The Alliance for Regenerative Medicine Releases St

WASHINGTON, DC--(Marketwired - Feb 14, 2017) - The National Academies of Sciences, Engineering and Medicine today issued a report that examines the scientific, clinical, ethical, legal and social implications of human genome editing. The Alliance for Regenerative Medicine (ARM) believes that genomic medicines, including genome editing, hold great promise for the treatment of a multitude of hereditary and acquired diseases where there is presently no effective treatment available.

ARM issued the following statement:

The Alliance for Regenerative Medicine (ARM) applauds the National Academies of Sciences, Engineering and Medicine for its very thorough and thoughtful report on the current scientific, technical, ethical, and policy issues relating to human genome editing. We support the need for responsible and ethically appropriate approaches to research and clinical use of these technologies following the seven guiding principles outlined in the report, as well as the need for continued public engagement and dialogue. We also commend the Academies for recognizing the profound impact genome editing will have on the development of a new class of medicines for many patients with presently incurable diseases.

We believe the report's recommendations that "existing regulatory infrastructure and processes for reviewing and evaluating somatic gene therapy to treat or prevent disease and disability" are sensible and will help create a safe path toward eventual clinical adoption and regulatory approval of therapeutics based on somatic cell genome editing.

In addition, we note the report's recommendations on heritable germline genome editing and the strict criteria to be met before ever considering clinical study. ARM will continue to monitor developments related to these applications, but until safety is proven and the risks associated with long-term consequences, both intended and unintended, are fully evaluated, we will remain solely focused on realizing the full therapeutic potential of somatic cell genome editing. Further, we must be satisfied that all relevant moral and ethical issues have been addressed and that a broad societal consensus exists as to the benefits and risks associated with editing the germline.

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.

ARM represents a number of companies and research institutions that use various gene therapy and genome editing technology platforms, including CRISPR/Cas9, zinc finger nucleases (ZFNs), homing endonucleases, vector-driven homologous recombination, transcription activator-like effector-based nucleases (TALEN) and meganucleases, amongst others to design therapeutics that address a wide range of, hereditary and acquired diseases.

About The Alliance for Regenerative Medicine The Alliance for Regenerative Medicine (ARM) is an international multi-stakeholder advocacy organization that promotes legislative, regulatory and reimbursement initiatives necessary to facilitate access to life-giving advances in regenerative medicine worldwide. ARM also works to increase public understanding of the field and its potential to transform human healthcare, providing business development and investor outreach services to support the growth of its member companies and research organizations. Prior to the formation of ARM in 2009, there was no advocacy organization operating in Washington, D.C. to specifically represent the interests of the companies, research institutions, investors and patient groups that comprise the entire regenerative medicine community. Today, ARM has more than 250 members and is the leading global advocacy organization in this field. To learn more about ARM or to become a member, visit http://www.alliancerm.org .

1. National Organization for Rare Disorders (2015). NORD developing 20 natural history studies for 20 rare diseases (Press Release). https://rarediseases.org/fda-awards-nord-2500...-for-rare- disease-research/ . 2. American Society of Gene & Cell Therapy (2016). Therapeutic Gene Editing: An American Society of Gene & Cell Therapy White Paper. http://www.asgct.org/UserFiles/file/Therapeut...v21_v1.pdf .