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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 The draft guidance tackles a long‑standing barrier that has delayed access to potentially life‑saving treatments for people with rare conditions and proposes new ways to spur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

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

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

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

For Dr. Allyson Berent, science and motherhood collided the day her daughter was diagnosed with Angelman syndrome. Today, as Chief Science Officer at the Foundation for Angelman Syndrome Therapeutics (FAST), she’s turning that urgency into progress, advancing the first gene therapy programs for Angelman syndrome and reshaping how rare disease research moves from discovery to treatment. She also co-founded AS 2 Bio, a drug development accelerator that is building a new model f

Kendra Riley is a public relations professional, mother, and a strong advocate for gene therapies. In 2020, two of her daughters were...