This week: New hope for people living with Huntington’s disease, and a report card on RFK Jr.’s effort to remake health in America.<br><br>The Food and Drug Administration reversed its opposition to a closely watched gene therapy for Huntington’s disease, clearing a path for its maker, the biotech company UniQure, to file for U.S. approval. Joining us to discuss this new development and what it means for the Huntington’s community is Lauren Holder, a Huntington’s disease advocate who is living with the early stages of the disease.<br><br>A pledge to “Make America Healthy Again” earned Robert F. Kennedy Jr. his job as the secretary of the Department of Health and Human Services. He entered the role with a long to-do list, and a goal of delivering measurable improvements within two years. How has he fared? Isabella Cueto, STAT’s chronic disease reporter, has been closely tracking Kennedy and the MAHA movement’s actions, and joins us to discuss.

406: New hope in treating Huntington's disease and a report card on RFK Jr.'s promises

The Readout Loud

# Full Transcript

**Podcast:** The Readout Loud

## Participants

- **Allison DeAngelis** (STAT)
- **Adam Feuerstein** (STAT)
- **Elaine Chen** (STAT)
- **Lauren Holder** (None)
- **Isabella Cueto** (STAT)

## Transcript

Welcome to this week's episode of The Readout Loud, a weekly biotech podcast from STAT. I'm Allison DeAngelis. I'm Adam Feuerstein. And I'm Elaine Chen. It's Thursday, June 18th, and on this week's episode, we're discussing a new hope for Huntington's disease patients. Yeah, that's right. Unicure has gotten the green light to submit its gene therapy for approval. Patient advocate Lauren Holder returns to the podcast to discuss.

Our colleague Isa Cueto is also joining to dissect how RFK Jr. is faring on his promise to remake health in America. But first, a word from our sponsor. I'm Galia Levy, SVP of Development at Kite, a Gilead company.

At Gilead and Kite, our work in oncology starts with a very simple question. How do we make progress truly meaningful for patients? Since 2020, our therapy has been used to treat more than 75,000 people worldwide with metastatic, triple negative, and HR positive HER2-nec breast cancer. We bring that same patient-first mindset to other areas of high unmet need, including CAR T-cell therapy for people with difficult to treat blood cancers.

To date, over 34,000 patients around the world have been treated with CAR T-cell therapies from our portfolio. Across oncology, this perspective guides how we design clinical trials and pursue innovation, so patients can access potential advances throughout their treatment journey. Learn more at gilead.com.

Listeners to this podcast have heard us talk quite a bit over the past year about the biotech company Unicure and its efforts to develop a gene therapy for Huntington's disease, a devastating neurodegenerative disease. Data from a small study showed its treatment slowed the progression of Huntington's by 75% after three years. Current medicines on the market don't slow disease progression. They only treat symptoms.

Efforts to bring Unicure's potentially disease-modifying treatment to patients has been delayed, though. The FDA, under former Commissioner Marty Makkari and Vinay Prasad, said Unicure study data were insufficient to support approval. Now, Makkari and Prasad are no longer at the FDA. And on Wednesday, we learned about a new and important development in the story.

Unicure and the FDA met again recently, and this time, the two sides hammered out an agreement that clears a path for Unicure to submit its gene therapy for U.S. approval. The company said that it will submit that application in the third quarter of this year. Joining us to discuss this new development and what it means for the Huntington's community is Lauren Holder. Lauren is a Huntington disease advocate who is living herself with the early stages of the disease.

She's also the producer and host of the Hope for HD podcast. Lauren, welcome back to our podcast. Thanks so much for...

406: New hope in treating Huntington's disease and a report card on RFK Jr.'s promises

Transcript

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406: New hope in treating Huntington's disease and a report card on RFK Jr.'s promises

Transcript

Built for investment professionals

Every day you wait, your competitors are findingedge in conversations you're not hearing.

Every day you wait, your competitors are finding
edge in conversations you're not hearing.