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While the potential for advanced therapies to provide curative treatments to patients around the world is very exciting, the pricing strategies and reimbursement models to support this innovation are quite complicated. In this article, I'll explore the cost vs. price paradigm, through a supply chain lens, highlighting hidden costs and their impact on reimbursement potential.

Industry experts address cell and gene therapy data challenges and what the product and process data-management ecosystem looks like for cell and gene therapy development and manufacturing.

Bringing cell and gene therapy drugs to market requires improvement in many areas, including analytical testing methods, which are used to determine the safety, strength, and purity of viral vectors.

Thorough consideration of cleaning and disinfection during facility design eases maintenance across the facility life cycle, optimizes workspace utility, and contributes to efficient facility operation.

While these new modalities are generating significant excitement among patients and caregivers, cell and gene therapy manufacturers are experiencing growing pains. The rapid growth and continued momentum of the industry has led to the issuance of new guidance. In this article we share insights for navigating within a novel and complex regulatory environment, mitigating risks posed by raw materials and implementing proper risk assessments during manufacturing.

Because L-Tyrosine is an important amino acid for protein synthesis and cellular metabolism, finding a solution to its solubility challenge can be critical to maximize process performance. 

The logistics of decoupling drug substance manufacturing from final drug product formulation is now possible using integrated single-use freeze-thaw assemblies that incorporate reliable components.

A recent study by the Tufts Center for the Study of Drug Development (CSDD) compared cycle times and development economics between multi- and single-source CDMO models.

As we explore immune mechanisms using technology, we find better ways to fight disease. Here, we focus on targeted, highly personalized cancer therapies called tumor-infiltrating lymphocytes.

There is an opportunity for medical monitors to redefine their role in a new era of tech-enabled, productivity-focused digital data analysis. In this white paper, we'll explain what distinguishes this emerging role from its more traditional predecessor, and we'll leave you with a keen sense of how to identify, hire, and empower a modern medical monitor to help your next study proceed toward submission more productively and with greater data integrity than ever before.

Typically, demand forecasts for drugs prior to launch reflect a huge range of expectations. This illustrates just how hard it is to forecast accurately. Instead of trying to make better predictions (which will never be wholly accurate nor truly able to remove risk), or erring on the side of overcapacity (which is a drag on ROC), developers and CDMOs should seek manufacturing flexibility.

A client engaged the Veristat team in a Phase 2 study of intracerebral stem cell injection in patients following an ischemic stroke. The goal was to look at limb recovery time and level in patients with stroke. The effort began with identifying the best time for a product to be delivered and then expanded into full-service involvement, including project management, clinical monitoring, medical writing, and regulatory affairs. Learn how we overcame unique challenges in this multicenter trial that led to the client publishing positive Phase 2a data.

Laboratories are an integral part of the pharmaceutical industry. They are used at each stage of the product development. See how this solution allows you to plan projects without having to build new labs and gain the ability to plan projects better, manage inventory, be nimble in responding to demand fluctuations, avoid under- and/or over-utilization of talent, and ensure reliable capital project planning that is based on actual utilization tracking.

Understanding how to incorporate technologies and strategies that decrease the reliance on brick and mortar sites and bring the study to the patient is critical across all phases of drug development in today's challenging environment.

The storage, handling, and transportation of products in bioprocess development presents risk of breakage, contamination, and product loss. Single-layer fluoropolymer bag assemblies eliminate these risks.

The rapid success of gene therapies also brings with it a number of challenges, the most significant being ensuring a sufficient viral vector supply, despite shortened development timelines. This infographic demonstrates how cells grown in suspension are much better suited for large-scale production of viral vectors, what to consider in order to successfully scale-up your viral vector production, including the challenges faced in upstream upscaling, and a specific solution we've developed to help you address these challenges.

See how LabConnect's breadth of industry experience and a unique model differentiate our services from those provided by other companies.

The most common storage is ultra-low temperature storage, but the fastest-growing is cryogenic, in vapor-phase liquid nitrogen. This transition is huge. Chain of temperature and custody data is imperative.

The breadth of digital PCR (dPCR) applications continues to grow and fulfill critical scientific needs. One of the major advantages the technology confers is the ability to count nucleic acid molecules directly, rather than extrapolating quantity based on standard curves, as with many other methods. By harmonizing diagnostic procedures on dPCR platforms, the scientific and medical communities can become more coordinated and efficient than ever before.

Frost & Sullivan recently invited industry leaders with gene therapy experience to participate in a new thought leadership forum, our Virtual Think Tank. This forum brought together leading minds in this emerging field to discuss key challenges and other insights related to reducing the cost of goods (COGs) for gene therapy.

When it comes to dermatologic conditions, gene therapy is still in its very early stages. However, we are seeing promising potential solutions for some rare genetic dermatology diseases, as well as ongoing research in more common skin conditions. In our first article, we reviewed gene transfer techniques and gene delivery systems that could potentially be used for treating dermatologic conditions. Now we turn our attention to the current state of gene therapy for dermatologic conditions.

Biopharma manufacturers can benefit by adopting a global single-use supply chain strategy early in the drug development life cycle and carefully evaluating their single-use equipment and materials suppliers.

While the solubility of free L-Cysteine is high at neutral pH, the applicable concentration in basal and feed media is limited. Read more about an available solution for solubility and performance challenges.

The design and implementation of risk management programs can be challenging. This white paper uncovers key lessons learned from our experience with over 120 RiskMAPs and risk evaluation and mitigation strategy (REMS) programs - which cover various therapeutic areas and targeted risks.

One of the new technologies emerging in the biotech manufacturing market is the use of viral vectors for cell and gene therapy. One commonly used viral vector in this space is lentivirus, due to its robustness to package and deliver target DNA. This work explores a variety of lentivirus harvest clarification options and items for consideration during manufacturing platform development of viral therapy production using suspension cell lines.

As drug developers face the ever pressing need to get molecules to market as efficiently as possible, firms large and small are increasingly turning to CDMOs for help.

Building your biotech team isn't easy, but it's central to your value. In fact, Roivant Sciences founder and CEO Vivek Ramaswamy believes people are the most important ingredient for launching a biopharma company. Here he shares more about why finesse, not formula, should drive your hiring decisions.

The pharmaceutical industry has grown from simple aspirins to IV delivery, antibiotics, and, most recently, cell and gene therapy. Products are more complex, tailored, and varied than ever, and the associated amount of data is growing, which requires a new level of attention to detail. Learn what features to look for when choosing a data management system to establish compliant data visibility across teams, sites, and partners and establish data integrity throughout the product life cycle.

Contract development and manufacturing organizations need to play a critical role in the scale-up and industrialization of cellular therapies by providing customized tools and processes. The manufacturing by design approach for addressing the industrialization of cell therapies allows for optimal production conditions.

The large size of plasmid DNA (pDNA) can present a challenge for sterile filtration unit operations within your gene therapy manufacturing, since the product can be retained by the filters, leading to both yield loss and low filtration capacity. Optimization of a sterilized-grade filtration unit can help increase yield and filtration capacity. Read on to learn more!

It's impossible to run a global clinical trial of an investigational diabetes drug without ancillary materials. And, as one leading multinational pharmaceutical company recently learned, managing ancillaries can be a challenge every bit as complex as the trial itself.

Single-use plastics are susceptible to the cold temperatures required to keep biologics inert, leading to bag failure and product loss in cold supply chains. Single-layer films are changing the game.

Despite the public attention and large population impacted by HIV, developing a cure has been particularly difficult. Will gene editing techniques, such as the CRISPR-Cas9 system, change the paradigm?

While quality management is a major priority across the life sciences industries, it takes specific precedence in viral gene therapy. Heather Burnell, head of quality at MilliporeSigma's viral vector manufacturing facility in Carlsbad, CA, highlights how quality management is embedded in the MilliporeSigma culture.

Have you noticed that it has become increasingly difficult to transport your material into and out of the United States? That's because it has!

The advent of CRISPR as a gene editing tool continues to revolutionize drug discovery and development.

Patheon''s Bill Weiser discusses how a new generation of CDMOs have positioned themselves to provide strategic solutions that not only complement product development but drive therapies to patients faster.

Multi-time emerging biopharma leader Kavitha Iyer-Rodrigues, founder of Zumutor Biologics, Inc., offers experience-based insight into maximizing the value of incubation time, and knowing when it's time to spread your wings and step out of the nest.

Decades of painstaking research have recently begun to yield gene therapy products that are delivering meaningful benefits to human health. With the rapid evolution of the gene therapy field, regulatory agencies have been working to keep pace with these scientific and clinical breakthroughs.

With viruses playing a greater role in everything from vaccine development to gene therapy, ensuring their rapid, accurate, and biologically relevant enumeration isn't just an analytical "plus;" it's the first step in the development of biological tools that support human health.

There are several challenges related to the development and manufacturing of adeno-associated virus (AAV)-based processes, including characterization, quantification, and downstream purification. LumaCyte's Radiance instrument, which uses laser force cytology (LFC) to measure the intrinsic biophysical and biochemical properties of single cells, has the potential to improve the characterization of cell-based AAV transfection and production.

The storage, handling, and transportation of products in bioprocess development presents risk of breakage, contamination, and product loss. Single-layer fluoropolymer bag assemblies eliminate these risks.

In this virtual roundtable, the creators of iCON, G-CON Manufacturing and Integrated Project Services - IPS, along with collaborating partner, Asgard Cleanroom Solutions, hold an open discussion, answering your frequently asked questions about the industry's first turnkey modular facility platform.

Learn how to overcome the challenges of combining multiple registries to generate richer data in order to better understand disease progression and real-world practice patterns.

It wasn't Paris in Spring, but the annual meeting of the International Society of Cellular Therapy (ISCT) 2020 did happen in May, albeit virtually. From the comfort of their home offices, with snacks at the ready, attendees devoured sessions on some key topics of the day through a cool digital interface replete with avatars. Here are my highlights from a session that introduced a new way to ship and transport cellular materials, putting patients front and center.

There's no question that in the digital age, pharma and biotech production must be supported by manufacturing execution systems (MES). These systems digitize end-to-end pharma manufacturing processes - from receiving goods via warehousing through to production and packaging. Using MES results in process improvements and significant efficiency enhancements.

Cell therapies have the potential to revolutionize the biopharmaceutical world, but today's processes, logistics, and delivery make for a challenging entry into the sector's growth curve. As the industry evolves, we have to answer (at least) three important questions when bringing these exciting new therapies to market.

Looking to scale up your liquid bioprocessing and decrease transfer times? Learn how high-flow, genderless, large-format connectors make scale and efficiency easy and reliable.

Gene therapy has the potential to change countless patients' lives. Diseases that lacked cures, or even treatments, may be addressed with these new classes of therapeutics. This potential has spurred new investments in the drug development and commercialization space.

This case study describes the use of dispersed storage and cold chain logistics around a central manufacturing facility to create a cost-efficient and robust supply chain.

Lentiviral vectors (LVV) are the most common delivery method for transducing T cells for CAR-T therapies, and their production and purification are major cost drivers in manufacturing. Effective and consistent purification strategies for LVV are a serious challenge. Although much work needs to be done to fully optimize each unit operation, we present here our results to date in designing a fully scalable, single-use consumables, closed, lentiviral vector purification process.

This joint webinar with Accenture explores the challenges within today's increasingly decentralized and complex supply chain and how sponsors and CDMOs plan to address them.

Explore the needs and requirements of equipment and processes to handle lower temperatures, including a complete, cold-resistant solution of bags, tubing, connectors, and shells for freeze/ship/thaw processes.

Though pharma companies do their best to understand the environment, it can be difficult to predict demand, and the consequences of incorrect forecasts can be very costly in a number of ways. 

Frost & Sullivan recently invited industry leaders with gene therapy experience to participate in a new thought leadership forum, our Virtual Think Tank. This forum brought together leading minds in this emerging field to discuss key challenges and other insights related to reducing the cost of goods (COGs) for gene therapy.

A new end-to-end system has been developed and implemented in several locations worldwide to address the issues encountered with formalin-fixed paraffin-embedded (FFPE) block storage, retrieval, and restocking.

Biopharma execs must consider how outsourcing fits into development and production strategies, and how to select the best CDMO partner for their business. Here's a step-by-step guide to those decisions.

Lentiviral vectors (LVV) are a common vehicle to deliver genetic material in CAR T-cell therapy and gene therapy applications. Production methods have been developed by adapting technologies from the bioprocessing sector. However, these downstream workflows are long, require substantial manual labor, and suffer from low yields of infectious virus. In this article we discuss process development pain points of a modern workflow and ways to address them.

While ultra-low temperatures are the current state-of-the-art solution for biospecimen storage, ambient storage is an interesting option and could be superior in certain limited situations. For many types of samples, it's possible that we will always have to use ultra-low temperatures for long-term storage. For other sample types, such as DNA and RNA, there are alternative solutions.  

In May 2020, G-Con conducted a survey to assess the impact of COVID-19 on the biopharmaceutical industry. We''re giving you a summary of the results in the infographic. How does this compare to your organization''s experience?

Process development (PD) is a critical component to the commercialization of viral vector-based therapies. While some gene therapy developers may want to speed through this operation to meet deadlines, doing so can put product quality, safety, and efficiencies at risk.

Dr Michael May, president and CEO, CCRM (Canada), discusses the impact of COVID-19 on the regenerative medicine sector, and how the current scenario fosters new collaborative models in a post-COVID-19 world for all stakeholders, at the inaugural Business of Regenerative Medicine Asia Pacific Symposium (Virtual) co-hosted by CCRM Australia and the NSW Stem Cell Network.

CRISPR''s speed, low cost, and ability to be used in many model systems redefines what is possible in drug discovery and development. However, current workflows for generating knockout cell lines using CRISPR-Cas9 can be complex and inefficient. In this study, we demonstrate a novel workflow that includes Droplet DigitalT PCR, high-resolution melt analysis, western blotting, and real-time PCR to generate and validate a knockout haploid cell line in less than five weeks.

This article captures learnings about the use of single-use technologies in biopharma manufacturing that can be applied to cell and gene therapies, with a special focus on connection technologies.

One of the most significant moves for a growing biotech or pharmaceutical company - sometimes considered as a graduation step - is the acquisition of property to conduct clinical trials and manufacture commercial products. In the first installment of this two-part series, we will explore what considerations companies need to make before they decide what type of property to pursue.

Though pharma companies do their best to understand the environment, it can be very difficult to predict demand, and the consequences of incorrect forecasts can be very costly in a number of ways. 

Every last day of February is Rare Disease Day, the biggest day of the year for the rare disease community. Read some highlights from this year''s event attended by researchers, physicians, regulators, and patient advocates.

Understanding the cell and gene therapy (CGT) development process from start to finish often comes down to learning the language. For individuals more familiar with the early R&D steps in this process, terms such as IQ, OQ, and PQ - associated with later manufacturing steps - may be completely new. Understanding IQ, OQ, and PQ will set you up for success in transferring your process to a good manufacturing practices (GMP) facility.

Today's cell therapy treatments are often made at a small scale, include manual preparation, and are usually produced for use in a clinical trial. Researchers spend days processing cellular material, monitoring its growth during the expansion phase, and preparing it for readministration to the patient. In return, cell therapy offers life-changing outcomes. But what if it were possible to get results on a much broader scale? Automation may hold the key to making this happen.

In 2019, G-CON Manufacturing celebrated its 10-year anniversary. Here is an overview of how far our technology has progressed over those 10 years.

A crucial component in reducing the cost of goods sold (COGS) in developing cell, gene, and stem cell therapies is through increased efficiency of the manufacturing process. Automation provides a suitable option to address this challenge. Those manufacturers who have become early adopters of digitization, automation, and continuous improvement are reaping huge benefits.

With the rapid growth of single-use systems (SUS) for drug production, especially by drug manufacturers with multiple locations across the globe, there are many risks associated with underestimating the regulatory compliance, manufacturing, and quality requirements of these systems. To minimize these risks, biopharma manufacturers can benefit by adopting a global single-use supply chain strategy early in the drug development life cycle and carefully evaluating their chosen single-use equipment and materials suppliers.

The secure, leak-free connection of sensors to single-use assemblies is paramount in ensuring both the accuracy of the measurements taken and the protection of the manufacturing process of expensive drug products.

Using a risk-based approach to generate an appropriate control strategy will help ensure your product meets characterization and validation requirements, enabling timely approval and launch to market.

va Industry thought leaders working in the viral vector manufacturing space recently participated in a virtual think tank (VTT), a unique thought leadership platform that brings together leading minds, to discuss viral vector manufacturing facility design. The focus of the most recent VTT was on the current challenges and trends influencing design considerations for viral vector manufacturing facilities and how they can impact companies developing cell and gene therapies.

L-Tyrosine is an important amino acid for protein synthesis and cellular metabolism; however, with a solubility of less than 0.5 g/liter in water at neutral pH, L-Tyrosine is especially challenging. One approach that is being rapidly adopted by the industry is to overcome these challenges by replacing such amino acids with dipeptides.

Still relying on spreadsheets, paper records, and disparate data systems? Enterprise systems too costly? Skyland PIMS? provides emerging and global drug sponsors and CMOs a collaborative, end-to-end workspace to seamlessly combine product, process, and patient data management with analytics, charting, and continued process verification (CPV) reporting.

The current implementation of single-use bag technology is reducing operating costs and mitigating the contamination risk of traditional stainless-steel freezing tanks and bottles. However, under frozen temperature these single-use components are brittle and prone to failure that can lead to product loss and contamination, especially during transportation. This application note will discuss the results of applying an approach using sensors and more suitable material to a frozen shipping study of single-use fluoropolymer bags.

Do pharmaceutical companies underestimate the challenges of transportation throughout the supply chain? There are many pain points when it comes to sending a therapy from A to B, but, with the right collaborations in place, these can be mitigated.

To be able to meet the growing needs of patients around the world, a company must have a strategy that can shepherd new biologics therapies throughout the development timeline.

Advances in immuno-oncology have led to the advent of CAR T-cell therapy, which combines a patient's own T cells with engineered T-cell receptors known as "CARs." The CAR enables the final product to produce chemicals in the hopes that the "enhanced" product or cells will bind to the cancer cells and kill them. In this free webinar, learn about the principles of CAR T-cell therapy and the ways these technologies can reach patients.

During the 12^th REMS (risk evaluation and mitigation strategies) Summit in Virginia, UBC's Natalie O'Donnell, corporate vice president, safety, REMS, and strategic engagement, UBC, and Justin Wilson, director, software development and technology, UBC, presented a session on the impact that technology has had on REMS and the potential effect it can have in the future.

Maintaining the appropriate storage temperature of biological material is critical for downstream use. However, due to the complexity of this material and its sensitivity to temperature we are seeing an increase in nontraditional storage temperature requests. In this article we'll explore some of the reasons why this is occurring and the associated challenges.

To address the growing need for speed and efficiency in viral vector manufacturing, GE developed a process for adenovirus manufacturing that not only meets regulatory requirements but is also scalable.

The traditional business model for in-house pharma manufacturing is nearly a thing of the past. More companies are turning to outsourcing to achieve flexibility and efficiency in a highly competitive market. 

Whether the experts come from a CRO or a regulatory consultancy, their help will be critical in helping develop the trial, meet with the Institutional Biosafety Committee, establish a timeline, and manage expectations. Here's why.

A single vendor offers access to a network of experts who can share knowledge about a project as it moves from phase to phase, thus helping to navigate its path toward commercial success. 

Life sciences industries require highly controlled environments for drug research development and manufacturing.

A leader in the development of restorative cell therapies has a pipeline of products that may offer rapid-onset pain relief and restored function for orthopedic use. By implementing Skyland PIMS into their manufacturing plan, the drug developer is able to securely manage critical data in a collaborative, cloud-based, 21 CFR Part 11 compliant workspace.

With the current increase in development and investment, gene therapy companies will increasingly be looking to streamline the manufacture and commercialization of their therapies. Choosing a contract development manufacturing organization (CDMO) to ensure a smooth road to commercialization is essential. Contact us directly to learn more about picking the right strategic partner.

This podcast discusses the project execution, challenges, achievements, and success with a global pharmaceutical company whose goal was to scale up their manufacturing operations in the U.S. and Europe for an approved CAR-T product.

How high-density cell banking accelerates upstream processes by providing a larger volume of concentrated cells at the start of a production run, shortening seed training from weeks to days. 

When quantifying nucleic acids via real-time or quantitative PCR (qPCR), high accuracy and reproducibility are essential. However, factors such as inconsistent pipetting and instrument design (specifically a lack of thermal or optical uniformity across the reaction plate) can lead to variations in fluorescence measurement.

Patient support services must continue to evolve and flex as the oncology market transforms. Here are five trends to consider for successful support of cancer patients in the future.

In 2019, G-CON Manufacturing celebrated its 10-year anniversary. Here is an overview of how far our technology has progressed over those 10 years.

It's no secret that drug forecasts are notoriously incorrect, and often by large margins. With no signs of improvement in forecasting accuracy, how do companies minimize risk?

Cost-effective manufacturing of lentiviral vectors (LV) at commercial scales has proven difficult and remains a pressing issue for the marketing of therapies that depend on their application.

Having a sound logistics strategy to ensure that a living drug is delivered to the right patient at the right time, location, and temperature is essential to patient safety and product effectiveness.

Pluripotent stem cells (PSCs) and induced pluripotent stem cells (iPSCs) are promising sources for the next wave of cell therapies and regenerative medicine. As with many cell therapies, one major technical hurdle is the scalable production of PSCs for commercial applications, specifically when processing these cell types in aggregate form. Read how the ekko system enables automation of the media exchange and subsequent harvest steps. 

The curing potential of cell and gene therapies is driving the industry to gain a better understanding of the evolving regulatory guidelines for these products, in order to bring them to market faster.

Learn about the challenges for sponsors and CROs when integrating a vast array of data sources and typical relational database structures, why they don't work for clinical analytics, as well as the ideal clinical data aggregation solutions for continuous, automatic data consolidation.

L-Cysteine and its oxidized form L-Cystine are essential amino acids used in cell culture and are present in all chemically defined media formulations. However, while the solubility of free L-Cysteine is high at neutral pH, the applicable concentration in basal and feed media is limited. Read more about an available solution for solubility and performance challenges.

With more complex molecules in development, changing capacity needs, uncertain forecasts, and increased competition, the market demands flexibility and innovative approaches to today's challenges.

In 1931, Swiss doctor Paul Niehans injected a preparation of live cells from a parathyroid gland into a dying patient. The patient subsequently recovered, and Dr. Niehans had a eureka moment - that injections of living cells can have tremendous therapeutic value.

A clinical hold from the U.S. Food and Drug Administration can significantly prolong the time and increase the cost of drug development, which is particularly concerning for emerging/small biotech and specialty pharma companies. In this blog, we discuss common reasons for clinical holds and provide useful tips for both avoiding and addressing them.

While it has been advanced over many years, the process of developing TIL therapies still suffers from certain challenges. This article attempts to lay bare some of these challenges and discuss how novel technological solutions can help overcome them.

This white paper addresses the unique production problems of cell and gene therapy and explains how a manufacturing execution system (MES) can address them. It provides guidance on how to select an MES and a case study of two CAR-T therapy companies scaling up with and without an MES.

The FDA requires packaging and distribution systems to protect the agent from contamination and damage. In distributing cryogenically frozen advanced therapies, this includes protection from temperature shock.

Successful technology transfer is reliant on a few key variables including robust process characterization and communication and collaboration among multiple teams and sites. 

CAR-T therapies are expensive, and lentiviral vectors (LV) production represents a large portion of these costs. High manufacturing costs for LV are driven by low efficiencies from limited scalability in adherent cell culture, as well as requirements for large manufacturing spaces and several manual manipulations that increase labor costs. Following are practical tips and a case study to develop a lentivirus packaging cell line.

Understanding how to incorporate technologies and strategies that decrease the reliance on brick and mortar sites and bring the study to the patient is critical across all phases of drug development in today's challenging landscape.

A manufacturer faced a challenge to scale up, close, and optimize a suspension-based manufacturing workflow for pluripotent stem cells (PSCs). In response, CCRM developed an optimized 14-day manufacturing workflow, which produces >10¹⁰ cells, in < 12 months.

A clinical-stage biotechnology startup asked Veristat to run complex U.S. and EU trials of their unique gene therapy. Veristat brought invaluable global capabilities and expertise to a small client team tackling their first clinical-stage program without the large infrastructure or resources to conduct studies in this space themselves. Our end-to-end support started at Phase 1 and will continue into post-marketing and regulatory follow-up.

Life sciences industries require highly controlled environments for drug research development and manufacturing.

Are you ready to take your therapies from the lab into production and looking for ways to implement a closed system design with single-use technologies?

Process development is a critical enabler to bring safe, effective, sustainable products to market to address patient needs. In this webinar we will discuss our strategies for developing lentivirus and adeno associated virus (AAV) and the impact early decisions can have on commercial readiness.

With the exciting advances in research and drug development, the importance of sample management has never been higher. If you have ever experienced sample management mishaps, we ask: Do you know where your samples are? In this article, we'll explore some of the common causes of inventory mismanagement and best practices to guard against sample loss and near misses.

When a new product, technology, or supplier is selected during the development and design phase of the biological manufacturing process, it is essential for a manufacturer to take a detailed assessment not only of their proposed supplier, but also the supplier's own supply chain and even beyond.

Autologous (patient derived) therapies represent unique challenges to processing. One patient, single-batch is a radical change from the scale our industry is accustomed to. At this level, the filling is more analogous to limited Phase 1 clinical trial materials or even preclinical applications. The logistics and required compliance of manufacturing these personalized therapies is drastically different and presents a combinatorial explosive problem. One aspect of this process where traditional methods fall apart is the filling of these therapies.

Modular cleanroom systems are increasingly chosen over a conventional construction approach due to their accelerated design and installation schedule advantages, as well as their reduced jobsite safety risk during installation.

While adaptive design is associated with many potential benefits, it may also present challenges to observing the basic ethical principles of research in human subjects. In this white paper, we review the features of some particular clinical trial design adaptations and discuss the ethical obstacles they can present and those they can potentially resolve. Using examples of both published and unpublished clinical studies, we highlight the importance of proper design and planning and appropriate ethical due diligence in the successful conduct of an adaptive design clinical trial.

Cryopreservation provides critical protection for cell therapies by minimizing genetic changes. But cooling too slowly or too quickly risks diminishing cell viability upon thaw. Cytiva's Peter Kilbride (senior research scientist) and Julie Meneghel (cryobiologist) discuss the importance of controlled-rate cryopreservation. Illustrating how mammalian cells change when frozen, they offer concrete cryopreservation strategies and identified temperatures at which it is safe to stop controlled cooling and transfer drug product to cryogenic storage.

As drug developers face the pressing need to get molecules to market as efficiently as possible, firms large and small are increasingly turning to CDMOs for help.

Pharmacovigilance (PV) activities must be carefully planned from the outset of the drug development process. Read more about available process automation solutions available and if your company could benefit from working with external partners to manage PV activities.

There are several challenges related to the development and manufacture of adeno-associated virus (AAV)-based processes, including characterization, quantification, and downstream purification. LumaCyte's Radiance instrument, which uses laser force cytology (LFC) to measure the intrinsic biophysical and biochemical properties of single cells, has the potential to improve the characterization of cell-based AAV transfection and production, improving the efficiency and accuracy of both processes and shortening development time.

Given the significant potential of formalin-fixed paraffin-embedding (FFPE) tissues, this material must be collected, documented, and preserved properly in order to gain its full potential.

The journey from research lab to market is a long one, and most drug candidates do not make it. Following are some tips to assist with up-front planning to prevent headaches down the road.

COVID-19 has dramatically changed the way we conduct clinical trials and left many sponsors without answers when it comes to the future of their studies. This podcast explores challenges sponsors see as a result of this crisis, the impact those challenges are having on trial procedures, and how modifications to the statistical analysis plan can point a path forward.

A recent study by the Tufts Center for the Study of Drug Development (CSDD) compared cycle times and development economics between multi- and single-source CDMO models.

Reprogramming induced pluripotent stem cells (iPSCs) remains an open and manual process, carried out using conventional basic research techniques. To advance cell and gene therapy (CGT) and to get products derived from iPSCs to the clinic, generation and maintenance of cells for therapeutics in a good manufacturing practices (GMP) setting becomes paramount. So, how is this achieved?

How can end users ensure the expected level of compliance in this new world of single-use systems? The secret: shared responsibility for validation with your supply chain.

Recently approved cell and gene therapies are delivering impressive results for patients who otherwise have exhausted all treatment options or have had no options available to them. However, manufacturers are faced with many challenges in the journey from development to commercialization. Within this challenging environment, a number of risk mitigation strategies related to the materials used to produce viral vectors can be employed to help accelerate progress toward commercialization of these remarkable therapeutics.

Consistent management and communication are the keys to success when managing the supply chain of any advanced therapy clinical trial. In this article we'll review two case studies that demonstrate the importance of supply chain management by focusing on scaling up for commercial distribution and on packaging challenges within a cryogenic environment.