Recognizing Gene Therapy

Floor Speech

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Mr. LATTA. Mr. Speaker, I rise today to highlight the transformative innovation coming out of Ohio--innovation that saves and improves lives. For families in my district, this scientific ingenuity matters. I'm proud of the evolution of biomedical science and its application to drug development.

To ensure this innovation will benefit our citizens, I encourage my colleagues to support a triad of legislative policies that will expedite market entry, identify patients, and remove barriers to patient access. In an era of government price controls, which are undermining previous congressional efforts to promote innovation, such policies are particularly essential to ensuring continued biopharmaceutical industry investment in new technologies to treat rare diseases. We can work to collectively pass legislative items to increase access to treat rare diseases, as well as enact policies that will improve newborn screening, better facilitate genetic testing, and enhance the level of care disease expertise and experience on Food and Drug Administration review teams to allow for more consistent application of benefit-risk, real world evidence, and patient experience data.

I'd like to flag that gene therapy is the next evolution in treatment for rare diseases like hemophilia, sickle cell disease, cystic fibrosis, phenylketonuria, hereditary angioedema, OTC deficiency, severe combined immunodeficiency disease, Pompe disease, and muscular dystrophy. It has the potential to be the first type of therapeutic intervention for hundreds of rare pediatric disorders that lack or had lacked any treatment option, including AADC deficiency, epidermolysis bullosa, metachromatic leukodystrophy, and adrenoleukodystrophy.

The biopharmaceutical industry has invested billions of dollars in gene therapy clinical trials for such conditions at institutions throughout Ohio, including Nationwide Children's Hospital in Columbus, Cleveland Clinic, Cincinnati Children's Hospital, Akron Children's Hospital, and Dayton Children's Hospital.

Gene therapy is the focus of the nearly $100 million in grants that the National Institutes of Health has awarded in recent years to researchers throughout Ohio. For example, Dr. Krystof Bankiewicz from Ohio State has received nearly $15 million for researching a gene therapy to treat AADC deficiency, which is a fatal neurodevelopmental disorder with only 135 identified patients in the world. Due to an enzyme deficiency, ``children lack muscle control, and are usually unable to speak, feed themselves or even hold up their head,'' according to Dr. Bankiewicz. By transferring a functional copy of the AADC gene to allow for endogenous AADC enzyme production, clinical trial data have demonstrated that this gene therapy prevents oculogyric crises, allows the child to develop control, sit up, and walk, and generally improves quality of life.

Our institutions and clinicians in Ohio are truly the leaders among peers in biopharmaceutical innovation for rare disorders. The development and commercialization of safe, effective, and durable gene therapy for these rare disorders is precisely what Congress intended when it enacted the 21st Century Cures Act (including the Regenerative Medicine Advanced Therapy program), the Orphan Drug Act, the Breakthrough Therapy program, and the Rare Pediatric Disease Priority Review Voucher program.

The American people deserve a return on the biopharmaceutical industry investment in bringing gene therapies to patients, as well as the NIH support of basic research and early-stage clinical studies. If patients have an incomplete diagnosis or if diagnosed patients are subject to step therapy or other coverage restrictions or denials, gene therapy will not benefit those who need it.

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