Sano blog | rare disease

precision medicine rare disease

The rare disease blueprint: What precision medicine can learn from the hardest trials

Rare disease exposes the limits of current clinical development models. Small, fragmented populations make patient identification difficult. Heterogeneous biolo...

The Genetics Podcast featuring Derek Ansel

rare disease podcast recap

Podcast recap: Derek Ansel on decision-making under uncertainty in rare disease clinical trials

Rare disease drug development involves decision-making under extreme uncertainty. Teams are asked to design trials without well-established endpoints, in small ...

Standardizing genetic testing and processing

rare disease genetic testing

Connecting patients to rare disease trials through scalable genetic testing infrastructure

At Seqera Sessions London 2026, Dr. Katie Barnes, Head of Clinical Genetics at Sano Genetics, outlined a practical challenge facing the field: how to move from ...

The Genetics Podcast featuring Anna Lindstrand

rare disease long-read sequencing podcast recap

Podcast recap: Anna Lindstrand on implementing short-read and long-read sequencing for rare disease diagnostics

Genome sequencing is now a core part of rare disease diagnostics in several healthcare systems. However, the path from sequencing technology to clinical impact ...

Patient engagement in rare disease trials

rare disease patient engagement

Patient engagement and education insights from rare disease trials

Recruiting and retaining patients in rare disease and genomic medicine research requires more than outreach. Many participants need support understanding geneti...

The Genetics Podcast featuring Matthew Goldstein

rare disease genetic testing podcast recap

Podcast recap: Matthew Goldstein on expanding access to genetic screening

Genetic screening has advanced rapidly over the past two decades. Sequencing is faster and far less expensive, and the ability to interpret genetic variants con...

patient recruitment rare disease

Why site selection is failing rare disease trials and what to do differently

Rare disease trials operate under structural constraints that make site selection more important than in common disease programs. Patient populations are small,...

rare disease patient engagement

Rare disease drug development requires designing for access from day one

Rare disease innovation has accelerated in recent years, particularly in cell and gene therapy (CGT). Yet for drug developers, one challenge remains persistent:...

The Genetics Podcast featuring David Dismuke and Steven Gray

rare disease podcast recap

Podcast recap: David Dismuke and Steven Gray on scaling AAV gene therapy

Adeno-associated virus (AAV) gene therapy has moved from proof of concept to approved products. As more programs enter the clinic and target larger patient popu...

rare disease patient engagement

Barriers to long-term follow-up in cell and gene therapy

Cell and gene therapy (CGT) programs are committing to 10-15 years of follow-up – often without the infrastructure to support it. FDA guidance for certain gene ...

Clinical research blog

The rare disease blueprint: What precision medicine can learn from the hardest trials

Podcast recap: Derek Ansel on decision-making under uncertainty in rare disease clinical trials

Connecting patients to rare disease trials through scalable genetic testing infrastructure

Podcast recap: Anna Lindstrand on implementing short-read and long-read sequencing for rare disease diagnostics

Patient engagement and education insights from rare disease trials

Podcast recap: Matthew Goldstein on expanding access to genetic screening

Why site selection is failing rare disease trials and what to do differently

Rare disease drug development requires designing for access from day one

Podcast recap: David Dismuke and Steven Gray on scaling AAV gene therapy

Barriers to long-term follow-up in cell and gene therapy

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