Sano blog | Lisa Conroy, MPH

thought leadership rare disease genetic testing protocol design

What the FDA's new potency guidance means for rare disease gene therapy trials

On November 2, 2026, the FDA issued its final guidance, Potency Assurance for Cellular and Gene Therapy Products, outlining a risk-based approach to ensuring th...

precision medicine patient recruitment rare disease genetic testing

FDA's new genome editing guidance: Why patient identification matters earlier in development

The FDA's draft guidance on individualized human genome editing products, issued on 27 October 2026, proposes a pathway that could allow a single biologics lice...

patient recruitment genetic testing population health

Population genomic screening is scaling. Is clinical trial infrastructure keeping up?

New findings presented at ASHG 2026 show how population genomic screening is identifying genetic risk at scale and changing preventive care. For sponsors develo...

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...

Cardiovascular genetic medicine will need a different patient-finding infrastructure

Genetic medicines are changing who cardiovascular trials need to find. Eligibility is increasingly defined by a genotype or a biomarker level, and the people wh...

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...

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...

press patient recruitment neurodegenerative disease patient engagement

Parkinson’s patients first to benefit from Sano Genetics and PKB’s new partnership

Sano Genetics and Patients Know Best (PKB) are excited to announce a new partnership to streamline and accelerate clinical trial recruitment processes, launchin...

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...

Clinical research blog

What the FDA's new potency guidance means for rare disease gene therapy trials

FDA's new genome editing guidance: Why patient identification matters earlier in development

Population genomic screening is scaling. Is clinical trial infrastructure keeping up?

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

FDA's rare disease framework could center patient identification

Cardiovascular genetic medicine will need a different patient-finding infrastructure

FDA approves emcitate for MCT8 deficiency

AATD gene editing race hinges on patient identification

Parkinson’s patients first to benefit from Sano Genetics and PKB’s new partnership

FDA approves first therapy for Sanfilippo Type A

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