FDA Webinar: Safety Assessment of Genome Editing in Gene Therapy
The FDA released a draft guidance on safety assessment of genome editing in human gene‑therapy products using next‑generation sequencing (NGS). Building on the January 2024 guidance, it focuses on non‑clinical study design, data quality, and submission requirements for DNA, epigenetic, and RNA editors. The guidance stresses quantitative NGS evaluation of off‑target editing, unintended on‑target outcomes, and chromosomal rearrangements. It recommends multiple nomination approaches—biochemical, cell‑based, and in‑silico—and requires consideration of human genetic variation that could affect guide‑RNA binding and editing risk. Sponsors must report detailed information for each nominated or confirmed site, including genomic coordinates, read counts, editing frequencies, alignment mismatches, and functional annotation. Documentation of assay modifications, quality‑control metrics, raw sequencing data, and analysis scripts is mandatory for IND or BLA submissions. By standardizing NGS‑based safety assessments, the guidance aims to reduce regulatory uncertainty and accelerate development of genome‑editing therapeutics. Early engagement with the FDA through INTERACT or pre‑IND meetings is encouraged to align study plans with agency expectations, potentially shortening time to market.
Financial Transparency & Efficiency of the Prescription Drug, Biosimilar, & Generic Drug User Fees
The FDA held its annual public meeting to detail financial transparency and efficiency of the Prescription Drug User Fee Act (PDUFA 7), Biosimilar User Fee Act (BaSUFA 3), and Generic Drug User Fee Amendments (GDUFA 3). CFO Benjamin Mons opened...
Expanded Access to Investigational Drugs for Treatment Use - Questions and Answers
The FDA released a 2025 guidance document that revises the expanded‑access program for investigational drugs, outlining how patients with serious or immediately life‑threatening conditions can obtain treatment when no approved therapy exists. The guidance defines three regulatory categories—individual patient, intermediate‑size patient...
Understanding Endpoints in Rare Disease Drug Development
The video explains why selecting appropriate endpoints is a linchpin in rare‑disease drug development, where traditional measures often lack precedent. It stresses that an endpoint must be a precisely defined variable that can be statistically analyzed to answer a specific...
AGDD 2025 | D2S04 - Nitrosamines: Known Issues and Practical Advice
The final session of the Advancing Generic Drug Development Workshop tackled nitrosamine impurities in immediate‑release oral generics, focusing on the unique challenges posed by BCS 4 drug substances. Presenters highlighted that out of roughly 160 nitrosamine‑impacted products, a substantial share falls...
ClinicalTrials.gov: Essentials for Academic Medical Centers – Pre-Recorded Training Video
The video is a pre‑recorded training for academic medical centers on ClinicalTrials.gov registration and results reporting. FDA and NIH experts outline legal obligations, the historical evolution of the registry, and the roles of the National Library of Medicine, FDA, and...
CDRH Patient Education Program: Patients as Advisors in Medical Device Clinical Studies
The FDA’s CDRH Patient Education Program introduces patients as advisors in medical‑device clinical studies, explaining how lived‑experience contributors can influence the development lifecycle from discovery through post‑market. The course defines a patient advisor as a non‑clinical participant who shares personal disease...
Oncology Nurses - The Frontliners in Oncology
The FDA Oncology Center of Excellence hosted a panel for National Nurses Week, highlighting oncology nurses as the front‑line drivers of cancer drug development. Senior advisors and seasoned nurses shared personal stories, underscoring the profession’s high public trust and its...
CDER - REdI Annual Conference 2026: Innovative Regulatory Strategies to Advance Medical Products
The FDA’s REdI Annual Conference 2026 gathered over 8,300 registrants from 106 countries to showcase innovative regulatory strategies that will shape the future of medical product oversight. The event’s theme highlighted the agency’s shift from traditional paradigms toward data‑rich,...
FDA Direct: The Power of Real-Time Clinical Trials
The FDA unveiled its inaugural real‑time clinical trial system, announced by chief AI officer Jeremy Walsh alongside Dr. Emma Meagher of the University of Pennsylvania and Dr. Jennifer Litton of MD Anderson. The initiative moves beyond theoretical panels, delivering an...
FDA Direct - Special Edition of FDA Direct
In a special edition of FDA Direct, the agency’s leadership reviewed a year of restructuring, record‑setting approvals, and a forward‑looking agenda focused on speed, safety and affordability. The discussion highlighted a near‑record 67 new drug approvals, an average review time...
Public Meeting Exploring the Scope of Dietary Supplement Ingredients
The FDA’s Office of Dietary Supplement Programs (ODSP) is convening a public meeting to examine how scientific and technological advances are reshaping dietary supplement ingredients. The agenda will cover the legal scope of the phrase “dietary substance for use by...
Vaccines and Related Biological Products Advisory Committee Meeting
The FDA's Vaccines and Related Biological Products Advisory Committee convened in open session to discuss and recommend strain composition for the 2026–2027 U.S. influenza vaccines. The meeting opened with roll call introductions from committee members and guest speakers—experts from academia,...
Adeno-Associated Virus-Mediated Gene Therapy - Advances, Immune Challenges, and Research Innovations
At FDA Grand Rounds, Dr. Ronit Mazur of CBER reviewed advances and persistent immunological challenges in adeno-associated virus (AAV)–mediated gene therapy, outlining how AAV’s favorable safety and durability have driven a surge in FDA approvals since 2017. She summarized AAV...
FDA Direct: Combating Rare Diseases at the FDA
The FDA Direct town hall marked Rare Disease Day with a candid conversation between FDA leaders Jim and Elizabeth, who both have personal ties to rare‑disease advocacy. Their discussion highlighted the agency’s growing focus on rare‑disease patients, the establishment...