Clinical research blog

Gene and cell therapies are providing new treatment for those with rare diseases. These therapies offer hope for long-term solutions—and even potential cures—for millions of people worldwide. However, for these treatments to truly make a difference, they must align with patient values, perspectives, and access needs.

In one of our recent webinars, Charlotte Guzzo, COO of Sano Genetics, led a discussion on the challenges and opportunities in rare disease research and drug development. The conversation featured Harriet Holme, chair and founder of PCD Research, whose professional and personal experiences shed light on the current difficulties of rare disease treatments and potential solutions. Read on for a quick recap of the webinar:

In a recent episode of The Genetics Podcast, Patrick Short hosted Dr. Nessan Bermingham, a scientist turned biotech entrepreneur and operating partner at Khosla Ventures. Dr. Bermingham has co-founded over six biotech companies, including Intellia Therapeutics and Korro Bio, and played an important role in advancing CRISPR-based genome editing and RNA editing technologies. Their conversation dove into the challenges and opportunities in biotech, from founding companies to navigating regulatory hurdles and leveraging cutting-edge science.

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 changing the face of the clinical trial landscape.

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 Kettering Cancer Center. Known for his contributions to precision oncology, Dr. Diaz has played an important role in the development of targeted therapies and immunotherapies that are reshaping cancer care. The discussion spans his landmark studies, the evolution of liquid biopsies, and the potential for precision medicine to expand into other fields. Here's a quick recap of the conversation:

On a recent episode of The Genetics Podcast, Patrick Short speaks with Dr. Susan Liebman, a professor emerita and researcher, about her personal and professional journey in genetics. Dr. Liebman shares her deeply moving story of uncovering the genetic underpinnings of her family’s health tragedy—a sudden, fatal cardiac event that claimed the life of her niece—and how it inspired her to write her memoir, The Dressmaker’s Mirror. The conversation goes into detail on the role of genetics in health, the importance of early screening, and the human side of science.

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 stakeholders, from biotechs to HCPs and patients.

Clinical trials are essential for bringing new treatments to patients, but they’re not without challenges. Recruiting the right participants, staying on budget, and avoiding delays are some of the biggest hurdles. Unfortunately, traditional methods of finding and enrolling patients often fall short—costing time, money, and sometimes even the success of the trial.

That’s where precision patient finding comes in. By using tools like genomic profiling and AI, this approach is making trials faster, more efficient, and ultimately more patient-friendly.

Precision patient finding is an evolving field that is growing in line with the demands of precision medicine and rare disease research. Unlike traditional patient recruitment methods, it harnesses technologies such as data analysis and advanced analytics, and utilizes comprehensive patient data (including genomic profiles) to rapidly identify groups of patients who have an increased likelihood of study eligibility and enrollment. Here we’ll explore some of the key elements of precision patient finding and how they interlink.