[Congressional Record Volume 169, Number 147 (Tuesday, September 12, 2023)]
[Extensions of Remarks]
[Page E830]
From the Congressional Record Online through the Government Publishing Office [www.gpo.gov]




                        RECOGNIZING GENE THERAPY

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                          HON. ROBERT E. LATTA

                                of ohio

                    in the house of representatives

                      Tuesday, September 12, 2023

  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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