Exosomes: Unveiling Their Potential in Cell and Gene Therapy

By Marites T. Woon (Associate Director, Nonclinical), Mialy Ramaroson (Director, CMC), Jessica Lee (Principal, Global Head Cell & Gene Therapy), William Lee (Vice President, Regulatory Affairs), and Richard Stewart (Vice President, Nonclinical)   Welcome to our three-part blog series on exosomes, the fascinating messengers of the cellular world. In this captivating exploration, we delve into the […]

Nonhuman Primates (NHPs): FDA Issues Guidance for Use in Nonclinical Safety Studies

By Alison Wakeford, PhD, Scientist, Allucent Richard Stewart, PhD, VP Nonclinical, Allucent Marcus S. Delatte, PhD, VP Regulatory Strategy, Regulatory and Drug Development Consulting, Allucent The Food and Drug Administration (FDA) issued a final guidance on 4 February 2022 to address the constrained supply of nonhuman primates (NHPs) for nonclinical safety assessments. As part one […]

Risk Assessment: Traditional and Novel Approaches to Assess and Manage Risks

Risk assessment is a complex process intended to protect human health by identifying drug-related hazards, determining dose-response relations for a product within relevant biological systems, and assessing exposure levels to characterize potential risks. These assessments may be conducted via traditional frameworks using data from in vitro and in vivo studies, as well as novel frameworks using pharmacokinetic and pharmacodynamic model-based […]

A Snapshot of Gap Analysis

When building a successful regulatory strategy, it is critical to keep the end objectives in mind and design a process that is tailored to your specific product. Planning for regulatory requirements can help you avoid non-compliant practices from the very beginning, but how do you make sure your strategy is comprehensive? The answer may be […]

Inspection Readiness: What is it, why it matters, and what you need to know to be prepared

Organizations must have inspection readiness since inspections can be unannounced or come at any time. Good Clinical Practice (GCP) inspectors use different approaches depending on the regulatory authorities for which they represent; however, all of them will verify the compliance status on GCP and other applicable regulations during the inspection. Planning ahead to anticipate all processes are compliant means an organization […]

The Multifaceted Role of the Nonclinical Expert at the Center of Drug Development Teams

Product development teams getting started on preclinical exploration of a potential treatment need a certain set of specialists on board. Key roles include scientists, clinical strategist, project manager, regulatory affairs director, and a nonclinical expert, a primary authority on product safety. In fact, inclusion of a nonclinical expert is mandated by regulatory authorities to ensure […]

What are Good Laboratory Practices (GLP) for Nonclinical Studies?

Good Laboratory Practices (GLP) provide regulations and the standard by which drug safety studies are conducted in nonclinical animal studies. In addition to ensuring the ethical treatment and welfare of animals, adhering to GLP regulations gives sound evidence of the validity, integrity, and reliability of nonclinical safety data. This nonclinical safety data will ultimately be […]

Understanding CDISC SEND Data and How to Be Compliant

Nonclinical study data submitted to the FDA must align with the Clinical Data Interchange Standards Consortium (CDISC) Study Data Tabulation Model (SDTM). The version of data standards for nonclinical studies is known as the Standard for Exchange of Nonclinical Data (SEND). SEND is chartered by CDISC, a globally recognized, not-for-profit organization that actively develops data […]

Pre-IND Meeting Guide: Benefits & How to Prepare

Regulatory guidelines allow for formal meetings between the FDA and sponsors or applicants to discuss the development and review of an investigational new drug (IND) application for drugs or biologic products. This meeting, known as a pre-IND meeting, is intended to address specific questions related to the initial US clinical study (also known as a first-in-human […]

Mind the Gap: The Importance of Gap Analysis in Securing Regulatory Approval

Significant pressure to achieve regulatory approval to start generating return on investment can affect any sponsor, but it is of the greatest importance to small biopharma companies without existing marketed products. Biopharma companies may have conducted successful research and put in the work to warrant approval and market access, but to an uninitiated sponsor bridging […]