Cell and Gene Therapies
Insights on delivering cell and gene clinical trials in multiple therapeutic areas based on lessons learned and best practices
Approaching the CAR T-cell therapy horizon
Approved CAR T-cell therapies have shown remarkable results in patients with certain types of blood cancers, but further innovations are needed before the technology can reach its full potential. In this whitepaper, we explore the most exciting clinical developments in CAR T-cell therapy, and relate them to limitations of approved therapies.
Mainstreaming Cell and Gene Therapies
The cell and gene therapy (CGT) market is rapidly transitioning from ultra-niche cutting-edge science to approved and available therapies that can address previously intractable and often devastating diseases.
Long-term follow-up studies of cell and gene therapies
Regulators typically expect that Sponsors collect up to 15 years of follow-up data on patients who’ve received cell and gene therapies either through a clinical trial or in clinical practice.
The affordability hurdle for gene therapies
Until now US payers have absorbed and managed the cost of ultra-expensive therapies, but how long can they continue to do so? Read our whitepaper to understand the payer perspective on advanced therapy medicinal products.
The promise and complexity of living therapies
As development of cell and gene therapies (CGT) accelerates, so will the demand for best practices, and better tools and solutions. For advanced therapies, the product and patient journey is entirely different from traditional trials. As such, biotech and pharma companies will need to overcome challenges and complexities from regulatory pathways and patient recruitment to logistics and manufacturing. Adopting strategic partnerships with deep CGT expertise with a broad spectrum of solutions and services is vital to the success of a development programme.
Enabling clinical development of Cell and Gene Therapies on a global basis
ICON's CGT team share insights on best practices, custom resources and innovative tools for enabling clinical development of cell and gene therapies on a global basis in this BioInsights podcast (40 mins).
Download the full transcript here.
Cell and Gene Therapy clinical development webinars
Best practices for laboratory solutions relating to cell and gene therapy clinical trials
In this webinar, we discuss factors to consider when executing CGT trials, illustrating how specialty molecular and flow cytometry approaches translate into best practices to support the unique patient, product, site, data and regulatory journeys.
Navigating the future of CGT: The potential for broader indication
This roundtable will explore the significant opportunities and challenges posed by CGT clinical trials, and how a CRO partner may be uniquely placed to help a pharma innovator navigate them.
The importance of early planning in CGT commercialisation
This roundtable will identify successful strategies that underpin currently-marketed therapies and examine how establishing a framework early in development helps keep long-term production and commercialisation goals on track.
Implementing novel development strategies for CGT clinical trials
This roundtable will review the necessary considerations and logistics of conducting CGT trials, and how sponsors and service providers can optimize their clinical research to maximize the prospect of success.
The changing face of cell and gene therapy
Considerable strides have been made in the development of cell and gene therapies since the first autologous CAR T therapy was approved by the FDA four years ago.
Designing and conducting effective long term follow-up studies
New regulatory requirements have sparked more interest in stand-alone long-term follow up studies for monitoring patient outcomes and safety.
Patient advocacy and access to innovative clinical trials in a changed environment (COVID-19)
Working with “living therapies” during COVID-19 has changed access, resource planning and management in CGT trials.
HTA and market access implications for gene therapies
A key challenge for manufacturers is to demonstrate value to payers in a health technology assessment (HTA) setting.
Achieving quality cell and gene therapies outcomes through multidisciplinary collaboration
Explore how cell and gene therapy related experience can promote quality of data and higher patient accrual.
Cell and Gene Therapy blogs and media contributions
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Media article: Lifecycle management for CGTs: Solving operational challenges in an expanding market
The development of cell and gene therapies (CGTs), such as CAR T-cells and autologous and allogeneic therapies, is gaining momentum. To deliver these cures to patients with unmet needs, life sciences companies must navigate clinical trials—a process that is more complex for CGTs than for small molecules.
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Media article: Practical Considerations for Managing CGT Supply Chains
More than in traditional trials, the unique manufacturing process and logistical complexity of CGTs require intense resource allocation, meticulous planning and rigorous controls. In this article, we outline practical considerations for managing your CGT supply chain to minimize risk, mitigate cost, and complexity.
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Media article: Innovative laboratory testing methods for clinical monitoring of cell therapies using flow cytometry DDPCR and QPCR assays
This article explores how unique applications of PCR and flow cytometry can be used to provide critical and complementary data that is needed to bring new life-saving treatment options to patients.
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Media article: Logistical considerations in mRNA vaccine development
In this article, ICON’s Andreas Dreps and Martin Lachs discuss the logistical considerations in mRNA vaccine development.
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Media article: Challenges of expanding CAR-T Cell Therapy into solid tumors
Learn more about the challenges associated with developing CAR-T therapies for solid tumors in this article.
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Media article: Mainstreaming cell and gene therapy – Realizing its potential
This article looks at the Cell and gene therapies (CGT) landscape, the challenges biopharma companies face in running clinical trials and tips on how to surmount those obstacles.
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Media article: CAR-T cell therapy in oncology
In this article, Brian Huber and Tamie Joeckel, consider how cell and gene therapies could change the future of cancer treatments and bring new options to patients.
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Blog: Navigating the complexity of CGT clinical trial design
This blog will explore some of the challenges of CGT clinical trial design, such as patient recruitment and data collection, and strategies for how to approach them.
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Blog: Clinical development challenges faced by advanced therapies for rare disease
Advanced therapies are a class of products based on genes, tissues or cells used to treat or deliver treatment to patients.
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Media article: Cell and gene therapy specific market authorisation guidances
Brandon Fletcher offers his insights on the key changes in regulatory guidance documents released by the US Food and Drug Agency.
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Blog: Cell and Gene Therapy: Specific Market Authorisation Guidances
Therapeutic developers may benefit from working with a partner whose regulatory expertise with CGTs will help ensure that development programmes are optimal and compliant.
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Media article: Gene therapy – affordability, access and reimbursement
Brian Huber considers the affordability, access and reimbursement challenges within the gene therapy market.
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Media article: ICON Q&A - talking affordability in cell and gene therapies
Tamie Joeckel discusses the challenges of making cell and gene therapies affordable for payers and patients alike.
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Media article: The new oncology: What makes CGT cancer trials different
Brandon Fletcher, CGT Principal, discusses how oncology is adapting to cell and gene therapy, and what can be done to improve take-up across the industry.
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Media article: Overcoming data flow challenges in cell therapy trials
How data flows in cell therapy studies differ from other studies and strategies to manage the high volume of data in CGT trials is highlighted in this Q&A with ICON expert Olivier Saulin.
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Media article: Lessons from the first generation of cell and gene oncology trials
A discussion on best-practice approaches to CGT studies and what the future could hold for this exciting area.
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Blog: Navigating complexity of cell therapy manufacturing during the COVID-19 pandemic
Over the past year, the COVID-19 pandemic significantly disrupted clinical research across the lifecycle including recruitment challenges, protocol amendments and delayed market entry.
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Blog: Navigating complexity in oncology cell and gene therapy clinical trials
Cell and gene therapies (CGT) are playing an increasingly important role in treating oncology patients.
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Blog: Cell and gene therapy trials - delivering differently
The success of cell and gene therapies (CGT) has transformed the delivery of clinical trial services, requiring the design of new workflows, processes and tools.
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Blog: Adoptive cellular transfer trials demand that participating sites act differently
The evolution of adoptive cellular transfer (ACT) for the treatment of lymphoma, leukaemia and myeloma patients has grown exponentially.
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Blog: Enabling clinical development of cell and gene therapies on a global basis
Clinical trials for cell and gene therapies (CGT) present unique challenges as they require special resources and training.
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Media article: Best practices in managing CGT from protocol design to data management
As this new frontier of medicine continues to expand a growing number of pharmaceutical and biotech companies will delve into CGT clinical trials.
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Media article: Navigating cell therapy manufacturing amid pandemic woes
Automation, digitalisation, and supply-chain strategies help mitigate vulnerabilities in both autologous and allogeneic cell therapy manufacturing.
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Media article: Navigating complexity in cell and gene therapy clinical trials
As more knowledge is revealed about the genetic underpinnings of cancers, cell and gene therapies (CGT) are playing an increasingly important role in treating oncology patients.(PDF)
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Media article: Cell therapies - the living end of growth opportunities
As the incidence of chronic diseases, such as cancer, is rising, so too is the interest from industry in the area of cell and gene therapies.
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Media article: Clinical development operational implications of the move from autologous to allogeneic ACT paradigms
Allogeneic CAR-T cells can be manufactured using T cells from just one, single healthy donor and can be used in multiple patients. (PDF)
Receive more insights on cell and gene therapies from ICON
Please visit ICON's Preference Centre and select 'Cell and Gene Therapies' under 'Therapeutic Areas of Interest' to receive new insights on cell and gene therapies.
Cell and Gene Therapies at ICON
ICON’s Cell and Gene Therapy (CGT) Solution was designed to expedite every aspect of a cell therapy clinical trial and address specific needs of CGT product development. The CGT Leadership Team leads your trials with innovative technologies and standardised tools for CGT trial execution, management of high data volumes, and extensive team and site training. Read more about ICON's services.