The promise of a host of new therapeutic modalities is good news for patients, however these novel platforms require unique discovery and development strategies that go beyond traditional approaches – many times into unchartered territory. Advanced medicines, including cell and gene therapy, gene editing, CAR Ts, and second generation oligonucleotides, require scientists to rethink previous drug development models and consider a path from target validation to the clinic that is unique to each program.
To increase the chance of success, development teams must identify and recognize challenges ranging from a complex manufacturing processes, drug candidate-specific safety risks, the potential for immunogenicity, to a development plan focused on small or disease-centric patient populations. It is extremely important to tap into experts with not only scientific knowledge but experience in outlying dynamic programs, and who continue to follow and understand a fast paced and rapidly evolving regulatory landscape.
Over the past few years, Charles River has been a partner in the nonclinical development of hundreds of advanced medicine programs with many designed in association with our Scientific Advisory Services (SAS) group. They are comprised of scientists with diverse backgrounds in nonclinical development. Several members of the team have direct experience gained from working in small biotech, large pharma, or regulatory agencies. Each member of the SAS team brings a unique perspective to the development process; the experience of one or more individuals can be leveraged to assist with program design, study conduct and data interpretation, and regulatory filing.
While a nonclinical plan is the desired outcome, collaboration starts with a conversation on the underlying scientific principles and goals for clinical development. When key attributes of a drug candidate and patient populations for therapeutic intervention are discussed, nonclinical plans can be built. They address which species to use, the possibility of using disease models, and uncommon routes of administration. A collaborative approach and deep understanding of each modality helps advance novel therapies into and through clinical trials, and design custom studies and programs that reduce risk and accelerate timelines.