STAT+: Even at a meeting in Rome, FDA shifts are top of mind for gene therapy field

ROME — When Tim Hunt, who leads an organization for cell and gene therapy makers, reached for some reasons for optimism for the jittery field at a conference here last week, he highlighted the recent approvals of rare disease treatments from the likes of Rocket Pharmaceuticals and Regeneron Pharmaceuticals.

There was one other headline he touted as potentially promising: “FDA vaccine chief to leave the agency again.”

The allusion to Vinay Prasad — the Food and Drug Administration’s top regulator of gene and cell therapies as well as vaccines until his departure from the agency last week — was an indication that even at this event, designed to emphasize the opportunities and roadblocks the sector faces in Europe, it couldn’t escape the decisions that are coming from 4,500 miles away.

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STAT+: OpenAI wants to ‘have their cake and eat it too’ with health AI policy recommendations

So far, artificial intelligence company OpenAI’s work in health care has been largely limited to nonregulated areas. In January, the company released ChatGPT Health for consumers. Then, it launched ChatGPT for Healthcare for hospitals, followed by ChatGPT for Clinicians. 

Along with the launch of its chatbot for clinicians last month, OpenAI also published a wish list that the company described as a blueprint for unlocking AI’s potential to change the broader health care system. The policy proposals outlined in the document, experts told STAT, are somewhat reasonable, but they also disproportionately benefit the company. 

“They’re trying to have their cake and eat it too,” said David Blumenthal, a former national coordinator for health IT and a health policy professor at Harvard University. “They’re trying to potentially sound like responsible parties in the current conversation while at the same time wanting the markets to stay open for their products.”

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Opinion: Trump’s executive order on psychedelics is the right move. But is my field ready for it?

I have spent the last 10 years of my life investigating psychedelics as novel treatments for mental health conditions. When President Trump signed his executive order on psychedelic medicines, my first thought was: He got this one right. My second thought: My field may not be ready for it.

The order itself is sweeping. It directs the Food and Drug Administration to issue priority review vouchers to accelerate approval timelines, expands right-to-try pathways for ibogaine, dedicates $50 million in ARPA-H funding to psychedelic research, authorizes the Drug Enforcement Administration to speed scheduling of FDA-approved psychedelics, and launches collaboration with Veterans Affairs on therapies for veterans.

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Biomarkers of ASD/ADHD and Factors Affecting Anxiety and Depression in Children and Young Adults

Conditions: ADHD – Attention Deficit Disorder With Hyperactivity; Autism Spectrum Disorder (ASD); Developmental Coordination Disorder (DCD)

Sponsors: University of Exeter; University of Southampton; University of Dublin, Trinity College; Carol Davila University of Medicine and Pharmacy; Jimma University; FUNDACION PARA LA INVESTIGACION HOSPITAL CLINICO SAN CARLOS; The International Centre for Diarrhoeal Disease Research, Bangladesh; University of Bari Aldo Moro

Not yet recruiting

Evaluating the Impact of Ketogenic Therapy on Symptom Severity and Metabolic Side Effect Profile Among Individuals Living With Bipolar Disorder in A Rural Southern Catchment Area

Conditions: Bipolar Disorder

Interventions: Dietary Supplement: Ketogenic foodstuffs; Other: Standard of Care Oral antipsychotics; Other: Standard of care long acting injectable medication for bipolar disorder

Sponsors: University of Alabama at Birmingham; Baszucki Family Foundation; Baptist Health System Montgomery

Not yet recruiting

Ketamine With Dialectical Behavioural Therapy (DBT) for Suicidality in Individuals With Treatment-Resistant Depression and Borderline Personality Disorder (KET-DBT)

Conditions: Borderline Personality Disorder (BPD); Treatment-Resistant Major Depressive Disorder; Treatment-resistant Bipolar Depression; Major Depressive Disorder (MDD); Bipolar Disorder (BD); Bipolar Disorder I or II; Suicidal Ideation

Interventions: Drug: Ketamine Hydrochloride; Drug: Midazolam Hydrochloride; Behavioral: Dialectical Behavioral Therapy (DBT)

Sponsors: Joshua Rosenblat

Not yet recruiting

A PIVOT towards single-cell functional genetic screening in plants

Nature Biotechnology, Published online: 05 May 2026; doi:10.1038/s41587-026-03141-0

We developed PIVOT, a single-cell screening platform for functional characterization of plant genes. PIVOT enables delivery of gene libraries to tobacco leaves with single multiplicity of infection and magnetic sorting of single plant cells by phenotype. We use PIVOT to recover regulators of cytokinin signaling from an Arabidopsis open reading frame library.