Center of Excellence – Cell and Gene Therapy
Cell and Gene Therapy (CGT) (also known as Advanced Therapy Medicinal Products or ATMPs) holds the promise to deliver transformative outcomes in a wide array of hard-to-treat diseases. Unfortunately, so far, most have failed in development, and few have been successful in the marketplace.
Our Center of Excellence – Cell and Gene Therapy helps clients around the world accelerate their novel, advanced therapeutic products to market. Our team has a combined 25 years of experience delivering unique lifesaving treatments to market in both EU and US regulatory environments. We have a diverse talent pool of technical, scientific, quality assurance (QA), and regulatory professionals to support you across the full development lifecycle to effectively and efficiently bring products to patients through clinical trials, marketing authorization, marketing authorization, and GMP manufacturing.
At ProPharma Group, our approach is, to begin with, the end in mind. We identify potential challenges and help craft strategies and solutions before you are confronted with them during a regulatory assessment.
Guidance and Services for Cell and Gene Therapy
There are many FDA/EMA regulatory, technical, and scientific challenges that can significantly impact the successful outcomes in product development for cell and gene therapies.
- Classification advice – is your product a CGT/ATMP, a medical device, or a combination?
- Design of GMP manufacturing facilities, including commission, qualification, and validation.
- Risk assessment and gap analysis of data packages against IND requirements or EU requirements for Clinical Trial Applications (CTA).
- Development of effective and customized Quality Management Systems (QMS).
- Consulting on comparability studies for your product before and after changes are made to the manufacturing process.
- Consulting on appropriate development strategies (e.g. biodistribution, tumorigenicity, orphan designations).
- Data summary and document writing (eg, IMPD, IB).
- Clinical Trial Applications, including genetically modified organism (GMO) applications.
- Preparation for Agency discussions with FDA and EMA.
- FDA and EMA classification and certification procedures for CGTs/ATMPs.
- Regulatory aspects of grant applications.
- Clinical investigations for biomaterials.
While the science, quality demands, regulations, and clinical use of Cell and Gene therapy evolves, companies need a partner that can define appropriate development strategies to manage the ever-changing regulatory environment and anticipate what will be required in years to come.
A Global Team Bringing Broad and Deep Expertise in Cell and Gene Therapy
Our Center of Excellence – Cell and Gene Therapy nurtures a think tank of Cell and Gene Therapy experts. We offer more than hiring just one expert resource with access to a full team of industry experts. We provide a comprehensive suite of integrated services and solutions to advance products while maintaining the highest compliance standards. Our team’s expertise supports you throughout the full lifecycle of Cell and Gene therapy product development from early-stage concept development through commercialization.