On June 3rd, 2014, BIO hosted a briefing entitled “The Untapped Potential of Advanced Modeling: How Animal Biotech Speeds Drug Development” to showcase the growing field of biomedical applications through animal biotechnology. Panelists specifically highlighted opportunities for GE animal models of human disease to help patients with devastating diseases. The panel included three experts in the field: Danielle Kerkovich, PhD, the Principal Scientist of Beyond Batten Disease Foundation, John Swart, PhD, President of Exemplar Genetics, and Jay Cormier, JD, Associate, Hyman, Phelps & McNamara, P.C.
Dr. Kerkovich highlighted her Foundation’s initiatives for battling Batten, a rare neurological disorder primarily found in children. As research and clinical trials are performed on each new potential treatment, innovators can face financial barriers and long development timeless, she argued. Beyond Batten is seeking better methods to perform these testing steps, and large animal models are being developed through animal biotechnology to speed development. Kerkovich called for non-governmental organizations to support this process through their collective strengths of informing federal funders, legislators, researchers, and regulators about the power and lowered cost of these models versus traditional mouse models.
Dr. Swart then introduced the possibilities available through GE livestock models due to their similarities in anatomy, human like disease symptoms, and use for therapeutic development. He notably illustrated positive impacts of models that are already in preliminary use for studying cystic fibrosis, heart disease, and degenerative muscular diseases. “Better models will enable researchers to discover new disease mechanisms, accelerate drug and device discovery, and even improve predictive efficacy,” he argued.
Wrapping up, Dr. Jay Cormier outlined the hurdles that FDA regulations can place on GE livestock models and the effects that this has on the industry. In 2009, FDA published a final guidance document indicating FDA’s current thinking on the regulation of GE animals under their New Animal Drug authorities. The law defines a “drug” as an article that is “intended to affect the structure or any function of the body of man or other animals” and the FDA treats the GE DNA as a new animal drug. He postulated that the reason FDA approvals can take a long period of time was that some of them induce inquiries from several layers of the Federal government. Dr. Cormier concluded his presentation by laying out arguments against the necessity of every step in the regulatory process for GE animal models as those animals will not be used for food or directly as human medicine.
Biotechnology flourishes in a supportive research and regulatory environment, and GE livestock models can be the key to unlocking the secrets behind some of the world’s most devastating conditions. Regulatory decisions on GE animals will impact our ability to find these cures more efficiently and though less cost for those most in need.