Sano blog | rare disease (7)

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...

rare disease podcast recap

Podcast recap: Building Noga Therapeutics and advancing gene therapy for rare diseases with Noam Baumatz

In the most recent episode of The Genetics Podcast, host Patrick Short speaks with Noam Baumatz, co-founder and CEO of Noga Therapeutics. Noam shares his deeply...

rare disease neurodegenerative disease

Innovating for impact: How natural history studies pave the way for rare disease therapies [webinar recap]

In a recent webinar, former Reata Pharmaceuticals CEO Warren Huff shared his journey leading the company from its founding in 2002 to its landmark acquisition b...

industry report rare disease genetic testing

Exploring inherited retinal disorders (IRDs): Causes, challenges, and future treatments [report]

Inherited retinal disorders (IRDs) are a diverse group of genetic eye conditions that impact millions worldwide, affecting light-sensitive cells in the retina a...

Clinical research blog