IGT

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 m…

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 commu…

This week, the FDA released draft guidance to help developers bring promising gene therapies to patients more efficiently by making greater use of existing scientific and regulatory knowledge.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

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 Inst…

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 The draft guidance tackles a long‑standing barrier that has delayed access to potentially life‑saving treatments for people with rare conditions and proposes new ways to spur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

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 stateme…

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 Pa…

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. K…

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 c…

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

Nate Plasman is a Duchenne Muscular Dystrophy (DMD) parent advocate, a devoted husband to his wife of 20 years, Sara, and a proud father...

Lauren Holder is a research ambassador, advocate, and committed caregiver. After discovering she was gene-positive for Huntington’s...

On June 17, the Institute for Gene Therapies (IGT), in partnership with the Alliance for Regenerative Medicine (ARM), convened a panel...

Amanda Moore is a devoted mother, advocate, and CEO of the Angelman Syndrome Foundation. After adopting her two sons, Jackson and Baden,...

Lesa Brackbill is a passionate mother, advocate, author, and member of IGT’s Patient Advocacy Advisory Council. After her daughter...

Authors: IGT Chairman Erik Paulsen, Scientific Academic and Medical Council Chair Donna Christian-Christensen, M.D. As our nation’s...

As we commemorate the fifth anniversary of the Institute for Gene Therapies (IGT) today, I want to take a look back at the strides we've...

IGT leaders recently penned a piece exploring the strides being made in gene therapy and highlighting the ongoing challenges that must be...

As Newborn Screening Awareness Month concludes, IGT applauds the tireless work of advocates who dedicate themselves 365 days of the year...

Author: Andrea Matthews, Founder, Children’s Sickle Cell Foundation, Inc.   Newborn screening (NBS) is an important public health measure...