Sano blog | rare disease

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FDA potency assurance guidance reshapes CGT development

FDA issued its final guidance Potency Assurance for Cellular and Gene Therapy Products on November 2, 2026, and a Federal Register notice of availability follow...

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The FDA's draft guidance on individualized human genome editing products, issued on 27 October 2026, proposes a pathway that could allow a single biologics lice...

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...

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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...

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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...

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...

Clinical research blog

FDA potency assurance guidance reshapes CGT development

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

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

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