Sano blog (6)

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...

patient recruitment patient engagement

From click to randomization: What truly predicts trial success?

Digital recruitment has become a standard part of clinical trial execution. Sponsors invest heavily in online outreach, patient referrals, and awareness campaig...

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...

public health genetic testing podcast recap

Podcast recap: Zornitza Stark on real-world impact from newborn genomic screening

As genetic medicine has continued to advance, experts have consistently highlighted the importance of newborn genomic screening as a practical avenue for early ...

Site patient estimates for clinical trials

screen failure patient recruitment

Understanding the gap between feasibility estimates and patient availability

Patient availability is often overestimated during trial planning. This usually starts at feasibility, where sites are asked to report how many patients they ha...

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...

Effective site enablement in precision medicine

patient recruitment patient engagement

What strong site enablement really looks like: Lessons from Sano’s site expert

Trial sponsors often assume that site enablement is largely complete once a trial launches. Feasibility assessments, training materials, and startup timelines a...

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...

Clinical research blog