Dr. Terence Flotte Calls for CGT Investment and Collaboration During Congressional Testimony

When asked what he’s most excited about in the cell and gene therapy field over the next five years, ASGCT Vice President Terence Flotte, MD, said it’s the clinical dissemination of gene-editing technology, including CRISPR, to treat diseases that he’s looking forward to.

But discontinued clinical gene therapy programs and funding challenges have made that goal very difficult, said Dr. Flotte, in response Thursday to follow-up questions from Rep. Cathy McMorris Rodgers (R-WA), Chair of the House Energy and Commerce Committee.

Dr. Flotte gave testimony as part of a panel of witnesses on Rare Disease Day before the group’s Subcommittee on Health about the potential of and challenges surrounding gene therapies to treat rare diseases.

Many researchers and scientists are at the mercy of the investment community, Flotte said. The advancement of cell and gene therapies requires participation from the National Institutes of Health (NIH), private industry, universities, and foundations.

“When one of those parts falls out, it is very difficult to continue some of these programs,” Dr. Flotte said. “For the families, the clocking is ticking.”

Dr. Flotte noted ASGCT supports robust funding for NIH and called for continued investment in basic, translational, and early clinical research. On the regulatory side, he said ASGCT will keep working with FDA to create a framework that encourages and supports treatment development.

Dr. Flotte also mentioned that around half of those affected by rare diseases are children and asked the committee to reauthorize the Pediatric Priority Review Voucher Program before it expires Sept. 30. He added gene therapy products represent 28% of all designations in the program.

To close out his testimony, Dr. Flotte said scientists and physicians “have an obligation to do everything in their power to help patients and families living with rare genetic diseases.”

Read the full testiomony     Watch the recorded hearing

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