Sano blog | rare disease

precision medicine patient recruitment rare disease genetic testing

What BEAM-301 reveals about the next challenge in precision medicine trials

Beam Therapeutics' early clinical results highlight the promise of mutation-specific therapies. But as treatments become more genetically targeted, they also ra...

precision medicine patient recruitment rare disease protocol design

FDA's rare disease framework could center patient identification

An FDA notice seeking public input is under review at the Office of Management and Budget, according to a Citeline report on October 12, 2026. The notice sugges...

thought leadership patient recruitment rare disease genetic testing

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...

The Genetics Podcast featuring Jenny Hsieh

rare disease podcast recap

Podcast recap: Jenny Hsieh on why women with Danon disease are being missed

Danon disease is X-linked, and much of the research to date has focused on males. In the latest episode of The Genetics Podcast, Patrick is joined by Jenny Hsie...

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...

precision medicine thought leadership industry report rare disease

Genetic eligibility shows up earlier in the pipeline than most plans assume

During enrollment planning, genetic eligibility is often treated as a therapy area question, settled once a program knows which organ system it targets. Genetic...

patient recruitment rare disease genetic testing neurodegenerative disease

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...

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FDA draft guidance expands single-trial effectiveness pathway

In June 2026, the FDA released a revised draft guidance, Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products, that clarif...

patient recruitment rare disease patient engagement

Long-term follow-up in clinical trials is a compounding asset for rare disease programs

Rare disease trials demand years of investment to identify, genetically screen, enroll, and engage patients. When the study closes, that effort disperses. Commu...

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...

Clinical research blog

What BEAM-301 reveals about the next challenge in precision medicine trials

FDA's rare disease framework could center patient identification

FDA approves emcitate for MCT8 deficiency

Podcast recap: Jenny Hsieh on why women with Danon disease are being missed

AATD gene editing race hinges on patient identification

Genetic eligibility shows up earlier in the pipeline than most plans assume

FDA approves first therapy for Sanfilippo Type A

FDA draft guidance expands single-trial effectiveness pathway

Long-term follow-up in clinical trials is a compounding asset for rare disease programs

OptiPrime speeds prime editing but patient identification lags

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