Sano blog | rare disease (2)

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 ...

The Genetics Podcast featuring Daniel Chung

rare disease genetic testing podcast recap

Podcast recap: Daniel Chung on the evolution of retinal gene therapy

In the latest episode of The Genetics Podcast, we spoke with Dr. Daniel Chung, Chief Medical Officer at Beacon Therapeutics. The conversation focused on how ret...

rare disease protocol design

Rethinking drug approval for ultra-rare disease: Inside the UK’s n-of-1 ASO trial

Last week, the rare disease field celebrated another breakthrough. A teenage girl with an ultra-rare genetic neurodegenerative disease was treated with a bespok...

The Genetics Podcast featuring Michelle Werner and Michael Severino

rare disease podcast recap

Podcast recap: Michelle Werner and Michael Severino on moving engineered tRNA and gene writing into the clinic

At the JPM Healthcare Conference 2026, the Genetics Podcast recorded a special episode from the Flagship Pioneering studio, bringing together two leaders advanc...

thought leadership rare disease patient engagement

Redefining accessibility in cell and gene therapies

Cell and gene therapy continues to advance across a growing number of rare genetic conditions. As more of these therapies move closer to the clinic, a central t...

Infrastructure gaps biopharma miss in rare and genetic trials

screen failure rare disease

The infrastructure gaps biopharma misses in rare and genetic trials

Rare and genetic programs increasingly depend on healthcare systems that are still adapting to the demands of precision medicine. Many of the constraints that a...

Genomics-driven drug development in pharma

precision medicine thought leadership rare disease

The forces shaping regulatory expectations in genomics-driven pharma

Regulatory expectations in genomics-driven drug development are evolving alongside scientific practice, clinical implementation, and policy. While formal guidan...

Clinical research blog