Participation in clinical trials can be burdensome and disruptive. In addition to practical inconveniences and discomfort, participants may have to take time off work, arrange for childcare, and travel long distances to sites. Nevertheless, patient populations can be highly engaged and motivated to participate in clinical research and impact the drug development process.
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 adeno-associated virus (AAV), to explore recent breakthroughs and setbacks. With several major biopharma exits, ongoing safety debates, and a wave of next-generation delivery platforms in development, this episode explored where AAV stands today and what needs to change for it to scale.
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 activities include steps such as site selection and initiation, regulatory and ethics submissions, and patient recruitment planning. Several factors can impact the speed of study start-up, such as the complexity of protocols and budget negotiations with sites.
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 ten-year effort to map Parkinson’s genetics at global scale and make the data broadly available, so researchers can move faster on mechanisms, biomarkers, and therapies.
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 global site landscape is shifting and what this means for trial sponsors. Joan A. Chambers, Senior Consultant at Tufts CSDD, led a session that provided an insightful look at what sponsors need to know now.
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 engagement with patients and participants. This may include providing education, genetic testing, or support to embark on the clinical trial process.
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 burdens make participation difficult. Overcoming these challenges requires a combination of thoughtful trial design and compassion for patients and families who agree to participate in trials.
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 progranulin-related frontotemporal dementia (FTD). Wanda has spent decades helping brain and plasma banks, connecting with scientists, and helping families. In this blog, we cover how Wanda became involved with FTD and the impact of her work.
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 Novartis. Panelists included representatives from sponsors like BMS and Merck, vendors like Tempus, and research sites too – all sharing perspectives on one of the industry’s most pressing challenges: how to effectively match patients to trials while building trust across the ecosystem. In this blog, we highlight some key takeaways from the discussion.
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 son Michael was diagnosed with SPG50, an ultra-rare disease with no available treatments. Faced with a devastating prognosis, Terry raised millions, learned about science and biotech, and built a team to create a gene therapy – all in under 3 years.