Cellular therapies are a therapeutic option for thousands of people with cancer, genetic, and degenerative diseases. Immense resources are being invested into this therapeutic area as hundreds of academic research groups, biotech companies, and top pharmaceutical companies are working on cell therapies in their development pipelines.

Cellular products can show therapeutic effects based on innate cellular capabilities, although many gene-modified cellular products are being developed to enhance, change, or add new functions to cells. The current most successful technology for cell engineering is chimeric antigen receptor (CAR) expression on the patients’ T lymphocytes. This artificial receptor enhances the capacity of T cells to detect tumor cells, kill them, and activate the whole immune system.

The CAR-T trials show impressive response rates and survival outcomes in relapsed and refractory patients with B-cell malignancies and multiple myeloma, leading to growing new approvals by FDA and the European Medicines Agency each day. Recently, clinical trials showed the benefit of CAR-T in second-line therapy for lymphoma. Soon, multiple myeloma patients at early stages of the disease will be enrolled for trials with CAR-T targeting B-cell maturation antigen.

There has been tremendous improvement in the safety profile of CAR-T therapies because of increased knowledge of early side effects and because of international consensus guidelines for clinical management. Real-world data and well-designed follow-up methodology show similar anti-tumor efficacy and survival outcomes in the long term and a good safety profile without new toxicities in the long term. The clinical and technological progress that represents CAR-T has been made possible by the close link among companies, regulatory agencies, patients, investigators, and sites.

Contract research organizations (CROs) have been the glue to coordinate such strategic partnerships, supporting all the stakeholders and equilibrating their genuine interests. Furthermore, the global resources of CROs have given access to patients and companies across different countries. Adaptability is a critical advantage to working with CROs and contract manufacturing organizations, as is having access to a multidisciplinary expert group of people and resources that provide options when selecting the specific services needed at any point throughout the cell therapy development pathway. Having experienced and expertise in conducting clinical trials and the desire to support state-of-the-art therapies can overcome the main challenges seen today in cell therapy product development.

Clinical sites expertise and trial design

The primary challenge in cellular therapy is building trust in patients, advocacy groups, and investigators. Biotech companies and enterprises must show solid preclinical data and strong support throughout the trial. The design of a protocol is also crucial. The protocol should fit patients’ reality and have safety, tolerability, and quality of life at the frontline.

Many characteristics of cellular therapy, such as supply chain, apheresis, and infusion, impact the recruitment of patients in clinical trials. The Foundation for the Accreditation of Cellular Therapy, the Joint Accreditation Committee of the International Society of Cellular Therapy, and the European Society for Blood and Marrow Transplantation have created standard quality guidelines for appropriate clinical management of patients involved in cell and gene therapy (CGT) trials or treated with approved therapies. These standards include a guide for donor evaluation, apheresis procedure, and handling of cellular products. The centers accredited by these committees show multidisciplinary experience teams and blood bank resources. The number of CGT trials opened exceeds the number of accredited centers, however. As these institutions are large academic reference centers, they sometimes lack resources due to health necessities, as has happened with the COVID pandemic. On the other hand, many candidates enrolled in CGT trials are treated and then have follow-up appointments in middle-sized community centers with limited experience, resources, and access to CGT trials. The partnership among different health resources will be critical in recruitment and accessibility in clinical trials.

Cellular therapies are also studied outside of oncology and hematology, such as neurology and cardiology, from regenerative medicine with pluripotent cells to the anti-inflammatory effects of mesenchymal stromal cells. The local delivery of the cells is sometimes needed to improve these products’ efficacy, which is challenging for a clinical trial. The product is not only a bag or vial with cells but a part of a complex translational platform. The investigator and site need specific training on delivery tools because this approach can cause related toxicity added to the cellular product itself. This issue should be considered when designing the product maximizing the option for easy delivery and fast learning requirements and thinking in the post-commercialization period.

The quality of the trial design in terms of monitoring the patients and establishing reliable data management is paramount in cellular therapy clinical trials. The lack of deep characterization tools and methods often does not reveal rare but critical safety concerns and can caused regulatory holds to clinical trials.The mandatory long-term follow-up in clinical trials with retroviral-based cell therapy is 15 years. Furthermore, long-term safety and efficacy assessment trials are mandatory after the product´s authorization to protect patients and collect data for further therapy development. The plan to maintain contact with patients and community sites is crucial. Decentralized trial methods preserve the quality of patient follow-up and data collection improving the quality of life of patients.

Cell therapy is an exciting therapeutic area, rapidly evolving with clinical and manufacturing challenges. CAR-Ts’ clinical success has driven companies to develop more effective, safe, and persistent cellular therapies using other cell types, such as natural killer (NK) cells and macrophages, to treat other cancers, including solid tumors. The current use of retroviral vectors to insert genetic information within cells can cause oncogenic genetic changes. The advent of genome editing technologies such as clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein-9 has enabled alternative strategies to improve genetically modified cells’ safety, efficacy, and manufacturing.

Autologous CAR-T implementation has demonstrated to the industry and the world how fast a transforming therapy can become part of the standard of care for the unmet needs of patients. It is time to design the path from small-scale treatment to large-scale allogeneic therapies that can impact a broader patient population. Allogeneic cell sources, “off-the-shelf” therapy, from healthy donors or pluripotent stem cells (PSC), are still in early phases but are open to patients, encouraging investigators, and simplifying operational challenges. The waiting time during the manufacturing process of three to four weeks is a severe limitation of autologous CAR-T cell therapy products that have currently been approved. Cells from induced PSC, in comparison, can be manufactured without limits from a single clonal cell bank, replicated, and expanded, resulting in a consistent and well-characterized product.

Some companies contract external manufacturing services at the beginning of development to have access to fast, adaptable technology until finding positive results in terms of feasibility and safety from early clinical trials. In the late stages of clinical development, the internally manufactured product may make more sense to control production and costs. Other CGT developers only get part of their starting material—cells or viral vectors—from specialized manufacturing laboratories. So, a “make vs. buy” approach can change through time, with the available data from clinical trials and the resources or partnerships that the company can develop through the clinical development path.

Decentralized manufacturing will be another approach to scalability, thanks to technology becoming increasingly portable and increasing experience in this field. There are also projects to develop CAR-Ts in vivo, for example, using viral vectors or nanoparticles, transfecting patients’ lymphocytes directly in the body, and avoiding the need to collect cells and manipulate them in the laboratory.

The manufacturing complexity of cellular therapies is unique, mainly in cell therapies containing viral vectors. Companies need to demonstrate a strong understanding of their product and the target population and have a purposeful plan to analyze therapy performance through clinical studies. The regulatory agencies are very active in developing new guidelines about the quality of cellular products.An expert clinical, chemistry, manufacturing, and controls, and regulatory consultant is likely to benefit patients and cellular products, easing the translation of the product to clinical application with proper safety testing and traceability documentation.

The challenge is to standardize assays on individual products, and companies are often forced to identify the most fit-for-purpose methodology and present it to regulators for approval. The possibility of designing a standard manufacturing approach could accelerate cell therapy manufacturing, ensure homogeneity and quality, and make the regulatory pathway easier.

Society is impressed with what science and human tenacity can achieve. Cell products are a considerable change in the therapeutic paradigm and require a new insight into the research structure and the relationship between stakeholders. The human resources, infrastructure, and investment needed to bring these therapies to market must be coordinated so that the knowledge and capabilities of pharma companies, CROs, investigators, and patients are all brought to bear to address challenges in development. The goal should be to conduct more adaptative clinical trials, resist uncertainty, and create efficient strategies for fast scale-up to make cell therapy accessible as the standard of care. The regulatory agencies also support recommendations on collecting patient-reported outcomes, tailoring operational plans, and manufacturing high-quality products. Expectations for these therapeutics are high, and it is time to make cell therapies a real option for patients.

A new approach is critical for patients with malignant and chronic diseases looking for a curative and safer therapies that can offer a better quality of life.

Izaskun Elorza, MD, is the senior medical director for cell and gene therapy at Parexel.

Articles

Features

The clinical trial research environment has evolved because of specific solutions designed to overcome uncertainty.

The Evolution of Cellular Therapies in a New Clinical Research Environment

Animal models are widely used in the preclinical testing phase of new active ingredients. Novel approaches for the design and development of 

 models has become more relevant to predicting drug safety in patients. However, to optimize an animal model’s ability to predict a corresponding human situation, the models need to be clearly defined, controllable, and as “humanized” as possible (1). Strategies such as using novel technologies—for instance, small interfering RNA for mediating gene knock-down—and systems that enable tight and reliable control of human (trans) gene expression in a time- and tissue-specific manner, are approaches that can optimize animal model prediction (1).

Furthermore, there have been new approaches to designing and developing safety assessments using human 

 systems have become increasingly important for both biopharmaceuticals and pharmaceuticals. These tests likely combine human stem cell cultures, or co-cultures, with animal disease models (1).

To further explore the state of animal model approaches in preclinical testing for bio/pharmaceuticals, 

 spoke with Andrew Reaume, president and CEO of Melior Discovery, a US-based provider of 

 What is the importance of animal models in preclinical studies for bio/pharmaceuticals?

 Animal models are generally regarded as providing the highest quality of preclinical proof-of-concept towards efficacy.For most therapeutic indications, positive activity in an animal model is regarded to be a necessity, prior to entering the clinic.

Given the tremendous cost and potential risk towards human subjects, animal models provide not only the confidence that a candidate therapeutic will have efficacy towards a given indication, but even help guide clinical trial design by providing answers to questions such as:What patient population should be initially targeted? What dose regiment will provide optimal therapeutic effect? What primary endpoint is most likely to illustrate therapeutic effectiveness? And so on. These are all examples of questions that may not be answered on a theoretical or mechanistic basis and in which activity in an animal model may provide insights.

What are the main differences between using 

 models, especially as pertains to the type of data generated?

 Animal models, as opposed to reductionist 

 models, represent a fully intact biological system with all of the complexities inherent in evaluating test articles in human subjects. For example, in psychotherapeutic active indications, such as schizophrenia, depression, and attention deficit disorder, 

 models simply do not provide a modeling of the complex neuronal connections and associated pathologies of their disorders needed for any kind of efficacy evaluation.

Even for disease states that are more readily evaluated with 

 systems, such as the anti-proliferative effects on human cells in culture for an oncology candidate, 

 systems do not provide a holistic picture of test article effects that factor in pharmacokinetic/pharmacodynamic relationships of a drug, effects of the drug on a target cell population in the context of surrounding tissue effects, and toxicity effects, among other important factors.

 animal models, and how are the data applied to the development program of a new drug candidate (specifically a large-molecule drug)?

 Cell culture models can be used to reveal target engagement—that the test article is modulating its intended target in the way that it was intended to.

Animal models provide a bigger picture view of test article effect—that the test article is not only modulating the intended target, but that this in turn is having the downstream therapeutic consequences that are intended. Moreover, they provide a view of the potential therapeutic benefit in the context of a safety and tolerability profile (i.e., are the therapeutic effects provided without undo toxicity effects in other organ systems).

 What are some of the latest innovations (technology, methodology, approach) in animal model studies, specifically applied to large-molecule preclinical studies?

 Advances have been made recently in the area of immune-oncology, which also represent some of the latest innovation in animal model studies. Animal models involving patient-derived xenografts (PDX) and immune modulatory therapeutics, such as chimeric antigen receptor T cell (CAR-T) and programmed cell death ligand 1 (PDL-1) are examples of the types of studies which we are able to only now routinely approach but were almost unheard of 10 years ago.

Many humanized genetic mice models have been developed to evaluate immunotherapy in PDX models.For example, humanized CD34+ mice (hu-CD34) are a robust 

 platform for analyzing the safety and effectiveness of potential new drugs to modulate specifically the human immune system. Also, models engrafted with human cord blood-derived hematopoietic stem cells develop multi-lineage engraftment and display robust human T-cell maturation and human T-cell dependent inflammatory responses. In addition, an improved human myeloid and natural killer lineage development is demonstrated in humanized NSG-SGM3 and NSG-Tg(IL15) mice.

 Are virtual models, either of animals or of human tissues/organs, comparable to 

 animal studies? In other words, could virtual models be an alternative for animal models?

 It is our firmly held belief that, given the tremendous complexity of biology, there is far more that we do not know about living systems than what we do know, in spite of our great advances in understanding the physiological and biochemical underpinnings of many disease states. As such, we will not be able to model disease adequately with artificial intelligence (AI) in the foreseeable future. For example, the challenge of computationally predicting whether a drug will be effective in a given disease far, far, exceeds that of having a computer autonomously and safely drive an automobile through the real-world environment.Therefore, animal models will remain a core tool necessary for advancing therapeutics into the clinic for many years to come.

 Are there other alternatives to animal models that would generate meaningful preclinical study data for a new drug development program (large molecule or small molecule), and if so, what are these alternatives?

 Not really. For the reasons stated earlier, computational approaches such as AI are not yet able to model the immense complexity of intact biological organisms. While AI approaches are used, the predictive quality towards efficacy of a test article almost invariably will not match that of an 

In some instances where a terminal disease involves antibodies to human-specific antigens (i.e., the therapeutic test article does not cross-react with a target in animals), there may not be a practical way to evaluate the test article in an animal model.In these instances, given the life-and-death nature of a disease in which no alternative therapeutic candidate is available, test articles may bypass the use of animal models.

1. J.T. Koppele and R. Witkamp, “Use of Animal Models of Disease in the Preclinical Safety Evaluation of Biopharmaceuticals,” in 

Preclinical Safety Evaluation of Biopharmaceuticals: a Science-Based Approach to Facilitating Clinical Tria

ls, J.A. Cavagnaro, Ed. (John Wiley & Sons, Hoboken, New Jersey, 2008), pp. 293–308.

Feliza Mirasol is the science editor for 

Preclinical testing is better able to evaluate complex drug candidates thanks to innovations in animal model approaches.

Animal Models Are Seeing Upgrades to Tackle Complex Molecule Testing

Carterra, a provider of technologies enabling high-throughput biology, and the La Jolla Institute for Immunology (LJI), a non-profit entity focused on immunology studies, announced that LSJ acquired Cartera’s LSA antibody discovery and characterization platform on Aug. 1, 2022. LJI first worked with Carterra and the LSA platform on the Coronavirus Immunotherapy Consortium (CoVIC), a project that attempted to find the most potent antibodies against COVID-19.

The LSA platform is designed to enable rapid characterization of hundreds of antibodies in the early stages, then provide in-depth analysis of leads during later stages of discovery. According to a company press release, relative to traditional methods, the LSA generates results 100 times faster while using 99% less sample. In addition to autoimmune and cancer targets, it is also designed to enable rapid characterization of sera samples from vaccine studies.

“The LSA was key to mapping the epitope landscape of the SARS-CoV2 spike, using the 400 antibodies in the consortium. That information was instrumental to understanding which target sites are still effective against emerging variants of concern,” said Erica Ollmann Saphire, professor at La Jolla Institute for Immunology, director of the CoVIC consortium, in the release. “Having seen what the LSA can do first-hand through our collaboration with Carterra as part of the CoVIC, we now look forward to using this instrument to propel our own discovery efforts.”

“We’re proud to see the benefit of Carterra’s LSA platform being brought to bear for global good,” says Tim Germann, chief commercial officer, Carterra, in the release. “Working with Erica and her team at LJI has been truly enlightening—they understand the importance of breaking down the old paradigm of slow and inefficient biologics discovery. Bringing the LSA to LJI represents their commitment to leading the effort to end the suffering from pandemics like Ebola and COVID-19.”

News

La Jolla’s acquisition of Carterra’s LSA Platform is designed to advance their immunological and infectious disease research.

La Jolla Institute for Immunology Acquires Carterra LSA Platform

Synthego, a US-based genome engineering company, announced on Aug. 2, 2022 that it has launched engineered cell libraries that further enable access to clustered regularly interspaced short palindromic repeats (CRISPR). These libraries, based on Synthego’s Eclipse Platform, provide arrayed CRISPR-edited cells that can be directly used in functional screening assays.

The new offering is a high-throughput cell engineering platform that delivers cell-based models for disease research. The platform provides highly predictable CRISPR-engineered cells at scale via the integration of engineering, bioinformatics, and proprietary science and aims to enhance the speed, scalability, and efficiency of the drug discovery process by optimizing the path between experimental design and execution.

This platform, the newest addition to Synthego's engineered cells product line, offers a custom-arrayed library of multi-guide knockout cell pools (immortalized or induced pluripotent stem cells [iPSC]). These libraries are delivered ready-made to researchers and enable them to bypass the need for equipment and reagent sourcing, transfection, generation of an extensive CRISPR library, and hiring and training of staff. This advantage, in turn, allows researchers to overcome common hurdles associated with CRISPR-based target discovery and instead focus specifically on the science, according to Synthego in a company press release.

With engineered cell libraries, scientists can specify the human or mouse cell type (immortalized or iPSC available at launch) and gene targets they want to knockout to generate a custom “Knockout Cell Library.” The cells are then edited on Synthego’s Eclipse Platform, which handles guide design, cell line optimization, editing through transfection, and assessment of editing efficiencies. This results in cells that are ready to screen upon arrival.

Benefits of the engineered cell libraries on the Eclipse Platform include:

Scalability and flexibility—engineered cell libraries allow researchers to maximize their screening power, which allows them to identify more targets earlier in the screening process.

Predictability and transparency—Synthego’s multi-guide technology can achieve reliably high knockout efficiencies, which offers researchers confidence in their screening process.

Cell engineering expertise and support—Synthego offers its expertise in automation innovation to provide greater consistency in outcomes and scalability.

Both the Eclipse Platform and the engineered cell libraries enable a wide range of applications in research and development across various disease areas and research disciplines, including oncology and neurology.

“Arrayed screening approaches are more sensitive, compatible with a broader assortment of downstream assays, and can yield data that is more readily interpretable than the more commonly used pooled approach but are underutilized due to lack of infrastructure and limited bandwidth,” said Travis Maures, chief technology officer, Synthego, in the release.

“We were able to quickly combine our proteomic expertise with Synthego's genome engineering capabilities in a matter of weeks,” said Nevan J. Krogan, director, Quantitative Biosciences Institute, University of California, San Francisco, in the release. “Normally, work such as this would take many years. We were able to quickly pinpoint which human genes are important for infection, and that allowed us to jump to which ones if we were able to drug them, could have a positive pharmacological effect on SARS-CoV-2 infection. That whole pipeline allowed us to identify several potential drug candidates, several of which we're still looking at.”

New Technology

Synthego has launched new engineered cell libraries to validate targets for quicker drug discovery timelines.

Synthego Aims to Accelerate Drug Discovery with New Engineered Cell Libraries

Bio-Rad Laboratories recently expanded its portfolio of recombinant monoclonal anti-idiotypic antibodies to include antibodies that are specific to Adcetris (brentuximab vedotin), Cosentyx (secukinumab), and the secukinumab-interleukin 17A (IL-17A) drug-target complex.

Brentuximab vedotin is an antibody drug conjugate used to treat Hodgkin lymphoma, while secukinumab is used to inhibit inflammatory response in several diseases, such as ankylosing spondylitis, psoriasis, and psoriatic arthritis.

According to a company press release from July 12, 2022, these antibodies can be used in the bioanalysis and drug monitoring of brentuximab vedotin, secukinumab, and their biosimilars.

The antibodies for antibrentuximab vedotin and antisecukinumab are approved for 

 testing services to support drug and biosimilar development, and patient monitoring.

Bio-Rad has expanded its range of recombinant monoclonal anti-idiotypic antibodies.

Bio-Rad Expands Portfolio of Anti-Idiotypic Antibodies

Shimadzu launched its new Nexera XS inert, a bioinert and biocompatible ultra-high performance liquid chromatograph (UHPLC), in July 2022. The system is designed to resolve the most common problems in biopharmaceutical analysis, such as the adsorption of target molecules to metal surfaces and mobile-phase corrosion caused by high salt content and extreme pH conditions.

The Nexera XS inert UHPLC resulted from cooperative research done with Yasushi Ishihama, a professor at the Graduate School of Pharmaceutical Sciences at Kyoto University in Japan under the Japan Science and Technology Agency’s Adaptable and Seamless Technology Transfer Program through Target-driven R&D (A-STEP) project from 2017 to 2020. The system features a metal-free sample flow path, which prevents unwanted interaction with biopolymers that are prone to adsorption onto exposed metal sites. Through this mechanism, the Nexera XS inert results in reduced sample loss and excellent peak shape for generating reproducible, high-sensitivity and high-quality reliable data, according to a July 19, 2022 company press release.

Furthermore, all wetted surfaces have been prepared from corrosion-resistant materials, which renders them stable against mobile-phase solvents that contain high concentrations of salts or acids. Nexera XS inert is particularly well-suited for analyzing biopharmaceutical molecules, such as antibodies, peptides, and nucleic acid drugs.

The new system offers the same analytical intelligence features as the other products in the Nexera series as well as the following features:

Unconstrained recovery and sensitivity: in the quantitative analysis of biomolecules, adsorption of the target compound to a metal surface can result in a dramatic decrease in sensitivity. The Nexera XS inert system comprises stainless-steel-casing-reinforced polyether ether ketone (PEEK) tubing, which is a high-performance engineered polymer with mechanical- and chemical-resistance properties. This ensures a system pressure tolerance of > 100 MPa while maintaining a bioinert flow path. The system also includes a ceramic injection needle, which offers metal-free injections and minimized carryover. By preventing adsorption to metal surfaces, the Nexera XS inert system thus achieves much higher sensitivity and better reproducibility than a general-purpose UHPLC system.

Assured reliability and reproducibility: high salt conditions in the mobile phase can result in the corrosion of stainless-steel parts in standard UHPLC systems, but, in the Nexera XS inert system, the wetted surfaces are prepared from highly rust-resistant materials, while still maintaining the high-efficiency flow-path (i.e., 105 MPa). Because of this design, the system remains unaffected by high salt content, ion pairing agents, or extreme pH conditions, ensuring long-term stability and reliable data acquisition.

Clear resolution without restrictions: the system’s ability to resist 105 MPa of pressure means it can use long separation columns packed with small-sized particles, which allows it to achieve ultra-high efficiency in a wide variety of applications. The newly developed PEEK-lined tubing comes with tool-free, finger-tightened fittings, allowing for reliable connections between columns, which enables the system to withstand a pressure of up to 105 MPa without creating any dead volume or risking damage by overtightening. All valves used in the Nexera XS inert system have been designed to inhibit metal adsorption for worry-free switching between multiple columns. This characteristic is useful for method development or for trap-and-elute analysis. An optional pHM-40 offers real-time measurement of the mobile-phase pH during the course of an experiment. This ensures accuracy and traceability, even when running a pH gradient, as used in ion exchange chromatography.

Consumables ideal for analyzing biopharmaceuticals: analyzing biomolecules, such as proteins, peptides and nucleic acids, requires columns with different separation modes. For some samples that are sensitive to metal ions, metal adsorption must be taken into consideration, and an optimal column would address this issue. The columns used in this new system (Shim-pack series of columns) is fitting for the analysis of a wide variety of biomolecules. In addition, adsorption of target analytes in the sample vial can greatly affect the analysis and requires special attention. Furthermore, a low-adsorption polypropylene vial (TORAST-H Bio Vial, Shimadzu) is also most fitting for peptide drug research, while low-adsorption glass vials (TORAST-H, Shimadzu) are suitable for general-purpose analysis.

Shimadzu’s new ultra-high performance liquid chromatography system is designed to solve common problems in biopharma analysis.

Shimadzu’s Nexera XS Inert Chromatography System Enhances Biopharma Analysis

Roche launched its Elecsys HCV Duo immunoassay (Elecsys) in July 2022, which is the first available immunoassay that allows for the simultaneous, independent determination of the hepatitis C virus (HCV) antigen and antibody status from a single human or serum sample, according to the company in a July 18, 2022 press release. This assay allows for the detection of the virus in the early stages of infection, or when a patient is recovering from the virus, or when a patient is showing signs of a chronic infection.

Elecsys uses dual detection of HCV core antigen and antibodies. According to the release, this allows it to enable significantly earlier diagnosis of active infection relative to antibody-only assays, as core antigen appears early in an infection and is a marker of ongoing viral replication. Earlier identification can lead to earlier intervention, less burden on the healthcare system, and better outcomes for patients.

“With improved hepatitis screening, healthcare systems have the opportunity to eliminate the disease through improved prevention, testing, and treatment services,'' said Thomas Schinecker, CEO, Roche Diagnostics, in the press release. “The addition of the Elecsys HCV Duo assay to our HCV testing portfolio can help in the fight to eliminate the hepatitis C virus. The launch of this innovative dual antigen and antibody diagnostic test underlines our commitment to support clinicians and their patients in reducing the impact of infectious diseases, where it’s needed most.”

According to the release, 58 million people were living with chronic HCV infection in 2019, but only 21% were aware of it. Additionally, 1.29 million people died of hepatitis C-related causes, such as cirrhosis or liver cancer. No vaccine is currently available for HCV, but effective treatment with antivirals can lead to a cure in 98% of patients.

Roche’s new test is the first available immunoassay that allows for simultaneous, independent determination of the hepatitis C virus antigen and antibody status.

Roche Launches Dual Antigen and Antibody Hepatitis C Diagnostic Test

Discovery Life Sciences (Discovery), a company specializing in biomarkers and biospecimens, announced its acquisition of AllCells, a provider of human primary blood and marrow-based cells. Discovery plans to combine its existing cell and gene therapy products and services with AllCells to create a business unit called AllCells, a Discovery Life Sciences company. AllCells CEO and President Danny Zheng will lead the entity.

“We are very excited to welcome the AllCells team to the Discovery family,” said Glenn Bilawsky, CEO, Discovery Life Sciences, in a July 12, 2022 company press release. “Bringing these two great companies together creates a scalable, end-to-end solution that gives our clients unprecedented access to reliable human cellular starting materials with integrated multi-omic analytic services to support cell and gene therapies.”

According to the press release, the acquisition grants Discovery access not only to their cell supplies, but also starting materials and analytic services to accelerate cell and gene therapy experiences. Additionally, Discovery’s newly acquired FDA-registered donor collection facilities and good manufacturing practice (GMP)-compliant cleanrooms are adjacent to characterization labs; this placement enables immediate processing and cryopreservation.

“We have built our organization to be an extension of our client's supply chain. By offering donor program consulting, high-quality apheresis collections, customization services, and GMP-compliant starting materials, our dedicated team will continue to provide a high-touch, client-focused approach to ensure our clients' needs are met with superior products and services to fit any timeline and on any scale,” said Zheng in the press release. “Our strategy parallels and reinforces Discovery's 'Science at your Service' approach, and our unified offerings will further accelerate our clients' strategic and vital cell and gene therapy programs.”

Discovery Life Sciences’ acquisition of AllCells is intended to bolster its cell and gene therapy products and services.

Discovery Life Sciences Acquires AllCells

PerkinElmer announced the addition of the PerkinElmer Flow Cytometry Instrument Concierge Services to its OneSource Laboratory Services portfolio on July 11, 2022. The concierge service is designed to provide biopharmaceutical labs with expert, on-site flow cytometry specialists to help manage instruments and processes, facilitating reduced downtime, increased productivity, and better data integrity and reproducibility.

According to a company press release, the service includes regulatory quality checks on cytometers, instrument cleanings and preventative maintenance, on-site troubleshooting, phone support, and initial repairs. Additional features include:

Instrument and software training and education

“Flow cytometry is an important part of drug discovery labs, but managing this complex technology in-house can be tedious and time consuming, especially for teams that lack expertise in this area or are experiencing high staff turnover,” said Gary Grecsek, vice president/general manager of OneSource Enterprise Laboratory Services, PerkinElmer,in the press release.“With our OneSource Concierge Service for flow cytometry, lab managers and scientists don’t have to spend time managing these instruments and processes and can focus on other critical efforts and discovering new drug candidates.”

The PerkinElmer Flow Cytometry Instrument Concierge Services will provide biopharmaceutical labs with expert, on-site flow cytometry specialists.

PerkinElmer Introduces New Cytometry Management Service

Merck, known as MSD outside the United States and Canada, launched its Merck Digital Sciences Studio (MDSS) in June 2022, which is aimed at enabling the generation of innovative technologies for drug discovery and development.

The objective of MDSS is to support early-stage biomedical startups with direct investment, access to Azure Cloud computing, and opportunities to pilot their respective technologies in collaboration with discovery and clinical scientists at Merck. Merck opened applications on its website for 12 spots for the first MDSS cohort, and applications for startups developing artificial intelligence (AI) and machine learning (ML) will be prioritized, according to a June 29, 2022 company press release. The program currently plans to include six startups at each of MDSS’ Newark, NJ, and Cambridge, Mass., venues.

The MDSS is a collaboration between Merck and the New Jersey Innovation Institute (NJII), a New Jersey Institute of Technology corporation. Investments have come from the Merck Global Health Innovation Fund, Northpond Ventures, and McKesson Ventures. In addition, Microsoft for Startups will provide technology support, including Microsoft 365 and Azure, as well as provide expert business advice and technical support.

Digital technologies have enabled the innovation of biomarkers for use in drug discovery and development, specifically in the areas of target identification, lead discovery, preclinical development, and clinical development. MDSS will allow collaborators to selectively accelerate and pilot novel and innovative digital technologies that strategically align with Merck’s life science research.

“The biopharmaceutical industry has only just started to harness the tremendous opportunity presented by emerging technological developments in AI and ML,” said Fiona Marshall, senior vice-president, Discovery, Preclinical, and Translational Medicine, Merck Research Laboratories, in the company press release. “The MDSS will provide a collaborative and entrepreneurial setting where scientists have access to the tools and expertise to fuel important innovations that advance drug discovery and development.”

“Startups in the biopharmaceutical industry fuel innovation, create high-quality jobs and generate great opportunities for a stronger, more diverse and inclusive life-sciences ecosystem,” said Simon Nynens, CEO, NJII, in the release. “NJII looks forward to providing the programming, coaching, and connections to bring these ideas to life.”

Merck, known as MSD outside the United States and Canada, now offers the Merck Digital Sciences Studio, designed to enable innovative tech for drug discovery and development.

Merck Digital Sciences Studio Supports Early-Stage Startups

Touchlight, a biotechnology company specializing in enzymatic DNA production, entered into a non-exclusive patent license agreement with Pfizer on July 6, 2022. Under the agreement, Pfizer gains rights to Touchlight’s enzymatic doggybone DNA (dbDNA) patent portfolio. Pfizer will have rights to use dbDNA worldwide for the manufacture and commercialization of its messenger RNA (mRNA)-based vaccines, therapeutics, and gene therapies.

Through the agreement, Touchlight will receive an upfront payment as well as clinical and commercial milestone payments and royalties upon commercialization. The financial terms of the deal were not disclosed.

Touchlight’s dbDNA technology has been shown to produce a minimal, linear, double stranded, covalently closed DNA vector via an enzymatic manufacturing process. dbDNA enables the rapid, synthetic manufacture of good manufacturing practice (GMP)-compliant DNA product from a small, simple footprint. Furthermore, it is a scalable GMP manufacturing process.

The technology can manufacture genes >20 kb. It also has the added advantage of being able to accommodate sequences typically unstable as plasmid DNA in 

; these include sequences found in viral vector and mRNA production. Beyond mRNA vaccine production, the technology is suited for the development of DNA vaccines, advanced therapies, and other therapeutics, according to a company press release.

“We are delighted to establish this agreement with Pfizer to license our mRNA manufacturing platform. This agreement is an example of our technology’s potential to enable companies across the genetic medicine sector to simplify and accelerate DNA manufacturing through the clinic and towards commercialization,” said Jonny Ohlson, founder and executive chair, Touchlight, in the press release.

Pfizer and Touchlight have signed a patent license agreement for Pfizer to use Touchlight’s doggybone DNA (dbDNA) in the manufacture of mRNA vaccines, therapeutics, and gene therapies.

BioPharm International's BP Elements, August 2022