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Clinical research blog

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

New study data shows a better way to find patients with liver disease and what it means for clinical trials

Finding patients who qualify for a clinical trial has always been one of the hardest parts of running one. In liver disease, particularly MASLD, it is especiall...
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Podcast recap: Vincent Dion on hijacking DNA repair machinery to treat Huntington's disease

More than three decades after the Huntington's disease gene was cloned in 1993, there is still no approved therapy that alters the course of the disease. Recent...
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Reducing screen failure starts with better trial design

When a clinical trial misses its enrollment targets, the instinct is often to look at recruitment through more channels, more outreach, and a bigger advertising...
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Podcast recap: Paul Valdmanis on long-read sequencing and the genetics of neurodegeneration

In the most recent episode of The Genetics Podcast, host Patrick Short speaks with Dr. Paul Valdmanis, Associate Professor at the University of Washington, abou...
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What four episodes of The Genetics Podcast reveal about the future of Alzheimer’s precision medicine

Alzheimer’s research is entering a new phase. For decades, the field has been shaped by the biology of amyloid plaques and tau tangles. Those remain central to ...
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Podcast recap: Sarah Marzi on how genetics, environment, and cell state shape neurodegenerative disease

In the most recent episode of The Genetics Podcast, Patrick Short speaks with Dr. Sarah Marzi, Senior Lecturer at King’s College London and Group Leader at the ...
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The long game: Precision trials shouldn’t start from zero every time

Precision medicine sponsors invest heavily to identify, educate, screen, consent, genotype, and support rare patients. In many programs, once a trial ends, that...
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Trust is the foundation of precision medicine

UK Biobank and similar resources have made an extraordinary contribution to biomedical research, enabling important advances across genomics, population health,...
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How Sano enables precision patient finding at scale

Recruitment in precision medicine and rare disease trials remains constrained by fragmented data, low prevalence populations, and heavy reliance on site-based p...
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Podcast recap: Suzanne Schindler on Alzheimer’s biomarkers

On the latest episode of The Genetics Podcast, Patrick Short speaks with Dr. Suzanne Schindler associate professor of neurology at Washington University in St. ...
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The rare disease blueprint: What precision medicine can learn from the hardest trials

Rare disease exposes the limits of current clinical development models. Small, fragmented populations make patient identification difficult. Heterogeneous biolo...
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Podcast recap: Derek Ansel on decision-making under uncertainty in rare disease clinical trials

Rare disease drug development involves decision-making under extreme uncertainty. Teams are asked to design trials without well-established endpoints, in small ...
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Podcast recap: Tim Jobson on earlier liver disease detection and the LiveWell study

Liver disease remains one of the few major disease areas where outcomes have not improved in line with other fields such as cardiovascular disease and cancer. I...
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Genetic literacy: Prevent drop-off in trial recruitment

In rare disease and genetically stratified trials, recruitment often depends on a single critical step: confirming that a patient carries the relevant genetic v...
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The psychology of kit return: What drives completion?

Genetic testing has become a core component of patient identification and stratification in modern clinical trials, particularly in rare disease and precision m...
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Podcast recap: Andrea Ganna on applying polygenic scores and EHRs in healthcare

On the latest episode of The Genetics Podcast, Patrick welcomed Dr. Andrea Ganna, an Associate Professor at the Institute for Molecular Medicine Finland (FIMM),...
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Connecting patients to rare disease trials through scalable genetic testing infrastructure

At Seqera Sessions London 2026, Dr. Katie Barnes, Head of Clinical Genetics at Sano Genetics, outlined a practical challenge facing the field: how to move from ...
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How strong eligibility signals unlocked scale in Parkinson’s trials

Recruitment in genetically stratified clinical trials is often constrained by a simple problem: large screening volumes do not translate into eligible patients....
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Precision liver study completes 1-year recruitment of nearly 1,000 participants using existing NHS data

Somerset, UK, 18th March 2026: Predictive Health Intelligence (PHI) and Sano Genetics today announced the completion of recruitment into the LiveWell study, wit...
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Podcast recap: Anna Lindstrand on implementing short-read and long-read sequencing for rare disease diagnostics

Genome sequencing is now a core part of rare disease diagnostics in several healthcare systems. However, the path from sequencing technology to clinical impact ...
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Patient engagement and education insights from rare disease trials

Recruiting and retaining patients in rare disease and genomic medicine research requires more than outreach. Many participants need support understanding geneti...
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Podcast recap: Matthew Goldstein on expanding access to genetic screening

Genetic screening has advanced rapidly over the past two decades. Sequencing is faster and far less expensive, and the ability to interpret genetic variants con...
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Why site selection is failing rare disease trials and what to do differently

Rare disease trials operate under structural constraints that make site selection more important than in common disease programs. Patient populations are small,...
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Rare disease drug development requires designing for access from day one

Rare disease innovation has accelerated in recent years, particularly in cell and gene therapy (CGT). Yet for drug developers, one challenge remains persistent:...
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Podcast recap: Ryan Dhindsa and Caleb Lareau on using biobank genomics to measure EBV persistence

In a recent episode of The Genetics Podcast, Patrick Short spoke with Dr. Ryan Dhindsa, Assistant Professor at Baylor College of Medicine and Investigator at th...
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Podcast recap: Nicky Whiffin on the discovery of ReNU syndrome and what it unlocks for genetic medicine

The majority of rare disease discovery has been linked to protein coding genes, where changes are easier to interpret and easier to capture in routine testing. ...
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Podcast recap: David Dismuke and Steven Gray on scaling AAV gene therapy

Adeno-associated virus (AAV) gene therapy has moved from proof of concept to approved products. As more programs enter the clinic and target larger patient popu...
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Barriers to long-term follow-up in cell and gene therapy

Cell and gene therapy (CGT) programs are committing to 10-15 years of follow-up – often without the infrastructure to support it. FDA guidance for certain gene ...
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Podcast recap: Daniel Chung on the evolution of retinal gene therapy

In the latest episode of The Genetics Podcast, we spoke with Dr. Daniel Chung, Chief Medical Officer at Beacon Therapeutics. The conversation focused on how ret...
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From click to randomization: What truly predicts trial success?

Digital recruitment has become a standard part of clinical trial execution. Sponsors invest heavily in online outreach, patient referrals, and awareness campaig...
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Rethinking drug approval for ultra-rare disease: Inside the UK’s n-of-1 ASO trial

Last week, the rare disease field celebrated another breakthrough. A teenage girl with an ultra-rare genetic neurodegenerative disease was treated with a bespok...
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Podcast recap: Zornitza Stark on real-world impact from newborn genomic screening 

As genetic medicine has continued to advance, experts have consistently highlighted the importance of newborn genomic screening as a practical avenue for early ...
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Understanding the gap between feasibility estimates and patient availability

Patient availability is often overestimated during trial planning. This usually starts at feasibility, where sites are asked to report how many patients they ha...
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Podcast recap: Michelle Werner and Michael Severino on moving engineered tRNA and gene writing into the clinic

At the JPM Healthcare Conference 2026, the Genetics Podcast recorded a special episode from the Flagship Pioneering studio, bringing together two leaders advanc...
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What strong site enablement really looks like: Lessons from Sano’s site expert

Trial sponsors often assume that site enablement is largely complete once a trial launches. Feasibility assessments, training materials, and startup timelines a...
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Redefining accessibility in cell and gene therapies

Cell and gene therapy continues to advance across a growing number of rare genetic conditions. As more of these therapies move closer to the clinic, a central t...
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The infrastructure gaps biopharma misses in rare and genetic trials

Rare and genetic programs increasingly depend on healthcare systems that are still adapting to the demands of precision medicine. Many of the constraints that a...
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Podcast recap: Arabella Bouzigues on building a global research infrastructure for frontotemporal dementia

In a recent episode of The Genetics Podcast, host Patrick Short talked with Dr. Arabella Bouzigues, postdoctoral researcher and coordinator of the Genetic Front...
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The forces shaping regulatory expectations in genomics-driven pharma

Regulatory expectations in genomics-driven drug development are evolving alongside scientific practice, clinical implementation, and policy. While formal guidan...
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Podcast recap: Lisa Gurry on scaling early genetic diagnosis at GeneDx

In the latest episode of The Genetics Podcast, Patrick spoke with Lisa Gurry, Chief Business Officer at GeneDx. The conversation focuses on how GeneDx has built...
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Lessons from recent FDA actions in precision medicine 

Over the past few years, several cell and gene therapies (CGTs) in precision medicine have received FDA Complete Response Letters (CRLs). As the agency increase...
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Podcast recap: Jeffrey Chamberlain on four decades of progress in gene therapy for muscular dystrophy

In the latest episode of The Genetics Podcast, Patrick spoke with Dr. Jeffrey Chamberlain, Professor at the University of Washington School of Medicine and Dire...
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Top five regulatory signals sponsors can act on now

The FDA’s activity in 2025 reflected a mix of scientific progress and operational instability. Precision medicine programs benefitted from new draft guidance, r...
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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 aro...
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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...
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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...
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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 oversee...
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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 suppo...
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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...
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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 stu...
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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 e...
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Cell and gene therapy: Systems gaps shaping next scale

At the recent Evolution of the Cell and Gene Therapy (CGT) Sector Workshop by the Alliance for Regenerative Medicine (ARM), speakers from across industry, acade...
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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 ...
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4 foundations for making AI and data ready for clinical development

AI is becoming a core component of drug development and clinical research. Models are improving and regulatory frameworks are evolving, but progress still depen...
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From fragmentation to fusion: How AI is unlocking the value of multi-omic data in drug discovery

In drug discovery, data is the key to success. Although more and more large-scale data is being generated every year, much of it is inaccessible or fragmented. ...
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Podcast recap: Justin Porcano on speeding up treatments for Usher syndrome type 1B

In the last episode of The Genetics Podcast, Patrick spoke with Justin Porcano, co-founder and executive director of Save Sight Now, a nonprofit accelerating th...
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Scaling the promise of gene editing: Why progress still falls short of potential

Gene editing has progressed significantly over the past decade, evolving from an experimental technology that was associated with safety and efficacy limitation...
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Podcast recap: Heidi Rehm and Slavé Petrovski live from ASHG on breaking barriers in genomics

In a special live episode of The Genetics Podcast, recorded at the American Society of Human Genetics (ASHG) meeting, host Patrick Short was joined by two of th...
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Podcast recap: Chris Hopkins on developing a one-time cell therapy for Fabry disease

On this episode of The Genetics Podcast, Patrick spoke with Chris Hopkins, CEO of Glafabra Therapeutics, a company developing a one-time autologous cell therapy...
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Innovation in AAV: Breaking through bottlenecks in yield, safety, and cost

At this year’s European Society of Gene & Cell Therapy (ESGCT) meeting, one theme resonated across sessions: adeno-associated viruses (AAVs) are entering a ...
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Closing the communication gap: A sponsor’s guide to building patient trust and retention

Participation in clinical trials can be burdensome and disruptive. In addition to practical inconveniences and discomfort, participants may have to take time of...
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Podcast recap: The state of AAV gene therapy and where it’s headed

On the most recent episode of The Genetics Podcast, host Patrick Short took a solo deep dive into gene therapy, with a focus on therapies based on delivery via ...
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Expediting study start-up: Best practices for sponsors

Study start-up is a critical stage of the drug development process and can have a major impact on trial timelines, budgets, and overall success. Start-up activi...
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Podcast recap: Sonya Dumanis and Andy Singleton on building a global genetics engine for Parkinson’s

On the most recent episode of The Genetics Podcast, we hosted Sonya Dumanis and Andy Singleton to unpack GP2, the Global Parkinson’s Genetics Program. GP2 is a ...
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Clinical trial site partnerships in 2025: What sponsors need to know

At the Partnerships with Sites summit in September 2025, new data from the Tufts Center for the Study of Drug Development (Tufts CSDD) underscored how the globa...
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Behind the scenes at Sano: Navigating the patient journey

Advocating for patients is one of the core pillars of Sano’s mission. Our approach to clinical trials is patient-centered and entails deep and meaningful engage...
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Overcoming rare disease trial hurdles: Q&A with Sano's Head of Clinical Engagement 

Rare disease trials are among the most challenging to design and execute. Patient populations are small and hard to reach, and personal and disease-related burd...
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Podcast recap: Wanda Smith on turning a family diagnosis into CureGRN

In last week’s episode of The Genetics Podcast, Patrick Short sat down with Wanda Smith, founder of CureGRN and long-time advocate for families affected by prog...
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Building trust in clinical trial patient matching: Takeaways from DPHARM 2025

At this year’s DPHARM conference, Sano’s VP of Commercial, Ben Jackson, joined a panel on clinical trial patient matching moderated by a clinical expert from No...
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Podcast recap: Terry Pirovolakis on turning his son’s diagnosis into Elpida Therapeutics

On the most recent episode of The Genetics Podcast, we hosted Terry Pirovolakis, founder and CEO of Elpida Therapeutics. Terry’s journey began the day his young...
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Q3 product spotlight: New testing options and smarter operations

At Sano, our mission is to accelerate precision medicine research through technology, making it easier for sponsors to run trials efficiently and effectively. E...
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The power of natural history studies in rare disease R&D

Natural history studies are central to rare disease research. Rare diseases are defined as conditions that affect less than 1 in 200,000 people in the US or les...
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Podcast recap: Samuli Ripatti on turning biobank data into clinical impact

On the most recent episode of The Genetics Podcast, Patrick speaks with Samuli Ripatti, director of the Institute for Molecular Medicine Finland (FIMM) and prof...
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Podcast recap: Jonathan Marchini on scaling statistical genetics from HapMap to millions of exomes

On the latest episode of The Genetics Podcast, Patrick sat down with Jonathan Marchini, Head of Statistical Genetics & Machine Learning at the Regeneron Gen...
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Podcast recap: Max Bronstein on the intersection of biotech and policy

On the latest episode of The Genetics Podcast, Patrick speaks with Max Bronstein, founder and CEO of Aviva Strategies, a health policy firm dedicated to support...
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Next-generation genetic therapies: AAV, RNA medicines, and CRISPR

Genetic medicines have been advancing at an extraordinary pace. Following the first wave of adeno-associated virus (AAV)–based gene therapies, the toolbox for g...
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Built for modern trials: How Sano supports advanced genomic therapies

The next generation of advanced therapies, particularly gene therapies, hold enormous promise for patients with rare conditions. However, their clinical develop...
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Podcast recap: Richard Wilson on the current state of gene therapy

In this week’s episode of The Genetics Podcast, we spoke with Richard Wilson, Senior Vice President and Primary Focus Lead for Genetic Regulation at Astellas, a...
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Decentralized clinical trials: At-home patient screening

Precision medicine trials increasingly depend on genetic or biomarker testing to determine eligibility. Each additional test introduces operational complexity: ...
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The N-of-1 revolution: What ASOs mean for rare disease families

In rare disease, families are often forced into roles that extend far beyond caregiving. When faced with diseases for which no treatment exists, parents have of...
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Podcast recap: Celebrating 200 episodes with Patrick Short

Last week marked a major milestone for The Genetics Podcast: our 200th episode. To celebrate, host Patrick Short revisited some of the most impactful conversati...
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Case study: Accelerating recruitment for a gene therapy trial

In recent years, the therapeutic landscape for genetic hearing loss (GHL) has advanced rapidly, with several programs reaching clinical development. However, tr...
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Podcast recap: Xin Jin on decoding brain disorders with in vivo Perturb-seq

In the last episode of the Genetics Podcast, Patrick speaks with Dr. Xin Jin, a pioneering neuroscientist at Scripps Research, about her team’s cutting-edge met...
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Clinical trial recruitment strategies for biotechs

After a decade of strong investment, the biotech sector in 2025 is navigating a markedly constrained funding environment. Early-stage companies, particularly th...
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Clinical trial budgeting: Smarter strategies for biotech

Clinical trials have always been burdensome on sponsors, but in the current biotech landscape, the pressure to execute trials promptly, efficiently, and in a co...
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Podcast recap: Benny Sorenson on patient-centered drug development for clotting disorders

In the latest episode of The Genetics Podcast, we sat down with Dr. Benny Sorenson, CEO of Hemab Therapeutics, to explore a relatively overlooked therapeutic ar...
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Top five scientific trends shaping precision medicine in 2025

The promise of precision medicine has been intensifying over the past decade. In 2025, the field appears to be at an inflection point due to a confluence of var...
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Podcast recap: Heiko Runz on population genomics and drug discovery

In the latest episode of The Genetics Podcast, we spoke with Dr. Heiko Runz, SVP of Neuroscience at insitro. With a background spanning clinical genetics, acade...
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What we learned from the FDA’s cell and gene therapy roundtable

As part of new efforts to improve FDA processes and enhance communication with stakeholders, they hosted a roundtable on cell and gene therapy on June 5th, 2025...
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Podcast recap: Molly He on multi-omics and sequencing innovation

In the latest episode of The Genetics Podcast, we spoke with Dr. Molly He, CEO and co-founder of Element Biosciences. With over a hundred patents and leadership...
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Behind the scenes at Sano: Where genomic data meets engineering

At Sano, data is at the core of everything we do. From mining our patient database to identify patients for trials to analyzing sequencing data, our engineering...
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Podcast recap: David Bumcrot on the untapped power of regulatory RNAs

In the latest episode of The Genetics Podcast, we had a captivating conversation with Dr. David Bumcrot, Chief Scientific Officer at CAMP4 Therapeutics. From he...
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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 c...
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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 ...
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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 ...
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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 valu...
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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 ...
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AI at the FDA: Impact on drug development

In May and June of 2025, the Food and Drug Administration (FDA) announced a series of developments related to the use of artificial intelligence (AI) across its...
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Personalized medicine for DMD: Genetics to treatment

Duchenne muscular dystrophy (DMD) is an inherited disorder that is characterized by progressive muscle degeneration, leading to loss of function and premature d...
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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 ...
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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-...
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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, affectin...
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Podcast recap: Frank Gleeson on targeting muscle regeneration to tackle Duchenne muscular dystrophy

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 regene...
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Q2 2025 product update: New features for seamless trial execution

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 ...
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Master protocols in precision medicine: Basket, umbrella, and platform trials explained

Precision medicine has fundamentally changed how diseases are defined and how therapies are developed — shifting eligibility from clinical phenotype to molecula...
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Podcast recap: Tim Spector on twin research, microbiome breakthroughs, and why diet is the key to personalized health

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. T...
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Biomarker testing logistics in precision trials: Introducing KitLink

Biomarker testing is foundational to precision medicine. It determines eligibility for targeted therapies and immunotherapies, informs treatment selection, and ...
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Transforming precision trials with biomarkers and stratification

As the demand for personalized therapies continues to grow, trial designs must evolve to reflect the biological variability between patients. Traditional models...
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Podcast recap: Dr. Daniel Geschwind on autism genetics

On a recent episode of The Genetics Podcast, Patrick spoke with Daniel Geschwind, Senior Associate Dean and Associate Vice Chancellor of Precision Health at UCL...
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Understanding CRISPR evolution: A decade of progress, a breakthrough, and what’s next

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 ...
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Uncovering genetic clues to long COVID: Insights from a global GWAS

Although long COVID cases have surged in recent years, the genetic factors that influence who develops the condition remain poorly understood. Traditional genom...
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Partnering to unlock the next chapter in lupus research

We’re proud to share that Sano Genetics plays a key role in the newly announced Lupus Nexus Foundational Analyses, a groundbreaking initiative led by the Lupus ...
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When systems align: Boosting site engagement through smart integration

Site disengagement can be a major barrier to successful and timely trial execution. A significant contributor to disengagement is overloading sites with redunda...
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Introducing the Sano Referral Manager: Bridging the gap from referral to randomization

Clinical trial recruitment is notoriously difficult, and nowhere is this more acute than at the referral stage. When a participant who looks potentially eligibl...
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Podcast recap: Dr. Helen O'Neill on genome editing, the CRISPR baby controversy, and transforming women’s reproductive health

In the most recent episode of the Genetics Podcast, Patrick spoke with Helen O’Neill, a molecular geneticist who is the founder of Hertility Health and an assoc...
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Supporting sites in modern trials: Tackling tech fatigue and enabling smarter decentralization

Fostering and maintaining a high standard for site engagement are key priorities for sponsors as they can enhance the likelihood of effective and timely trial e...
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Clinical trial recruitment for rare disease: AI + JIT

Clinical trial recruitment remains one of the most persistent bottlenecks in drug development. Across therapeutic areas, trials routinely face delays, cost over...
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Modernizing rare disease trials with patient-centered innovation

While there have been significant strides in the development of drugs for rare diseases over the past few decades, only 5% of rare diseases have FDA-approved tr...
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Podcast recap: Dr. Madhuri Hegde on the future of newborn screening and genomics at scale

In the most recent episode of The Genetics Podcast, Patrick speaks with Dr. Madhuri Hegde, SVP and Chief Scientific Officer at Revvity. Madhuri’s work has helpe...
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Podcast recap: Dr. Euan Ashley on ultra-rapid genome sequencing, wearables, and the science behind elite athletes

In a recent episode of The Genetics Podcast, Patrick Short sits down with Dr. Euan Ashley, Chair of Medicine at Stanford University, author of The Genome Odysse...
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Podcast recap: Kent Rogers on building a path for N-of-1 therapies and transforming rare disease treatment

In a recent episode of The Genetics Podcast, Patrick spoke with Kent Rogers, CEO of EveryONE Medicines, about the future of individualized medicines, especially...
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Building the blueprint: A strategic framework for precision medicine in autoimmune and inflammatory disease

Precision medicine has the potential to transform how we understand and treat autoimmune and inflammatory diseases, but only if we can align innovation with inf...
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Precision medicine in autoimmune & inflammatory care

The way we treat autoimmune and inflammatory diseases is undergoing a fundamental shift. These conditions are deeply heterogeneous. Patients with the same diagn...
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Podcast recap: Matt Burgess on genetic counseling, ethics in rare disease, and demystifying genetics

In the most recent episode of The Genetics Podcast, Patrick sits down with fellow podcaster and genetic counselor Matt Burgess. Matt is the host of Demystifying...
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Precision medicine advancements for autoimmune and inflammatory disease

Autoimmune diseases include more than 80 conditions defined by immune system dysfunction in which the body mistakenly attacks healthy tissue. Treating autoimmun...
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Precision medicine in autoimmune & inflammatory diseases

Despite growing momentum behind precision medicine, autoimmune and inflammatory diseases remain some of the most underserved areas in healthcare. Affecting an e...
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Podcast recap: Sal Rico on gene therapy breakthroughs, safety lessons, and what’s next for precision neurology

In a recent episode of The Genetics Podcast, Patrick spoke with Dr. Sal Rico, Chief Medical Officer at Encoded Therapeutics. Having worked across small molecule...
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Why high costs are holding back precision medicine trials

Precision medicine offers more targeted treatments, better patient outcomes, and even the potential to transform how we approach disease. But getting there isn’...
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Building better data ecosystems for rare and ultra-rare conditions

In rare and ultra-rare diseases, data availability is one of the most significant constraints in research and drug development. With thousands of rare condition...
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Beyond recruitment: Building lasting relationships with trial participants

As clinical trials become more complex and patient populations more diverse, the traditional focus on recruitment alone is no longer enough. Building long-term ...
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Podcast recap: Leeland Ekstrom on large-scale genomics, real-world data, and the future of precision medicine

In the latest episode of The Genetics Podcast, Patrick Short chatted with Leeland Ekstrom, CEO and co-founder of Nashville Biosciences (NashBio), a for-profit s...
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Podcast recap: Sharon Terry on patient-led research, biobanking, and the future of rare disease advocacy

In the latest episode of The Genetics Podcast, host Patrick Short sat down with Sharon Terry, the CEO of Genetic Alliance, to discuss her extraordinary journey ...
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Overcoming barriers to gene therapy for genetic hearing loss

Despite the huge potential of new technologies to enable precision approaches to diagnosing, managing, and treating genetic hearing loss, significant barriers a...
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Gene therapy and precision medicine for hearing loss

Hearing loss affects over 1.5 billion people worldwide, with nearly half of all cases linked to genetic factors. Despite this, diagnosis and treatment options h...
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Podcast recap: Dr. John Lepore on the expanded human proteome and the future of drug discovery

In the latest episode of The Genetics Podcast, Patrick spoke with Dr. John Lepore, physician-scientist and CEO of ProFound Therapeutics. ProFound Therapeutics, ...
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Hearing loss treatment: Gene therapy and AI advances

For decades, treatment for congenital and genetic hearing loss has largely relied on devices such as hearing aids and cochlear implants. While these technologie...
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Podcast recap: Dr. Raghib Ali on Our Future Health and large-scale genetic research

In the latest episode of The Genetics Podcast, Patrick spoke with Dr. Raghib Ali, CEO and CMO of Our Future Health (OFH), a UK initiative aimed at recruiting 5 ...
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Advancing the diagnosis of genetic hearing loss

Diagnosing genetic hearing loss hasn’t always been straightforward. With so many different causes and overlapping symptoms, traditional methods have often strug...
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Podcast recap: Anthony Bleyer and Stan Kmoch on hunting rare kidney disease mutations

In the most recent episode of The Genetics Podcast, Patrick Short sits down with Dr. Anthony Bleyer, Professor of Medicine at Wake Forest University, and Stan K...
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Podcast recap: Michelle Werner on tRNA therapeutics, basket trials, and innovation in rare disease

In a recent episode of The Genetics Podcast, Patrick sat down with Michelle Werner, CEO of Alltrna and CEO Partner at Flagship Pioneering. If you’ve been follow...
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Podcast recap: Jocelynn Pearl on systems biology, decentralized science, and innovation in cancer cell therapy

In last week’s episode of The Genetics Podcast, Patrick spoke with Jocelynn Pearl, Director of Cancer Cell Therapy at the TAM Center. With a career spanning gen...
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Building compliance into precision medicine recruitment

In precision medicine trials, recruitment and compliance are interdependent workstreams. Every step that brings a patient closer to enrollment, from digital out...
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Clinical trial recruitment: Conversion and compliance

Most clinical trials fail to recruit enough participants on time. In rare disease and genetically stratified studies, the challenge is compounded: the patient p...
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Podcast recap: Dr. Eric Adler on the future of cardiovascular medicine

In last week’s episode of The Genetics Podcast, Patrick Short spoke with Dr. Eric Adler, Professor of Medicine at UC San Diego and Chief Medical Officer at Lexe...
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Podcast recap: Dr. Versha Pleasant on addressing racial health disparities and expanding access to genetic testing

In last week’s episode of The Genetics Podcast, Patrick sat down with Dr. Versha Pleasant, Clinical Assistant Professor at the University of Michigan and Direct...
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The importance of a patient-centric approach to patient recruitment

Patient recruitment efforts are often designed with the best of intentions, incorporating processes that fulfill every regulatory requirement. However, without ...
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Key takeaways from an important conversation about IRD research

On October 18th, 2024 20/20 Onsite, Sano Genetics, and Clinical Enrollment announced the launch of a new partnership to offer a comprehensive solution for ident...
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Podcast recap: Dr. Jonathon Hill on the past, present, and future of long-read sequencing

In a recent episode of The Genetics Podcast, Patrick Short is joined by Dr. Jonathon Hill, Associate Professor at Brigham Young University and Co-Founder and VP...
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How biotechs can optimize data management and clinical trials to scale precision medicine with limited resources

Effective data management and innovative clinical trial designs are pivotal in advancing precision medicine, particularly for biotech companies operating with b...
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Podcast recap: Angela Bradshaw on Alzheimer’s research, collaboration, and community

We were excited to welcome Angela Bradshaw, Director of Research at Alzheimer Europe, to The Genetics Podcast this week. Angela shared her journey from academic...
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Scaling precision medicine in biotech: Tools & methods

Precision medicine is built on the premise that treatment and prevention strategies should account for individual variability in genes, environment, and lifesty...
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The power of data-driven insights in MASH

Metabolic-associated steatotic hepatitis (MASH) is primarily managed through lifestyle modifications, such as adopting a restricted diet high in antiinflammator...
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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...
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Podcast recap: Dr. Erik Ingelsson on RNA editing, target discovery, and the future of precision medicine

Recently on The Genetics Podcast, Patrick sat down with Dr. Erik Ingelsson, Chief Scientific Officer at Wave Life Sciences, to explore some of the most exciting...
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Precision medicine landscape for cardiometabolic conditions

Precision medicine approaches to cardiometabolic conditions are still in their infancy but stand poised to transform treatment approaches and are increasingly r...
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The state of precision medicine in cardiovascular and metabolic disease [report]

Cardiovascular disease remains a leading global health concern, claiming millions of lives each year and disproportionately affecting communities worldwide. Fac...
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Podcast recap: Genomics-driven drug discovery with Dr. Eric Green

In the latest episode of The Genetics Podcast, host and Sano Genetics CEO Patrick Short welcomes Dr. Eric Green, CEO and Founder of Trace Neuroscience. Eric has...
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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...
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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...
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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...
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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...
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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,...
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Podcast recap: Dr. Ness Bermingham on biotech innovation, CRISPR, and building successful ventures

In a recent episode of The Genetics Podcast, Patrick Short hosted Dr. Nessan Bermingham, a scientist turned biotech entrepreneur and operating partner at Khosla...
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Technologies and tools enabling precision patient finding

There are a number of innovative tools and technologies which are enabling precision patient finding. From AI to e-consent, here we explore how they are changin...
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Podcast recap: Dr. Luis Diaz on immunotherapy, liquid biopsies, and the future of cancer detection

In the latest episode of The Genetics Podcast, Patrick Short was joined by Dr. Luis Diaz, head of the Division of Solid Tumor Oncology at Memorial Sloan Ketteri...
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Podcast recap: Dr. Susan Liebman on genetic testing, advocacy, and the power of storytelling

On a recent episode of The Genetics Podcast, Patrick Short speaks with Dr. Susan Liebman, a professor emerita and researcher, about her personal and professiona...
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How precision patient finding reduces research costs

Precision patient finding has the potential to radically reduce the cost of trials and create a more centralized approach which serves to benefit a multitude of...
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Precision patient finding: the key to speed, savings, and trial success in biotech [whitepaper]

Clinical trials are essential for bringing new treatments to patients, but they’re not without challenges. Recruiting the right participants, staying on budget,...
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What is precision patient finding?

Precision patient finding is an evolving field that is growing in line with the demands of precision medicine and rare disease research. Unlike traditional pati...
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Overcoming challenges in precision neurology

Implementing precision neurology comes with its own set of complexities—from decoding the brain's intricate workings to addressing the ethical challenges of usi...
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Podcast recap: Dr. Jim Wilson on the genetics of isolated communities

In the latest episode of The Genetics Podcast, Patrick speaks with Dr. Jim Wilson, Professor of Human Genetics at the University of Edinburgh, about his work st...
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Neuron23 Unveils Groundbreaking Phase 2 Trial in Early Parkinson’s Disease

Sano is proud to support Neuron23 in the NEULARK Phase 2 clinical trial, a groundbreaking study exploring NEU-411, a brain-penetrant LRRK2 inhibitor with the po...
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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...
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What’s driving progress in precision neurology?

Precision medicine is transforming neurology by enabling more accurate diagnoses and tailored treatments. Here, we explore key components driving this progress,...
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Podcast recap: Eric Fauman on leveraging genetic data for drug discovery at Pfizer

In a recent episode of The Genetics Podcast, host Patrick Short sits down with Eric Fauman, Executive Director and Head of Computational Biology in Pfizer's Int...
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How precision medicine is transforming neurological health care

Millions of people worldwide are affected by neurological disorders like ALS, Alzheimer’s, and Parkinson’s. These conditions bring unique challenges for patient...
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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...
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Lupus Research Alliance Launches the DREAM Knowledge-Exchange Platform to Accelerate Precision Medicine in Lupus

Today, the Lupus Research Alliance (LRA) launched the Data Repository, Exchange, and Analytics Platform (DREAM)—a pioneering knowledge-sharing platform dedicate...
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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...
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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...
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Podcast recap: Q3 insights with Dr. Veera Rajagopal

In the latest episode of The Genetics Podcast, Patrick welcomes back Dr. Veera Rajagopal, a drug discovery scientist at Regeneron, for his quarterly discussion ...
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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...
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Sano Genetics named in Bloomberg UK’s Startups to Watch 2024

LONDON – 22 October 2024: Sano Genetics, the UK-based precision medicine company accelerating precision medicine clinical trials, has been named as one of Bloom...
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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 ...
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Podcast recap: Bridging genomics, business, and equity in healthcare access with Mark Taylor

In the latest episode of The Genetics Podcast, host Patrick Short interviews Mark Taylor, Head of Strategic Partnerships at the National Institute for Health an...
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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...
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Inherited retinal disease: Mobile eye care partnership

A new partnership will bring together specialist eye care, genetic testing, and patient recruitment to advance both clinical research and care for people affect...
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Genetic risk factors and precision medicine in chronic kidney disease

Chronic Kidney Disease (CKD) is a genetically heterogeneous condition, with over 60 genetic diseases known or suspected to affect the kidneys across both monoge...
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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...
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Webinar recap: Precision medicine ethics: Navigating genetic screening with care

In a recent webinar hosted by Lindsey Wahlstrom-Edwards, Partnerships Lead at Sano Genetics, expert speakers explored the complexities of genetic testing and it...
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Podcast recap: Exploring the future of health monitoring with Dr. Mike Snyder

On last week's episode of The Genetics Podcast, host Patrick Short was joined by Dr. Mike Snyder, Director of the Center for Genomics and Personalized Medicine ...
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Precision medicine worldwide: Market & regional report

Precision medicine replaces the one-size-fits-all model of treatment and prevention with an approach that accounts for individual differences in genes, environm...
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Why does precision medicine matter?

Precision medicine is reshaping healthcare by moving away from a one-size-fits-all approach to more personalized care. By understanding each patient's genetic m...
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Podcast recap: How genomics is rewriting the taxonomy of disease with Dr. Lon Cardon

In the latest episode of The Genetics Podcast, host Patrick Short welcomes Dr. Lon Cardon, CEO of the Jackson Laboratory, to discuss his career journey, the ris...
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The internationalization of precision medicine: Challenges of implementation

While precision medicine holds great promise for both patients and the growth of global markets, there are significant obstacles to be overcome to enable its wi...
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Longevity research trends: The epigenetic clock & microbiome

As the global population ages, understanding the genetic, biological, social, and environmental factors influencing longevity has become a central area of resea...
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Podcast recap: Uncovering the secrets of gene regulation with Nadav Ahituv

In the latest episode of The Genetics Podcast, host Patrick Short welcomes Nadav Ahituv, Director of the Institute for Human Genetics at the University of Calif...
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Global market expansion and the future of precision medicine: The case for internationalization [whitepaper]

Breakthrough technologies and data innovation are driving cost reductions and increasing return on investment in precision medicine, fueling accelerated interna...
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Podcast recap: Understanding cell aging and its role in disease with Marco Quarta

In this episode of The Genetics Podcast, host Patrick Short sits down with Marco Quarta, Co-founder and CEO of Rubedo Life Sciences, to discuss the biology of a...
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