Sano blog | rare disease (7)

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...

rare disease genetic testing

Challenges in developing new therapies for Inherited Retinal Disorders (IRD)

Inherited retinal diseases (IRDs) are a leading cause of genetic vision loss, yet they remain difficult to identify and diagnose. Genetic testing plays a pivota...

precision medicine rare disease genetic testing

Eyeing the future: Genetic insights driving personalized eye care

Over 350 hereditary eye conditions are currently known, collectively affecting around one in every 1,000 people globally. These genetic disorders can influence ...

precision medicine rare disease genetic testing

Seeing the future: Breakthroughs in genetic eye disease diagnosis and therapy [Whitepaper]

Genetic eye diseases present significant challenges for patients and healthcare providers alike. Conditions like retinitis pigmentosa, Stargardt disease, and Le...

rare disease genetic testing podcast recap

Podcast recap: Exploring the future of AAV gene therapy with Paul Wille

In the latest episode of the Genetics Podcast, host Patrick Short spoke with Paul Wille, Director of Product Development at Abeona Therapeutics, about adeno-ass...

Clinical research blog