In clinical research and development, comparison to a control is highly recommended by regulatory agencies for the purpose of demonstrating evidence of clinical efficacy of a drug or device. The control could be a placebo, a known standard-of-care or an available market-approved drug or device. With some diseases or certain classes of devices, a viable...
Today there are over 10,000 rare diseases affecting more than 30 million people in the U.S.1 Many of these disorders are life-threatening, and more than 90% do not have a U.S. FDA-approved treatment2. Novel gene therapies offer hope for improving the health of patients afflicted with these conditions. Securing clearance for these treatments is...
We’re currently experiencing breakthrough times for clinical research in gene therapies. These complex treatments offer much-needed hope to more than 30 million U.S. patients affected by over 10,000 rare diseases1, many of which are life-threatening and the vast majority of which have no available treatment. Gene therapy products must meet the same key regulatory...
Data-driven solutions for your most difficult CGT hurdles Cell and gene therapy research is often a high-stakes undertaking, involving incredibly complex science and constantly shifting regulatory terrain. We work diligently to minimize your development risks through our insightful regulatory support, expert-led chemistry, manufacturing and controls (CMC) strategies, and data-driven clinical execution. To read more, download...
As liquid biopsies become more commonplace, gaining wider FDA acceptance and even becoming standard of care in some instances, the technology is giving rise to important new questions. In this webinar, we will explore the technique’s growing importance in early cancer detection and address common questions about its use. Topics will include: Speakers: Charlie Chrisawn,...
Speakers: Charlie Chrisawn, Executive Director, Program Strategy, In Vitro Diagnostics Michael Edwards, Senior Director, Regulatory Affairs, MedTech
There is increasing focus on the development of new cell and gene therapies (CGT) in rare disease. The majority of CGT approvals have been based on small, open-label, non-randomized, single-arm studies which either evaluate treatment effectiveness without a comparator or utilize a historical control comparator to evaluate treatment efficacy. Among the several unique study design...
Speakers: Abie Ekangaki, Ph.D.Vice President, Statistical Consulting Kenneth Ndugga-Kabuye, MD, FACMGVice President, Cell & Gene Therapy
Cell and Gene Therapy Expert Dr. Kenneth Ndugga-Kabuye to Lead Clinical Strategy With Collaboration From Operations, CMC and Regulatory Experts MORRISVILLE, N.C., April 17, 2023– Premier Research announced today the addition of Kenneth Ndugga-Kabuye, M.D., FACMG, as VP, Cell & Gene Therapy to the strategic Cell & Gene Therapy (CGT) Leadership Team. Ndugga-Kabuye joins experts...
April 20, 202311:00 a.m. EDT There is increasing focus on the development of new cell and gene therapies (CGT) in rare disease. The majority of CGT approvals have been based on small, open-label, non-randomized, single-arm studies which either evaluate treatment effectiveness without a comparator or utilize a historical control comparator to evaluate treatment efficacy. Among...