.