Development of cell and gene therapies is growing rapidly, given the major advances in genomic technologies and increasing scientific understanding of genetic regulation and immunology. The Cell and Gene Therapy (CGT) field has unprecedented potential to effectively treat and/or cure genetic-based rare and orphan diseases and expand the reach of personalized medicine. This blog will...
Designing an adequate nonclinical program to support the safety of cell or gene therapy products is not always straightforward. In this blog, we will address common questions we receive from our clients and aspects to consider when designing those nonclinical studies. In which species should the toxicity study be conducted? Does it have to be...
For cell therapies, the roadmap to first-in-human studies is marked by critical scientific and regulatory milestones that require careful planning, expertise, and collaboration from various stakeholders. From rigorous nonclinical studies, to cell manufacturing processes, to overcoming regulatory complexities, each milestone represents a significant step forward in bringing these transformative treatments to patients in need. In...
Developing a novel gene therapy requires a level of comfort with change and unpredictability. The path to regulatory approval is rarely straightforward, and roadblocks can enter at any turn. Premier Research was approached by a sponsor of an adeno-associated virus (AAV)-based gene silencing technology to conduct a natural history study, with a goal to identify...
March 7th, 202411 a.m. EST For cell therapies, the roadmap to first-in-human studies is marked by critical scientific and regulatory milestones that require careful planning, expertise, and collaboration from various stakeholders. From rigorous nonclinical studies, to cell manufacturing processes, to overcoming regulatory complexities, each milestone represents a significant step forward in bringing these transformative treatments...
May 7 – 11, 2024 Booth #936 Baltimore, Maryland Dive into cutting-edge gene therapy, network with leaders, explore collaborations, and discover innovations. Don’t miss the chance to visit our exhibit – join us for a transformative experience at the forefront of gene and cell therapy!
March 14, 2024 Exclusive Opportunity: Cell & Gene Therapy Consulting Session Discover a unique opportunity to participate in one-on-one discussions with Premier Research experts. Our specialists in cell and gene therapy will be accessible on specific dates for personalized consultations. Seize this opportunity to acquire valuable insights. Secure your spot by signing up now. Leading...
Gene therapies are complex, and so are the clinical trials necessary for establishing their safety and efficacy. Given their complexity and the potential need for long-term follow up, gene therapy studies require highly qualified sites and highly engaged patients. In fact, the ability to select the right sites and patients can be the difference between...
Cell and gene therapies offer great promise for rare diseases, where patients—many of them children—may be severely ill or debilitated and treatment options are often limited. Development of these advanced therapeutics is complex due to both disease-specific and treatment-related factors and executing clinical trials that meet the expectations of regulators requires careful attention to study...