Gene Therapy products continue to advance as unique therapeutic tools, offering potential cures to several genetic diseases for which there are no or limited treatment options.
Regulatory guidance on gene therapy products evolves at the same time, supporting the developers in their journey from early research phase to pivotal clinical studies, and eventually marketing authorization.
An increase in regulatory guidance has been seen in the last two years, with both FDA and EMA releasing final versions of their recommendations on quality, non-clinical and clinical requirements for Gene Therapy products.
The novelty of Gene Therapy products means that process and product understanding are still evolving in the field, assisted by advances in both manufacturing and analytical technologies.
Acknowledging the challenges associated with Gene Therapy development, regulatory bodies allow for a degree of flexibility in their expectations, while ensuring patient safety is guaranteed at all stages of development.
Accelerated approval pathways are also made available to applicants guarantee timely access of Gene Therapies to patients. While beneficial from a patient access perspective, these programs impose additional challenges to the Chemistry, Manufacturing and Control (CMC) development, due to the compressed timelines.
In this webinar, we will explore the regulatory guidance available from FDA and EMA, with a focus on the CMC aspects.
Phase appropriate CMC approaches to meeting regulatory expectations will be discussed, showing how a core of quality control can be built at initial phases to ensure product safety, and then gradually evolve to meet the increasingly rigid regulatory framework.
The challenges associated with the evolving CMC development will be discussed, and suggestions will be provided to anticipate and mitigate them, both from a process development and a product quality control point of view.
We will then focus on the execution of comparability studies to support process changes, and how current regulatory guidance can be used to define suitable protocols.
Francesco Lanucara joined PharmaLex in June 2021 as a Senior Manager, Regulatory Affairs CMC, where he provides expert leadership and support for CMC project management, gap analysis, CMC strategies, authoring and reviewing of regulatory submissions of biological medicinal products.
Before joining PharmaLex, Francesco worked as Senior Technical Specialist in Allergan biologics, where he worked on CMC analytical development and regulatory strategy for a number of biological molecules, with a focus on gene therapy products.
Francesco earned his PhD in Pharmaceutical Sciences in 2008 from the University of Rome and has since been working on analytical method development, CMC and regulatory strategy of biotechnological products.