BIO in Ohio: A gene therapy hub

BIO on the American Road visits Columbus gene therapy hub

The gene therapy research and production hub of Columbus was spotlighted when the BIO on the American Road tour visited the Ohio capital on April 28.

Sites BIO visited in Columbus:

• Nationwide Children’s Hospital produced two of the first eight gene therapies approved by the Food and Drug Administration (FDA)—for Duchenne Muscular Dystrophy and Spinal Muscular Atrophy Type 1 (SMA1). They’ve accelerated tech transfer, to help innovations go commercial.
• Ohio State University (OSU) discussed pioneering work at The James–the second-largest cancer hospital in the country–and efforts to make clinical trials faster and more inclusive of rural communities.
• Sarepta’s gene therapy work in Columbus led the Massachusetts-based firm to invest in the 140,000-square-foot GTCOE, which opened in 2021 and houses both gene therapy research and manufacturing.

Expediting clinical trials: “Nationwide Children’s prides itself on expediting first-in-human clinical trials,” by exploiting the presence of researchers and clinical facilities hospital official Dennis Durbin said. Meanwhile OSU is helping organize a network of Big Ten schools seeking to centralize Institutional Review Boards (IRBs) and contracting for clinical trials.

What they’re saying: “Ohio is unique in that we truly represent the entire life science value chain right here within our borders,” said Eddie Pauline, President and CEO of Ohio Life Sciences (OLS), noting Amgen’s $900 million manufacturing expansion, pharma delivery services, and more.

BIO’s view: “Every state wants to be a leader in biotech. It takes people, capital, great universities, facilities—and a vision,” said BIO President & CEO John F. Crowley. In roundtables and presentations at various locations, BIO and local leaders in the sector discussed strategies for building on Ohio’s advantages and increasing its leadership.

Read more on Bio.News.

BIO and its members ‘elevating women’s health’

The theme for this year’s National Women’s Health Week, which starts Sunday, is “Prevention, Innovation, and Impact.” BIO is working on the innovation part.

Why it matters: Only 6% of private health care investment goes toward women’s health, and 90% of that goes to cancer, reproductive health, and maternal health, a recent report finds. There’s insufficient research into other women’s health issues and into the way common conditions like heart attacks impact women differently.

BIO’s Women’s Health Task Forceseeks to correct this insufficiency, promoting innovation and investment to bring new treatments for women to the market, Michele Oshman, BIO’s Chief Patient Advocate and Women’s Health Task Force executive sponsor, told BIO’s panel at the Health Executive and Research Summit.

BIO members focused on women’s health collaborate: Organon offers accelerator services that Daré Bioscience accessed to help commercialize their first FDA-approved product, notes Daré President and CEO Sabrina Martucci Johnson. Daré also invites women to own their healthcare solutions through a small investor program.

Legislation to fund research into menopause, reintroduced April 30 by Sens. Patty Murray (D-WA) and Lisa Murkowski (R-AK), would give $275 million over five years for “strengthening and expanding federal research, health care workforce training, awareness and education efforts, and public health promotion and prevention activities.”

BIO’s view: “The mission of BIO’s Women’s Health Task Force is to ensure a robust pipeline of biopharmaceutical innovations for women’s health,” according to Oshman. “We are committed to elevating women’s health.”

Read more on Bio.News.

What Else to Read

President Trump nominates Nicole Saphier, MD, to be surgeon general. Saphier, a radiologist, is director of breast imaging at Memorial Sloan Kettering Cancer Center-Monmouth and has served as a Fox News contributor since 2018. Her nomination is to be considered by the Senate Health, Education, Labor & Pensions Committee, which did not advance the previous nominee, Casey Means. Read more here.

BIO President & CEO John F. Crowley appears on CNBC Squawk Box. He mentioned “signs of an increasingly healthy biotech market” and noted that we are now marking the first 50 years of biotech. He also discussed the importance of maintaining U.S. leadership in biotech, for economic and national security reasons, and urged reforms that can guarantee that leadership for the next 50 years. Watch the interview here.

Reports detail lag in National Institutes of Health (NIH) grant giving. Due to a steep drop in awards, NIH only approved 13% of applications for research grants in this fiscal year, Stat reports. Two other analyses in April found slow grant giving at NIH, the world’s largest public funder of biomedical research. NIH grant giving in March was about a third of the pace under the previous administration, The New York Times said, and was less than half the pace of 2025, according to The Washington Post.

Beltway Report

During last week’s House Ways and Means Committee hearing with hospital CEOs, Chair Jason Smith (R-MO) criticized 340B abuses in his opening statement: “Large hospital systems also manipulate the 340B drug pricing program to keep steep drug discounts for themselves instead of passing the savings to low-income patients. There is little evidence that the $290 billion in discounts given to hospitals under the 340B program since Obamacare was ever reinvested in patients. Even worse, there is evidence that hospital abuse of 340B actually directly led to increases in Obamacare premiums.”