Sano blog | rare disease (4)

biomarkers rare disease

The power of natural history studies in rare disease R&D

Natural history studies are central to rare disease research. Rare diseases are defined as conditions that affect less than 1 in 200,000 people in the US or les...

The Genetics Podcast featuring Max Bronstein

precision medicine rare disease podcast recap

Podcast recap: Max Bronstein on the intersection of biotech and policy

On the latest episode of The Genetics Podcast, Patrick speaks with Max Bronstein, founder and CEO of Aviva Strategies, a health policy firm dedicated to support...

rare disease genetic testing

Next-generation genetic therapies: AAV, RNA medicines, and CRISPR

Genetic medicines have been advancing at an extraordinary pace. Following the first wave of adeno-associated virus (AAV)–based gene therapies, the toolbox for g...

precision medicine rare disease

The N-of-1 revolution: What ASOs mean for rare disease families

In rare disease, families are often forced into roles that extend far beyond caregiving. When faced with diseases for which no treatment exists, parents have of...

patient recruitment rare disease

Case study: Accelerating recruitment for a gene therapy trial

In recent years, the therapeutic landscape for genetic hearing loss (GHL) has advanced rapidly, with several programs reaching clinical development. However, tr...

The Genetics Podcast featuring Benny Sorenson

rare disease podcast recap

Podcast recap: Benny Sorenson on patient-centered drug development for clotting disorders

In the latest episode of The Genetics Podcast, we sat down with Dr. Benny Sorenson, CEO of Hemab Therapeutics, to explore a relatively overlooked therapeutic ar...

FDA roundtable for cell and gene therapy

thought leadership rare disease

What we learned from the FDA’s cell and gene therapy roundtable

As part of new efforts to improve FDA processes and enhance communication with stakeholders, they hosted a roundtable on cell and gene therapy on June 5th, 2025...

precision medicine rare disease podcast recap

Podcast recap: David Bumcrot on the untapped power of regulatory RNAs

In the latest episode of The Genetics Podcast, we had a captivating conversation with Dr. David Bumcrot, Chief Scientific Officer at CAMP4 Therapeutics. From he...

The Genetics Podcast featuring Melissa Haendel

rare disease genetic testing podcast recap

Podcast recap: Melissa Haendel on building a rare disease data ecosystem from ontology to equity

In a recent episode of The Genetics Podcast, Dr. Melissa Haendel, Director of Translational Informatics and Precision Health and Professor at the University of ...

The Genetics Podcast featuring Huma Qamar