Sano blog | rare disease

precision medicine rare disease patient engagement

Who tells patients when a gene therapy program pauses?

When a gene therapy sponsor pauses a clinical program, the operational machinery responds within hours. The medical monitor convenes a safety review. Regulatory...

precision medicine patient recruitment rare disease genetic testing patient engagement

Operation Trailblazer and the clinical trial access gap

On June 23, 2026, the US Department of Health and Human Services launched Operation Trailblazer, a cross-agency initiative coordinated across the FDA, NCI, NCAT...

Huntington's disease patient recruitment rare disease genetic testing neurodegenerative disease

What two FDA gene therapy reversals in one week means for rare disease sponsors

In the space of five days, the FDA reversed its position on two rare disease gene therapies it had previously rejected. On June 17, the agency agreed that UniQu...

The Genetics Podcast featuring Timothy Yu

precision medicine rare disease podcast recap

Podcast recap: Tim Yu on the rise of N-of-1 genetic medicines

Most genetic medicine is built for populations large enough to support a clinical trial. N-of-1 medicine is built for the opposite case: a single patient whose ...

precision medicine rare disease

The rare disease blueprint: What precision medicine can learn from the hardest trials

Rare disease exposes the limits of current clinical development models. Small, fragmented populations make patient identification difficult. Heterogeneous biolo...

The Genetics Podcast featuring Derek Ansel

rare disease podcast recap

Podcast recap: Derek Ansel on decision-making under uncertainty in rare disease clinical trials

Rare disease drug development involves decision-making under extreme uncertainty. Teams are asked to design trials without well-established endpoints, in small ...

Standardizing genetic testing and processing

rare disease genetic testing

Connecting patients to rare disease trials through scalable genetic testing infrastructure

At Seqera Sessions London 2026, Dr. Katie Barnes, Head of Clinical Genetics at Sano Genetics, outlined a practical challenge facing the field: how to move from ...

The Genetics Podcast featuring Anna Lindstrand

rare disease long-read sequencing podcast recap

Podcast recap: Anna Lindstrand on implementing short-read and long-read sequencing for rare disease diagnostics

Genome sequencing is now a core part of rare disease diagnostics in several healthcare systems. However, the path from sequencing technology to clinical impact ...

Patient engagement in rare disease trials

rare disease patient engagement

Patient engagement and education insights from rare disease trials

Recruiting and retaining patients in rare disease and genomic medicine research requires more than outreach. Many participants need support understanding geneti...

The Genetics Podcast featuring Matthew Goldstein

rare disease genetic testing podcast recap

Podcast recap: Matthew Goldstein on expanding access to genetic screening

Genetic screening has advanced rapidly over the past two decades. Sequencing is faster and far less expensive, and the ability to interpret genetic variants con...

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