We recently attended the GBA1 conference in Montreal, which brought together researchers, clinicians, biotech leaders, and patient advocates to discuss cutting-edge developments across GBA1-associated diseases, including Parkinson’s disease (PD), Gaucher disease, and dementia. The meeting revealed major scientific progress and critical gaps in the systems surrounding patient care, trial design, and genetic access. In this blog, we’ll share some of our key learnings.
Myasthenia gravis (MG) is a chronic neuromuscular disorder (NMD) that is characterized by muscle fatigue and weakness. It is considered a rare disease, affecting between 150 and 200 cases per million people worldwide.
On a recent episode of The Genetics Podcast, Patrick spoke with Frank Gleeson, co-founder and CEO of Satellos Bioscience, a Canadian biotech pioneering a regenerative approach to Duchenne muscular dystrophy (DMD). They discussed Satellos’ focus on muscle stem cell regeneration, how this fills a critical therapeutic gap, and the promising results of their early clinical trials.
At Sano Genetics, our mission is to make clinical research in precision medicine faster, more inclusive, and more efficient. In our Q2 2025 product webinar, we unveiled new features that can help study teams streamline patient onboarding, scale testing logistics, and deliver more personalized participant experiences.
Precision medicine is reshaping how we view and treat disease. However, with that promise comes complexity: new therapies are increasingly targeted, diseases are redefined by molecular profiles, and patient populations are more fragmented than ever. Traditional clinical trial designs often can’t keep up. Accordingly, master protocols have emerged as an innovative solution that is built for the precision era.
On a recent episode of The Genetics Podcast, Patrick spoke with Tim Spector, Professor of Genetic Epidemiology at King’s College London and co-founder of Zoe. They discuss surprising insights on heritability from decades of twin research, the profound impact of the microbiome on health and disease, and the effect of diet on the gut microbiome.
Running a clinical study with biomarker or genetic testing is no small feat, especially when your operations span countries, labs, and logistics partners. For many biotechs, what seems like a straightforward requirement can quickly snowball into bottlenecks, delays, patchwork solutions, all of which add up to unexpected costs.
As the demand for personalized therapies continues to grow, trial designs must evolve to reflect the biological variability between patients. Traditional models often overlook subtle, yet important, differences in how individuals respond to treatment. Conversely, patient stratification into different subgroups based on shared characteristics can greatly improve trial efficiency and effectiveness.
On a recent episode of The Genetics Podcast, Patrick spoke with Daniel Geschwind, Senior Associate Dean and Associate Vice Chancellor of Precision Health at UCLA and Professor of Human Genetics, Neurology, and Psychiatry. They discussed how autism genetics has evolved over two decades, what makes neurodevelopment so complex, and how emerging technologies like RNA therapeutics could transform care for rare genetic forms of autism.
News of a ‘CRISPR baby’ broke this week, almost seven years after the first CRISPR baby scandal unleashed a media frenzy and years of ethical debates. But this time, the story marks a very different chapter in the evolution of human gene editing. Rather than an unregulated experiment carried out in secrecy, this new case involved a safe, custom-designed CRISPR therapy, developed with clinical oversight, to treat a life-threatening genetic disorder in a newborn. The therapy targeted a disease-causing mutation in the infant’s DNA and was administered after birth, not at the embryonic stage.