Sano blog | Lisa Conroy, MPH

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...

Rare disease recruitment problems start earlier than most sponsors think

Rare disease trials have a recruitment problem, but the problem often begins well before recruitment starts.

biomarkers precision medicine patient recruitment

Autoimmune cell therapy pauses expose stratification gaps

Novartis and Bristol Myers Squibb have paused multiple trials of their autoimmune cell therapies following inflammatory side effects. Novartis placed holds on i...

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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