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Clinical research blog

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

How Sano Genetics accelerated recruitment for a Parkinson’s disease gene therapy trial

Recruitment for genetically stratified clinical trials is a critical bottleneck for sponsors. Identifying individuals with specific variants, building trust around complex therapies, and keeping people engaged throughout the process can create friction that slows progress.

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Podcast recap: Rachel Salzman on vectorized RNAi for neuromuscular disease

In the last episode of The Genetics Podcast, Patrick spoke with Dr. Rachel Salzman, CEO of Armatus Bio, a biotech company developing vectorized RNA interference (RNAi) for autosomal dominant neuromuscular diseases. The lead programs target facioscapulohumeral muscular dystrophy (FSHD) and Charcot-Marie-Tooth disease type 1A (CMT1A), using AAV to deliver engineered microRNAs for long term silencing of disease genes.

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Psychiatric precision medicine: What’s actually changed for trials in 2025

Psychiatric precision medicine has been a promising area of research for nearly two decades, but only recently has data emerged that can influence real clinical trial design. In 2025, several developments suggest that stratification based on genetics, pharmacokinetics, and mechanistic biology is becoming more actionable. New genetic responder signals, validated EEG-based subtypes, and the first biomarker-enriched depression trial have pushed the field past its long-standing theoretical phase.

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Podcast recap: Slavé Petrovski on what millions of genomes can actually teach us

In the last episode of The Genetics Podcast, Patrick spoke with Dr. Slavé Petrovski, Vice President of AstraZeneca’s Center for Genomics Research. Slavé oversees one of the largest human genomics resources in industry and uses it to support target discovery, biomarker development, disease prediction, and other programs of work. The conversation covered his career path, how large biobanks change the questions we can ask, how to identify protective biology in populations, and why computational sustainability now matters as much as technological advances.

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How spatial omics is reshaping biomarker development in neuroscience

Spatial omics have revolutionized biomedical research in recent years. The use of spatial techniques unlocks cell-specific understanding of disease, which supports the development of precision therapies aimed at the right cells and pathways. This has been especially valuable in oncology, where cellular heterogeneity and cell-to-cell interactions influence tumor development and treatment response. 

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Behind the scenes at Sano: Building the infrastructure that powers precision medicine

At Sano, every product, pipeline, and participant experience depends on secure, reliable, and scalable infrastructure. That foundation is shaped by our platform engineering team, who work mostly behind the scenes so that everyone else can move faster and more safely.

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Podcast recap: Ellen Reisinger on gene therapy for hereditary deafness

In the last episode of The Genetics Podcast, we spoke with Dr. Ellen Reisinger, Professor at the University of Tübingen. Ellen has spent nearly twenty years studying the molecular mechanisms of hearing, particularly the role of otoferlin (OTOF) in inner hair cell neurotransmission. Her early work was fully basic science, focused on gene expression during cochlear development and the fundamental biology of hair cell function.

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3 recent signals that hint at the next phase of rare disease development

Recent activity in the US and UK offers important signals for how personalized and rare disease therapies may evolve. Scientific teams are building structured editing platforms, regulators are considering new evidence models, and national systems are exploring flexible licensing approaches. Taken together, these shifts reflect growing recognition that traditional development and regulatory pathways cannot accommodate the rising number of gene-specific and variant-specific therapies. They point toward practical strategies that could make next-generation therapies more feasible to develop and deliver. This blog highlights three developments and what they could mean for future sustainability and commercial viability in precision medicine.

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The evolution of cell and gene therapy: 6 themes shaping the next phase of growth

At the recent Evolution of the Cell and Gene Therapy (CGT) Sector Workshop by the Alliance for Regenerative Medicine (ARM), speakers from across industry, academia, and advocacy explored what needs to happen next for cell and gene therapies to reach full maturity. The consensus was clear that innovation in CGT is progressing faster than the systems that support it. The next challenge is to make these therapies scalable, reliable, and accessible. In this blog, we cover six key themes that emerged from the discussions.

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Podcast recap: Ananth Sridhar and Sun-Gou Ji on building a sustainable model for rare disease innovation

On the latest episode of The Genetics Podcast, we spoke with Ananth Sridhar, Chief Operating Officer for cardiorenal programs at BridgeBio, and Sun-Gou Ji, the company’s Vice President of Computational Genomics. The conversation explored how BridgeBio’s distinctive hub-and-spoke model enables sustainable rare disease drug development, how genetics drives their discovery work, and why the right economic model is essential for tackling small patient populations.

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