Sifting Through Rarity: Advanced Patient Screening for Rare Disease Trials

By Maria-Cruz Morillo (Global Therapeutic Operations Lead, Rare Disease) Because rare diseases affect a small population of people who are dispersed across the world, it makes country and site selection, patient identification, clinical trial recruitment and long-term retention particularly complex. Furthermore, many rare diseases are difficult to diagnose — in some cases they have been […]

Mastering Clinical Trial Design: Will FDA’s Latest Guidance on Master Protocols Encourage Increased Adoption?

By Sugato De (VP, Regulatory Strategy, Head of Medtech) and Michelle Hoffner O’Connor (Clinical Strategy Scientist II)  On December 21, 2023, the Food and Drug Administration (FDA) issued a draft guidance entitled “Master Protocols for Drug and Biological Product Development.”  Master protocols originated as an efficient mechanism to determine which therapies may drive the most […]

Fast-Tracking Drug Development: Role of Mechanistic PK/PD Modeling

By Neha Bhise (Associate Director, Clinical Pharmacology), Soha Freidy (Pharmacometrician I), and Jessica Roberts (Director, Pharmacometrics) Mechanistic pharmacokinetic (PK)/pharmacodynamic (PD) modeling is a quantitative approach that has been successfully used to identify potential for success across various stages of drug discovery and development. These models are useful to distinguish characteristics of promising compounds from those […]

Balancing Act in Preclinical Development: Strategies for Assessing and Managing Neurotoxic Risks in CNS Therapeutics

By Marcus Delatte (VP, Regulatory Strategy), Alison Wakeford (Scientist), Devin Welty (VP, Clinical Pharmacology), and Rachel Rozakis (Sr. Clinical Pharmacologist) Background Central Nervous System (CNS) disorders are typically severe and may produce various behavioral, anatomical, and physiological changes in millions of patients, which complicates developing effective therapeutics and managing product-related neurotoxicity.  Therefore, careful attention should […]

Cell and Gene Therapy Advancements: Early-Stage CMC Strategies for Exosome Innovation

By Marites T. Woon (Associate Director, Nonclinical), Mialy Ramaroson (Director, CMC), Jessica Lee (Principal, Global Head Cell & Gene Therapy) Welcome back to our exploration of the transformative world of exosomes in the second installment of our blog series. In our initial dive, we uncovered the intriguing potential of exosomes as dynamic messengers in intercellular […]

Anti-Depression Medication: Current Landscape and Product Development Trends

Alison Wakeford, PhD, Scientist, Allucent Marcus S. Delatte, PhD, VP Regulatory Strategy, Allucent Background According to the World Health Organization, depression is a common mood disorder, affecting approximately 280 million people globally.1 Depression is characterized by persistent feelings of sadness and loss of interest, occurs across almost all age groups, and manifests in various forms […]

Advancing Oncology Drug Development: The Importance of Single-Arm Trials

By Marites T. Woon, PhD (Associate Director, Nonclinical) and Jessica Lee, MD, PhD (Principal, Global Head Cell & Gene Therapy) According to a report by the American Society of Gene and Cell Therapy, oncology remains one of the top areas of focus for investigational cell and gene therapy (CGT) products in Q1 of 20231, underscoring […]

Inroads To Approval: Navigating Formal FDA Meetings to Optimize Your Drug Development Program

Matthew Hight, PhD, RAC, Associate Director, Regulatory Affairs, AllucentJoshua Taylor, PhD, RAC, Senior Director, Regulatory Affairs and Head of Clinical Strategy, Allucent Marcus Delatte, PhD, Vice President, Regulatory and Drug Development Consulting, Allucent Welcome to our comprehensive three-part blog series where we will seek to guide you through the intricate world of formal meetings between the […]

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 […]

Cannabis and Cannabis-Derived Products for Therapeutic Development

Part One: Sources of Cannabis and Cannabis-Derived Compounds Alison Wakeford, PhD (Nonclinical Scientist I), Laura L. Erwin, PhD, and Marcus S. Delatte, PhD (VP, Regulatory Strategy)    FDA Issues Final Clinical Research Guidance on Cannabis   The Food and Drug Administration (FDA) issued a final guidance on 24 January 2023 that provides the agency’s current thinking on topics […]