Moving into the future with cell and gene therapies

Cell and gene therapy – modified to heal

Unlike tissue or stem cells transplantations, most cell and gene therapies have their substantially modified starting materials in common. The results are gene therapeutics (manipulated nucleic acids), genetically modified cell therapeutics, or customized and/or stimulated, expanded cells.

This means that cells are specifically isolated, stimulated, expanded, and/or genetically modified according to the clinical picture. Apart from tumor diseases, genetic defects, congenital metabolic diseases, autoimmune diseases, and tissue injured in accidents can also be treated. The therapeutic approach is not designed to improve the clinical picture in the classical sense, but to heal the cause.

Customized therapy in small batches

Novel preparations are primarily developed in scientific research institutions and clinics and are usually produced in small batches and for individually designed therapy. This has the enormous advantage that the latest findings and innovations can be incorporated into development. However, these facilities often lack regulatory knowledge and financial resources for GMP-compliant drug development. Valicare will be happy to help you realize your goals.

Cell and gene therapy development is a matter of time

The challenge for cell and gene therapies in the development phase is to implement GMP, outline the risks, and define appropriate risk minimization/control measures based on the state of knowledge. To ensure the success of clinical development, subsequent development steps must be considered in a timely manner so that processes, materials, and equipment are still available in the original (or comparable) configuration and the momentum for continuous improvement remains.

Audits for cell and gene therapies

The guideline for the manufacture of ATMPs also specifies the requirements for GMP conformity (Part IV of the GMP guideline). To provide you with optimal support on the way to obtaining a manufacturing authorization (MA) and later in manufacture, we offer you two options for reviewing and improving your processes. 

• Inspections as part of the process of obtaining an MA 

• Third party audits as part of supplier qualification 

Here we take on the challenge of a risk-based approach. 

Your benefits

Our experts have extensive experience in the requirements of pharmaceutical development. We will be happy to consult and support you with:

• GMP-compliant ATMP manufacturing processes including risk assessment
• All processes and GMP requirements for your cell and gene therapies
• Planning and construction of facilities
• Immediate development of manufacturing and quality control concepts
• Development of an optimized pharmaceutical quality management system (PQM)

Would you like to shape the future with your cell and gene therapeutics? Our experienced GMP experts will be glad to support you from development to approval. Get in touch with us now. Together we will take your therapy to the patients.