Advances in sequencing, the emergence of antisense oligonucleotide therapies, and the advent of induced pluripotent stem cell technology are among the combination of factors that have made bespoke medicines for individual patients scientifically feasible. In the case of ultra-rare, genetic diseases, a child may face a progressive, life-threatening condition with no available treatments. As we continue our series on discovery with Charles River Laboratories, we take a look at how the organization partnered with global collaborators and a patient’s family to design, screen, and advance a highly selective antisense oligonucleotide therapy targeting a pathogenic allele underlying a child’s ultra-rare epileptic encephalopathy. We spoke to Charles River Lab’s Associate Research Leader Blanca Torroba and Group Leader of Neuropharmacology Johanna Uhari Väänänen about how they moved from concept to a candidate for a custom ASO therapy, steps they took to accelerate the process, and the challenges of advancing N of 1 medicines to an IND.nbRb

Immunogenicity testing helps establish the safety of experimental therapies by ensuring drugs don’t trigger a potentially dangerous immune response. While regulators had long relied on a fixed, three-tier immunogenicity testing model, the industry is now shifting to a flexible, risk-based approach that continues to meet regulatory requirements and ensure patient safety. We spoke to Katie Sime, principal research scientist at Charles River Laboratories, about when reduced-tier approaches make sense, what regulators want to see; and why adaptive strategies can particularly benefit resource-constrained biotechs by balancing scientific rigor, patient safety, and budget constraints.

At Charles River Laboratories, scientific advisors and study directors have complementary roles in nonclinical drug development. Scientific advisors bring years of scientific and regulatory experience to program-level strategy, while study directors act as operational captains who execute individual studies under good laboratory practice. Close collaboration between the two plays a critical role in helping clients build coherent, IND-enabling safety packages. We spoke to Pramila Singh, senior principal scientific advisor at Charles River Laboratories, about how scientific advisor–study director collaborations are crucial for creating robust, regulatory-ready study designs, how they help prevent costly errors, and how they can accelerate development timelines.

Biologic drug development increasingly relies on sensitive, human relevant platforms capable of detecting both intended and unintended target engagement well before first in human studies. Charles River Laboratories is leveraging its Retrogenix cell microarray platform alongside traditional tissue cross-reactivity studies to gain a more comprehensive view of target and off-target biology. We spoke with Charles River Labs Associate Director of Advanced Modalities Mark Aspinall O’Dea and Charles River Labs Senior Scientific Director James Raymond, about how combining these two approaches helps sponsors select better leads, design safer studies, and meet regulatory expectations for complex modalities.

The journey from discovery to IND is rarely linear. It demands rigorous target validation, continuous regulatory engagement, and the use of translationally relevant models to effectively de risk programs before they advance. In this installment of Big4Bio’s ongoing series with Charles River Laboratories, we look at how the company’s One Step approach guides sponsors from discovery concept to IND-ready clinical candidate. We spoke to Manu Kohli, principal scientific advisor for Charles River Labs, about how the company’s integrated development model works, the benefits of having an end-to-end drug development solution under one roof, and how this approach helps deliver submission-ready assets on time and on budget.

Drug metabolism and pharmacokinetics, often referred to as DMPK, is not just a box to check on the way to advancing an experimental therapy into the clinic. In reality, it is an essential part of determining whether a program can result in a safe, effective, and commercially viable medicine. We spoke to Michael Templin, director of scientific advisory services at Charles River Laboratories, about how a thoughtful DMPK strategy can identify liabilities early, how emerging tools like AI-driven prediction and organoid systems are reshaping the DMPK toolkit, and its role in influencing business decisions such as program prioritization and competitive differentiation.

As the biotech industry shows renewed strength with rising stock prices and growing deal values, thousands of biotech industry leaders, investors, and innovators will gather in San Francisco in January for the annual Biotech Showcase. The event, which runs concurrently with the J.P. Morgan Healthcare Conference, has become an essential gathering place for emerging companies seeking visibility, funding, and deals. In addition to a packed agenda over three days, the conference will host 7,000 meetings between biotech companies and potential investors and partners. We spoke to Sara Demy, founder and CEO of Demy-Colton — which co-produces the Biotech Showcase with the EBD Group — about what’s in store at this year’s event, the large number of partnering meetings that are scheduled, and what participants can do to get the most out of the conference.

Charles River Laboratories is leveraging its deep discovery sciences expertise with advanced computational tools to accelerate drug development, improve safety, and reduce animal use. In this episode of our continuing series on discovery sciences with Charles River Labs, we explore the company’s use of the Logica AI platform and virtual control groups. We spoke to Charles River Labs Senior Science Director Alison Jones and Director of Digital Product Management, Discovery, and Safety Assessment Laura Lotfi, about how AI is accelerating the discovery process, how data is a differentiator, and the enduring role of human scientific judgment in therapeutic innovation.

Organoids¬—miniaturized, three-dimensional, lab-grown, tissue cultures that mimic organs¬—can be used to improve drug discovery and safety testing by providing more predictive human biology models than traditional 2D cultures or animal testing. In this episode in our continuing series on discovery sciences with Charles River Laboratories, we explore the use organoids as powerful tools within the arsenal of New Approach Methodologies. We spoke to Charles River Labs Senior Research Leader Ludovico Buti, and Charles River Labs Chief Technology Officer for Discovery David Fischer, about the use of organoids, how Charles River is integrating these powerful tools into its work, and their potential to transform early discovery.

As we continue our Big4Bio occasional series on discovery and safety assessment with Charles River Labs, we explore new approach methodologies, or NAMs, and their integration with antibody discovery and development. We spoke to Charles River Laboratories’ Senior Principal Scientific Advisor Sarah Gould and Charles River Laboratories’ Scientific Business Director Omar Aziz about the evolving landscape of predictive toxicology, regulatory efforts to advance alternatives to animal testing, and the role of collaboration and strategy in accelerating therapeutic programs from concept to the clinic.