Why BridgeBio Stopped Galactosemia Gene Therapy

GY007: A Baker's Yeast That Eats Galactose, with Dr. Judy Fridovich-Keil and Dr. Shauna Rasmussen

In this episode, Ryan sits down with two veterans of the show — Dr. Judy Fridovich-Keil and Dr. Shauna Rasmussen, both of Emory University — to walk through a recent study from their lab in collaboration with a small biotech called GutsyBio. The study tested whether a specially selected strain of baker's yeast (Saccharomyces cerevisiae), called GY007, could break down dietary galactose in the gut before it ever reaches the bloodstream. In the rat model, it worked: rats that received the yeast before being given galactose did not show the same Gal-1-P or galactitol spikes in plasma and brain that the untreated rats did.

Why BridgeBio Stopped Galactosemia Gene Therapy

BridgeBio recently stopped their research and development of gene therapy for Galactosemia. This came as a shock to many of us. I talked to the CEO of BridgeBio on the phone, and in this episode I report back what he told me.

Gene Therapy for Galactosemia: Feat. Mandy and Clayton from BridgeBio

In this episode of The Galactosemia Podcast, Ryan sits down with Clayton Beard and Mandy Rohrig from BridgeBio to explore the promise and challenges of gene therapy for galactosemia. They break down how gene replacement works, why viruses are used as delivery systems, and what families should know about the current state of research. To get in touch with Mandy - Amanda.Rohrig@bridgebio.com

Maureen Bell's Story of Living with Galactosemia

In this episode of The Galactosemia Podcast, I sit down with Maureen Bell, who was diagnosed with classic galactosemia just seven days after her birth in 1972—long before the condition was part of newborn screening. Maureen shares what it was like growing up in the seventies, eighties, and nineties with galactosemia, from childhood struggles and dietary restrictions to the emotional challenges of premature ovarian insufficiency. Now 53, Maureen reflects on how the galactosemia diet has evolved over the decades, what it meant to be part of the very first galactosemia conference, and how she turned personal challenges into powerful advocacy—including speaking at national conferences and even before Congress.

Exploring Bone Marrow Transplant in Galactosemia

In this episode, we dive into new research on bone marrow transplantation as a potential approach to treating Classic Galactosemia. Dr. Judy and her team share insights from their recent study, walking us through why they pursued this line of research, how the experiments were designed, and what they discovered. We talk about the role of biomarkers, the challenges of translating findings from animal models to human patients, and the importance of continued NIH funding to push rare disease science forward. Along the way, the team reflects on their own experiences in the lab and what it means to see their work connect to families living with Galactosemia