Sano blog | genetic testing

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FDA approves emcitate for MCT8 deficiency

On September 28, 2026, the FDA approved Emcitate (tiratricol) to treat peripheral thyrotoxicosis in patients with MCT8 deficiency, also known as Allan-Herndon-D...

precision medicine patient recruitment rare disease genetic testing

AATD gene editing race hinges on patient identification

In September 2026, three in vivo gene editing programs reported progress against the same rare disease, aimed at the same mutation. On September 7, YolTech repo...

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FDA approves first therapy for Sanfilippo Type A

On 17 September 2026, the FDA granted standard full approval to FAYUVI (rebisufligene etisparvovec-hopf), a single-dose intravenous AAV9 gene therapy from Ultra...

The Genetics Podcast featuring Marleah Dean Kruzel

genetic testing podcast recap

Podcast recap: Marleah Dean Kruzel on reframing hereditary cancer uncertainty

In the latest episode of The Genetics Podcast, Patrick sits down with Dr. Marleah Dean Kruzel, Professor of Communication at the University of South Florida. He...

precision medicine patient recruitment rare disease genetic testing protocol design

FDA draft guidance expands single-trial effectiveness pathway

In June 2026, the FDA released a revised draft guidance, Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products, that clarif...

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Genomic newborn screening: The operational challenge ahead

A Nature News Feature published on 19 August 2026 synthesized the global wave of genomic newborn screening programs and opened with a concrete result. In the GU...

precision medicine patient recruitment rare disease genetic testing

OptiPrime speeds prime editing but patient identification lags

On August 12, 2026, the David Liu lab at the Broad Institute published OptiPrime, a machine-learning model that predicts how well prime-editing guide RNAs (pegR...

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Inherited genetics linked to CAR-T toxicity and expansion

CAR-T therapies carry meaningful safety risks, and toxicity is known to vary with factors including disease characteristics, patient factors and the therapy its...

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Why precision medicine trials fail on the way from protocol to patient

Precision medicine clinical trials are designed by some of the most capable scientists in drug development. The biomarker hypotheses are well-reasoned, the endp...

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...

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