Sano blog | rare disease (8)

rare disease podcast recap

Podcast recap: Michelle Werner on tRNA therapeutics, basket trials, and innovation in rare disease

In a recent episode of The Genetics Podcast, Patrick sat down with Michelle Werner, CEO of Alltrna and CEO Partner at Flagship Pioneering. If you’ve been follow...

patient recruitment rare disease

Clinical trial recruitment: Conversion and compliance

Most clinical trials fail to recruit enough participants on time. In rare disease and genetically stratified studies, the challenge is compounded: the patient p...

patient recruitment rare disease genetic testing

Key takeaways from an important conversation about IRD research

On October 18th, 2024 20/20 Onsite, Sano Genetics, and Clinical Enrollment announced the launch of a new partnership to offer a comprehensive solution for ident...

genetic epidemiology rare disease podcast recap

Podcast recap: Dr. Hilary Martin on genetic complexity in neurodevelopmental disorders

In the latest episode of The Genetics Podcast, host Patrick Short is joined by Dr. Hilary Martin, Group Leader in Human Genetics at the Wellcome Sanger Institut...

Innovation and regulation in gene and cell therapy

precision medicine rare disease

Gene therapy regulation: FDA-accelerated approval & orphan drug designation

As of March 2023, 3,900 gene therapy trials were noted as ongoing, completed, or approved across 46 different countries. An analysis of the second quarter of 20...

rare disease patient engagement

Areas for patient-centric innovation in cell and gene therapy research

There are strong indications that patient acceptance of cell and gene therapies increases significantly following the provision of clear, easy-to-understand inf...

Gene and cell therapy: How innovation is changing rare disease treatment

precision medicine rare disease genetic testing

Cell and gene therapy: How innovation is changing rare disease treatment

Cell and gene therapies (CGTs) are reshaping what it means to treat disease. Instead of managing symptoms over a lifetime, these approaches aim to address disea...

rare disease patient engagement

Aligning innovation with patient needs in gene and cell therapy development

Gene and cell therapies represent a fundamental shift in how rare diseases are treated. Rather than managing symptoms, these therapies target the underlying gen...

Webinar recap: Breakthroughs and barriers in rare disease drug development

patient recruitment rare disease

Rare disease drug development: Barriers and solutions

Rare disease drug development operates under a unique set of structural constraints. Patient populations are small and dispersed, diagnostic timelines are long,...

Michelle Teng ơn the genetics podcast featured image

precision medicine rare disease podcast recap

Podcast recap: Michelle Teng on transforming immunotherapy with machine learning

Last week on The Genetics Podcast, Patrick Short sat down with Michelle Teng, CEO and co-founder of Etcembly, to explore her work at the intersection of immunot...

Clinical research blog