FIH Studies
Nonclinical Considerations (IND-enabling) for Cell and Gene Therapy Products
Critical elements of a preclinical (IND-enabling) program strategy for cell and gene therapy products include the availability of well-characterized product, choice of the right preclinical model system(s), and pivotal preclinical study designs. A preclinical model selection and study design should be optimized to address specific efficacy and safety attributes of a candidate cell therapy or a gene therapy product.
Availability of a Well-Characterized Product (Pre-IND and IND Stage) Studies
A successful product’s journey towards first-in-human studies (IND-enabling) begins with well-characterized raw material. The quality of raw material can affect the safety, potency, and efficacy of a product. The sponsors need expertise and cutting-edge support in CMC, and good regulatory advice, in order to expedite product approval directly from FDA/EMA or their respective regulatory agencies. As a cell and gene therapy CDMO solutions provider, we offer cell and gene therapy manufacturing products and services, making us your premier CDMO partner from therapeutic development to GMP manufacturing, whether that be for your plasmid, viral vector, or cell sourcing/manufacture.
While there are various guidance documents available for the IND-enabling stage in the US and CTA stage in Europe, they do come down to a case-by-case approach. Given the diverse and often multifunctional nature of development of these biological medicinal products, it is not possible to design one-size-fits all preclinical (IND-enabling) program for cell and gene therapy products.
Cell and gene therapy preclinical programs must be customized to support the feasibility of the proposed application procedure and administration route, assess the candidate product’s specific therapeutic properties, and fully characterize potential safety concerns.
Partner with our multi-disciplinary team of scientific experts who specialize in strategy and execution along complex pre-clinical pathways for cell and gene therapy products. With our global presence and regulatory expertise, we have helped many clients from basic research to IND-enabling and beyond.
10 Regulatory Truths for Biotech Founders
Do not leave your breakthrough to a chance; download this complimentary infographic as a concise checklist to help you stay on track and reach your milestones.
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Cell Therapies
An Investigational New Drug Application (IND) for your cell therapy services requires an interdisciplinary approach to safety assessment, pathology, toxicology, and regulatory experts – that means IND-enabling studies developed by integrating Safety with Discovery. Your cell therapy IND programs are supported case-by-case and based on product attributes, thereby placing personalization and customization at the center of your product goals. Charles River’s Scientific and Advisory Services (SAS) is a team that has extensive expertise in designing development strategies for advanced therapies. We can help you navigate challenges with the development of your cell therapy product.
Gene Therapies
Are you interested in exploring tailored preclinical support for gene therapy toxicology and regulatory product provision? Gene therapy studies present unique challenges, and it is imperative that you select a Contract Research Organization that is equipped to overcome these challenges and ready to partner with you to solve them. You need support to navigate complex scientific and regulatory challenges and our end-to-end offers are available to you under one umbrella.
Gene Therapy Testing Services
Meet the unique and diverse challenges of gene therapy with a team of experts spanning:
- Safety Pharmacology
- Toxicology
- Immunology
- Bioanalysis
- Molecular biology
- Nonclinical strategy
- Regulatory insights
Successful development of ATMP such as a gene therapy, involves a program that is exclusive to a product candidate and patient population, while encompassing broad regulatory expectations. No matter the endpoints we will help you get there.
FOXG1 Research Foundation CEO Nasha Fitter shares milestone updates: FDA Fast Track, IND clearance, and the first FOXG1 gene therapy trial now in reach.
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Why Work With Us
- Global Experience
With 75 years of experience, we have a unique and unsurpassed portfolio to support the development and execution of your cell and gene therapy programs from start to finish. - Proven Track Record
Conducted 1,000+ cell and gene therapy programs for our clients with 100+ years of combined scientific experience. - High Quality Expertise
Our toxicology experts, immunologists, discovery scientists, and regulatory advisors will work closely with regulatory reviewers at the Center for Biologics Evaluation (CBER), the European Medicines Agency (EMA) and other regulatory authorities to ensure your program’s success -- providing complete support for your regulatory and compliance needs. - Cutting-Edge End-to-End Support
With our comprehensive end-to-end portfolio, we have the cell and gene therapy products and services, along with scientific and regulatory experts, to fully support your integrated program.