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Clinical research blog

Explore our blog for insights into the big questions in precision medicine and clinical research.

The future of AI in clinical research: A conversation with Sano’s CTO

At Sano Genetics, our mission is to deliver and facilitate clinical trials that accelerate the development of precision medicine treatments for patients. As a company rooted in data, analytics, and software, we are constantly attuned to technological advancements that can make our work faster, more effective, and more impactful. This mindset drives us to continuously explore and adopt tools that improve operational efficiency and empower our team to work more productively.

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Podcast recap: Melissa Haendel on building a rare disease data ecosystem from ontology to equity

In a recent episode of The Genetics Podcast, Dr. Melissa Haendel, Director of Translational Informatics and Precision Health and Professor at the University of North Carolina at Chapel Hill and Phenotypic Lead at Alamya Health, joined the show to discuss how data integration, patient voice, and national-scale collaboration can transform diagnosis and care for rare diseases. As the co-founder of the Monarch Initiative and a driving force behind the Mondo disease ontology, Melissa is helping shape the future of equitable and precise genomic medicine.

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Podcast recap: Huma Qamar on pioneering gene therapy for retinal disease

In a recent episode of The Genetics Podcast, Dr. Huma Qamar, Chief Medical Officer at Ocugen, shares her extraordinary journey from a small town in Pakistan to the forefront of next-generation gene therapy in the United States. A physician, scientist, and public health advocate, Dr. Qamar is leading Ocugen’s efforts to develop durable, one-time gene therapies for rare inherited eye diseases such as retinitis pigmentosa (RP), Stargardt disease, and geographic atrophy.

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Podcast recap: Frank Gentile on harnessing autophagy to treat neurodegenerative diseases

In a recent episode of The Genetics Podcast, we talked to Frank Gentile, Chief Executive Officer of Casma Therapeutics, to explore how autophagy could be a valuable target for treating neurodegenerative and lysosomal diseases. Coming from a venture capital background, Frank offered a fresh and strategic perspective on the science being done at Casma. 

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Smarter trials with AI: For engineers and data teams

Technological advances and biomedical breakthroughs have triggered a paradigm shift in precision medicine. Clinical research is currently at a turning point at which scaling up processes in an efficient manner and adapting clinical trial models to fulfill changing requirements will help transform the ecosystem. The availability of large-scale datasets and maturing of artificial intelligence (AI) tools that can process them at scale can help fill persistent gaps in clinical research and traditional models.

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AI at the FDA: A new era for drug development and precision medicine

In May and June of 2025, the Food and Drug Administration (FDA) announced a series of important developments related to the use of artificial intelligence (AI) across its processes. These developments signaled a significant move towards integrating technological advancements into the FDA’s routine tasks to enhance productivity. 

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Personalized medicine in Duchenne muscular dystrophy

Duchenne muscular dystrophy is an inherited disorder that is characterized by progressive muscle degeneration, leading to loss of function and premature death. The global prevalence of DMD is approximately 19.8 per 100,000 live male births. As our understanding of the genetic and molecular underpinnings of DMD deepens, so too does the potential for highly personalized treatments that are tailored to individual genetic profiles. 

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Podcast recap: Andres Moreno-Estrada on mapping Latin America's genetic legacy and its future

On a recent episode of The Genetics Podcast, Patrick was joined by Andres Moreno-Estrada, population geneticist and head of the Human Evolutionary Genomics Lab at LANGEBIO in Mexico. They discussed the creation and insights of the Mexican Biobank, the genetic diversity of Latin America, ancient human migration, and the role of locally-led research in shaping public health and scientific equity.

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Insights from the GBA1 conference: A call for precision and inclusion

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.

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How precision medicine is advancing myasthenia gravis treatment

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. 

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