Cell & Gene Therapies have emerged as a new treatment paradigm for many rare diseases and medical conditions. The rapidly growing market of ‘living’ medicines has the potential to provide long-term therapeutic benefits for patients, however their development could be both risky and cost intensive. As indicated by EU and US regulatory authorities, the number of investigational new cell and gene drug applications has tripled in the last three years. According to the estimates of global data, in 2021, out of the total number of CGT clinical trials, the oncology segment alone consists of 1,375 ongoing trials. Phase 2 accounted for the largest share of more than 52.8% of the total revenue as the majority of cell and gene therapies are in this phase of the clinical development It is expected the industry could see a tsunami of approvals –up to 31 cell & gene therapy launches, including more than 29 adeno-associated virus (AAV) therapies, are expected in 2024 alone. To effectively navigate cell & gene therapy product development, obtaining sufficient information on the specific technological, clinical, and commercial issues is crucial. Implementation of novel clinical solutions relying on patients’ stratification (biomarkers), Real World Evidence (RWE), pricing models and appropriate regulatory approval paths from the early stages of product development all the way to marketing authorization and post-approval, constitute interlocked parts towards long term value creation of cell & gene therapies in the commercial environment.
Among the topics to be discussed will be:
• Outlook, trends, and prospects
• Cell & gene product and process development
• Manufacturing and commercialization aspects
• Smart clinical trial design methodologies
• Real World Evidence generation
• Regulatory strategies in US, EU, and ROW
• Potential solutions to current pricing models
• Reimbursement of Cell & Gene therapies
• Case studies and lessons learned
This 20 hour comprehensive course will be valuable to employees involved in the processes of the cell and gene product lifecycle. This includes EMA classification (ATMPs subclasses), product design emphasizing technological advances of genome editing such as CRISPR/Cas, ZFNs and TALEN, product development and commercialization with focus on European, USA and Japan markets. Further, operational (Contract, Manufacturing & Chemistry), clinical trial design aspects and ‘up-to-date’ regulatory landscapes will be discussed highlighting successful marketing authorization and reimbursement strategies.
The cell and gene therapy product lifecycle course will be of great benefit to professionals engaged in R&D, CMC, Product Development, Clinical Design & Development, Regulatory, Strategy, Operations, Market Access Management, and other Business Decision Makers.
The course can be customized to address specific organizational, departmental, or functional issues and delivered on site.
The cell and gene therapy product lifecycle course provides a solid foundation to understand scientific, global regulatory principles and approval strategies of Cell & Gene Therapies. It allows to comprehend the interconnected activities through the cell and gene product development lifecycle. The course serves as an introduction to the cell & gene product development processes and will familiarize participants with the steps involved in developing cell & gene medicines against rare diseases and other medical conditions from Early Product Development via SMART, targeted Clinical Trials to Commercialization and post-approval Patient Follow-up.
Ensuring the safety and efficacy of gene and cell therapies is an essential yet daunting task during development. The medicines are highly individualized, and both the European Medicinal Agency (EMA) and the U.S. Food and Drug Administration Agency (FDA) have many regulations outlining the approval of these products. For these reasons, safety and risk minimization for patients begins during medicinal product design. For example, by implementing desired properties of viral vectors.
Legal compliance is a challenging endeavor in emerging fields of medicine like cell and gene therapy. Developers must consider both the United States Food and Drug Administration (FDA) and the European Union’s European Medicines Agency (EMA) requirements.
The FDA defines CGT medicines as biological products and regulates this market segment under the Center for Biologics Evaluation and Research (CBER). The EU has its own term, Advanced Therapy Medicinal Products (ATMP). Both agencies recognize that CGT is an emerging field with unique safety and efficacy requirements and have introduced new regulatory pathways to accelerate development and compliance.
The medical industry is searching for ways to scale up cell and gene therapy manufacturing. As the field emerges, unique challenges present themselves:
The clinical trials for cell and gene therapies are significantly different from those of other medical fields. Clinical studies aim to identify safety concerns within medical products. But while many drugs are chemical in nature and have a direct impact on the patient, CGTs are released into host cells. For example, gene therapies with a viral vector serve as the carrier are released into the nucleus of the host cell.
The cell and gene therapy sector will call for cold storage to preserve and transport its medicines and products. The demand for cryogenic facilities, liquid nitrogen, and other storage mediums with temperatures below -80 degrees Fahrenheit rose during COVID-19 to store heat-sensitive CGT products.
Like any emerging field of medicine, cell and gene therapy suffers from limited knowledge. The risk profile for CGT products is relatively unknown. Gene expression is often unpredictable, and clinical trials must measure physiological change carefully in their test subjects.
Determining optimal dosage strategies for cell and gene therapies will be a significant talking point for researchers in the industry. What dosage induces a therapeutic response in the patient without leading to toxicity? How do you prevent issues like liver or kidney damage when administering CGT treatments?
In addition to developmental challenges, cell and gene therapies are currently set back by high dosage costs and affordability concerns for patients. Zolgensma, one gene therapy drug for treating spinal muscular atrophy, notably costs $2.1 million per dose.
To empower an emerging field like cell and gene therapy, collaboration among companies, agencies, patients, and academic institutions will be necessary to alleviate bottlenecks and streamline research and commercial manufacturing.
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