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Statement on FDA Draft Guidance to Leverage Prior Knowledge for Gene Therapy Development

This week, the FDA released draft guidance to help developers bring promising gene therapies to patients more efficiently by making greater use of existing scientific and regulatory knowledge. The Institute for Gene Therapies (IGT) Chairman Congressman Erik Paulsen issued the following statement: “This is a welcome and practical step forward for patients living with rare and serious diseases who cannot afford to wait. By showing developers how to build on existing CMC, no

34 minutes ago

IGT Statement on House Re-Introduction of the MVP Act

Yesterday, Representative Brett Guthrie (R-KY), Chairman of the House Committee on Energy and Commerce, alongside Reps. Jake Auchincloss (D-MA), John Joyce, M.D. (R-PA), Scott Peters (D-CA), Mariannette Miller-Meeks (R-IA), and Don Davis (D-NC), reintroduced the Medicaid VBPs for Patients Act, or the MVP Act in the U.S. House. This follows Senate reintroduction of the MVP Act last year. The Institute for Gene Therapies (IGT) Chairman Congressman Erik Paulsen issued the follo

Mar 12

IGT Applauds FDA’s Draft Framework to Speed Individualized Therapies, Urges Continued Alignment of Regulation with Breakthrough Treatments for Unmet Needs

The U.S. Food and Drug Administration (FDA) this week released a draft Plausible Mechanism Framework for recognizing the value of accelerating development for individualized therapies for rare and ultra-rare diseases. The draft guidance tackles a long‑standing barrier that has delayed access to potentially life‑saving treatments for people with rare conditions and proposes new ways to spur rare‑disease innovation through clear scientific criteria, closer regulatory collabora

Feb 26

IGT Statement on Passage of Congressional Funding Package

Today, Congress passed a critical funding package including a five-year extension of Pediatric Priority Review Vouchers (PRVs) in the Give Kids a Chance Act and bipartisan legislation aimed at reducing barriers for patients facing red-tape when trying to access innovative treatments via the Accelerating Kids Access to Care Act . IGT Chairman Congressman Erik Paulsen issued the following statement:  “IGT applauds Congress for advancing this legislation with critical provisi

Feb 4

IGT Statement on Congressional Funding Package

Today, Congressional leaders released a bipartisan compromise to fund the federal government ahead of the January 30 shutdown deadline. The legislation included several key provisions the Institute for Gene Therapies (IGT) has been long advocating for: a five-year extension of Pediatric Priority Review Vouchers (PRVs) in the Accelerating Kids Access to Care Act . IGT Chairman Congressman Erik Paulsen issued the following statement: “We welcome the inclusion of a five-year e

Jan 21

IGT Applauds Administration’s Decision to add MLD, DMD to the Recommended Uniform Screening Panel

The Health Resources and Services Administration (HRSA) has announced the decision to add metachromatic leukodystrophy (MLD) and Duchenne muscular dystrophy (DMD) to the federal Recommended Uniform Screening Panel (RUSP). IGT Chairman Congressman Erik Paulsen issued the following statement: “IGT is grateful for the leadership of U.S. Department of Health and Human Services Secretary Robert F. Kennedy, Jr. and HRSA Administrator Tom Engels in recognizing the urgent need for

Dec 18, 2025