As Congress considers Most Favored Nation (MFN) pricing proposals, lawmakers are overlooking a critical consequence: children with rare diseases could lose access to potentially curative gene therapies. The Institute for Gene Therapies (IGT) is raising concerns that MFN pricing mechanisms would effectively unravel the Medicaid Best Price reforms designed to protect vulnerable patient populations. These reforms have enabled innovative payment models that make high-cost, one-ti

World Sickle Cell Day, observed annually on June 19th, raises global awareness about sickle cell disease (SCD): a hereditary blood disorder affecting millions worldwide that causes red blood cells to deform into a rigid, sickle shape, disrupting oxygen flow and causing severe pain, organ damage, and life-threatening complications. About 1 in 13 African Americans carry the sickle cell trait, and many do not know they have it. Recent advances in gene therapy have reached a life

IGT Chairman Erik Paulsen in BioSpace The FDA must provide consistent and predictable regulatory frameworks if the U.S. is to maintain its leadership in gene therapy, one of the most consequential therapeutic fields of our generation. The resignation of FDA Commissioner Marty Makary on May 12 was the latest disruption in what has been a difficult period for the rare disease and gene therapy communities. For families living with devastating genetic conditions, and for innovato

This week, the FDA released draft guidance to help developers bring promising gene therapies to patients more efficiently by making greater use of existing scientific and regulatory knowledge.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

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

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