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Cell and Gene Therapy Product Lifecycle - Introduction Course - Gene Editing, CRISPR/Cas, TALEN Technologies

Course Fee

$2650.00 Regular Registration

$2450.00 Early Bird Pricing (Register 30 Days in Advance)

Gene & Cell Therapy Product Lifecycle Course Description

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

Who Should Attend

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.

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Learning Objectives

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. 

Agenda


Welcome

  • General overview
  • Capturing the potential of Cell and Gene Therapies (CGTs)


Harnessing the potential of Cell and Gene Therapies (CGTs) 

  • Definitions and classification of Cell and Gene Therapies
  • Subclasses of ATMPs according to EMEA 
  • Guiding principles of product development
  • Current uses
  • Current challenges
  • Autologous vs allogenic therapies
  • Somatic vs germline therapies
  • In vivo vs ex vivo therapies
  • Development of CAR-T therapies
  • Case studies/Examples


The Current Global Cell & Gene Therapy Landscape

  • The outlook for Cell & Gene Therapies
  • Exploring the current clinical trial landscape and products gearing up for approval
  • The key focus areas to continue the trend of rapid growth in gene therapy
  • Build, buy and partner strategies
  • Case studies/Examples


Genome editing 

  • Zinc-finger nucleases, TALEN technology 
  • Examples of Zinc-finger proteins
  • Examples of TALEN products
  • CRISPR/Cas9 for gene manipulations
  • Applications of CRISPR and development status
  • Gene editing in somatic and germline cells
  • Ethical considerations
  • Case studies/Examples


Technological and clinical challenges & limitations during cell & gene product development 

  • Viral vectors in gene therapies: AV, AAV, lentiviral vectors
  • Non-viral vectors (carriers) in gene therapies
  • Vectors leveraged in cell & gene therapies clinical trials 
  • Cell and gene therapy data management through a product’s lifecycle: research, pre-clinical development, manufacturing, supply chain, clinical
  • Data management systems
  • Operating models
  • ‘Chain of identity’ management
  • Data tracking and sharing
  • Case studies/Examples


De-risking clinical trials

  • Randomized Control Trials
  • SMART Clinical trials design
  • Innovative models for evidence mapping of clinical trials data
  • De-risking the costs of pivotal clinical trials
  • Long term customer and patient engagement - Healthcare provider (HCP) network
  • Case studies/Examples


"Creative" Solutions for clinical development of Cell and Gene Therapies: RWD/RWE

  • Randomized Control Trials
  • Clinical trials in rare diseases
  • Single arm clinical trials and comparators (RWD)
  • Clinical trials data sources and evidence
  • Criteria & limitations of RWD/RWE
  • RWD/RWE during Product development lifecycle
  • Innovative models for evidence mapping of clinical trials data
  • Case studies/Examples


Applications of biomarkers for rare disease patients benefitting from precision Cell & Gene Therapies

  • The diagnostic odyssey of rare disease patients
  • Types of biomarkers
  • Applications of biomarkers through the product lifecycle
  • Companion diagnostics – regulatory approval paths 
  • Case studies/Examples


Analytical Development for Cell and Gene Therapies Products

  • A Gene Therapy Medicinal Product (GTMP) according to regulators
  • Extended characterization studies and their relevance for release testing and criteria of a GTMP
  • ‘Sufficient’ data for safety, efficacy, quality, and consistency of GTMPs
  • Orthogonal and gold standard analytical methods to support with benchmarking activities
  • Bioassays & potency assays
  • Physico-chemical methods
  • Case studies/Examples


Commercial challenges: understanding product and process development 

  • Overview of gene therapy manufacturing
  • Quality Target Product Profile (QTPP): CMAs, CPPs, CQAs
  • Quality by Design System (QBD)
  • Manufacturing of conventional biologics and AAVs
  • Status of the viral vector for CGTs according to EMEA and US FDA (‘starting material’ vs ‘critical raw material’)
  • Quality attributes risk assessment
  • Upstream process parameters
  • Downstream process steps
  • Stability Study
  • Improving product comparability
  • Costs of Goods (COGs) reduction via automatization
  • Case studies/Examples


Advanced therapy medicinal products (ATMPs) - EMA legislation 

  • ATMPs regulation: rationale
  • EMA Committee for Advanced Therapies (CAT)
  • PRIority Medicines (PRIME) designation
  • Regulatory pathways to Marketing Authorization (MA)
  • The hospital exemption scheme
  • The centralized procedure (CP)
  • Early access mechanism - Conditional MA, Exceptional Circumstances MA and Accelerated Assessment
  • Orphan designation
  • Incentives in the ATMP regulation (scientific advice, ATMP classification, certification)
  • What is new?
  1. New centralized procedure for clinical trials submission
  2. GMO national requirements
  • Case studies/Examples


Cell and Gene Therapies – US FDA legislation 

  • General Regulatory Framework
  • The Center for Biologics Evaluation and Research (CBER)
  • FDA’s Office of Tissues and Advanced Therapies (OTAT)
  • Investigational New Drug (IND) regulations 
  • Expedited pathways for CGTs (FTD, BTT, RMAT, PR, AA)
  • The Biologics License Application (BLA) procedure for commercialization
  • Case studies/Examples


Approval pathways for GCTs in Japan

  • Japanese regulations on CGT medicinal products
  • Expediated pathways for CGTs in Japan
  • Priority review
  • Conditional Approval
  • Term limited approval
  • The Sakigake early access scheme
  • Case studies/Examples


Cell & Gene Therapy Development and patient engagement

  • Patient perspectives from the early stage of product development 
  • Managing expectations among different stakeholders
  • Partnering with patient communities
  • Post-authorization obligations for the benefit-risk balance reporting
  • Post-approval commitments 
  • Case studies/Examples


Potential solutions to current pricing models and reimbursement for Cell and Gene Therapies

  • Conventional therapy vs Cell & Gene Therapy
  • Challenges for HTA bodies and reimbursement
  • Multi-stakeholder perspectives
  • Existing reimbursement systems
  • Innovative payment models
  • Potential implementation barriers and opportunities of practical implementation
  • Case studies/Examples


The product development roadmap (Mini workshop)

  • Ideation
  • Discovery
  • Prototyping
  • Detailed Design
  • Validation & testing
  • Commercialization
  • Case Studies/Examples


The Talent Roadmap (Mini workshop)

  • High Performance Teams
  • Aligning People with Business Strategy
  • Ability to identify, attract, develop, and retain top talent
  • Organizational culture: feedback, development, and engagement
  • Case Studies/Examples


Course Review

  • Wrap-up
  • Final Q&A
  • Course Evaluation


Certification exam

  • Queries


Resources and Recommended Reading

  • Regulatory guidance documents
  • Health authority websites
  • Industry organizations and publications
  • Textbooks

FAQs

  • How do you ensure the safety and efficacy of gene and cell therapies throughout the development process?

    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.

    Learn more

  • How do you navigate the regulatory landscape and obtain necessary approvals from regulatory agencies such as the FDA and the EMA?

    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.

    Learn more

  • How do you optimize and scale up the manufacturing process for gene and cell therapies, including the selection of appropriate cell lines and vectors?

    The medical industry is searching for ways to scale up cell and gene therapy manufacturing. As the field emerges, unique challenges present themselves:

    Learn more

  • How do you design clinical trials that effectively demonstrate the safety and efficacy of gene and cell therapies in target patient populations?

    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.

    Learn more

  • How to ensure long-term product stability, including the preservation and storage of cell-based therapies and the durability of genetic modifications?

    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.

    Learn more

  • How to monitor and manage the risk of adverse events and side effects associated with gene and cell therapies?

    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.

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  • How to develop appropriate dosing and administration strategies for gene and cell therapies, taking into account factors such as the duration of therapeutic effect and potential for off-target effects?

    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?

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  • How to manage the cost-effectiveness and affordability of gene and cell therapies, including the development of reimbursement and pricing strategies?

    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.

    Learn more

  • How to foster partnerships and collaborations with academic, industry, and patient organizations to support the development and commercialization of gene and cell therapies?

    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. 

    Learn more

Registrant Information:

Each person attending a course will be asked to set up an Attendee Profile Account during the registration process. Creating an Account helps you view your order history and manage your training programs. If you are registering for others, please set up an Account in the Attendee’s name. If you are registering more than one person, you’ll need to set up a separate account for each Attendee.

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