A view on

We are back! Discover the latest in pharmaceutical innovation from CPHI Milan with A View On! This episode explores advancements in bioconjugates, AI-driven drug development, targeted capsule delivery, and new solutions in cell and gene therapy that are transforming patient care. Tune in to hear from Lonza experts on the future of pharma and biotech.

Tracing the Evolution and Future of Capsule Manufacturing   In this episode, we are joined by Ljiljana Palangetic, Associate Director of Hard Capsules R&D, and Bram Baert, Senior Director of Regulatory Affairs from Lonza, to delve into the intricacies of capsule manufacturing and the evolution of drug delivery technologies.  Your grandmother might have told you to “just swallow your medicine,” suggesting that you may have to endure something unpleasant but necessary. Today, however, this old saying might not ring true, as capsules have become ubiquitous in modern medicine. Favored by 44% of consumers, capsules simplify medication intake with their ease of swallowing and ability to mask unpleasant tastes. From ancient Egyptian leather pouches to modern high-tech production lines, capsules have undergone significant transformations, seamlessly integrating into our daily lives. Swallowing one's medicine has never been so easy.   Yet the role of capsules has expanded far beyond taste masking. Today, they are engineered to deliver drugs to specific parts of the intestine, dissolve at controlled rates, and even contain multiple medications in one unit. This adaptability not only improves patient compliance but also caters to a myriad of medical needs. As we look toward the future, the potential for capsules in drug delivery is boundless, driven by continuous innovation and a deep understanding of materials science.  As mentioned in the podcast, if you haven't already listened to episode 9 of this season, you can find out more about targeted drug delivery using capsules here.    Curious to Know More?  Join us in this conversation hosted by Martina Hestericova with Lonza's Ljiljana Palangetic and Bram Baert as they unveil the advancements in capsule manufacturing technologies and their impact on modern drug delivery systems.    KEY TERMS IN CONTEXT:  Regulatory Affairs are crucial for ensuring that all pharmaceutical products, including capsules, adhere to legal and regulatory standards. Professionals in regulatory affairs navigate the complex landscape of pharmaceutical manufacturing, particularly focusing on consumer and patient safety, by collaborating with health authorities to establish and update regulations that ensure the safety and efficacy of capsules.  Two-piece Capsules consist of a cap and a body that fit together, making them a versatile choice for different types of medication delivery. The design innovations of two-piece capsules have evolved significantly since their inception in the mid-19th century. They accommodate a multitude of materials such as powders or granules and playing a crucial role in modern automated manufacturing processes.  Designed to pass through the stomach intact and dissolve in the intestines, enteric capsules are crucial for drugs that can be deactivated by stomach acid or may cause irritation to the stomach lining. This technology ensures that medication is released in the part of the gastrointestinal tract where its absorption is optimized, thereby enhancing both the drug's effectiveness and patient comfort.  The use of polymer solutions is integral to forming the shells of capsules, particularly in technologies where a capsule is dipped into the solution, allowing the polymer to dry and harden. The choice of polymer affects the capsule's dissolution rate and stability, which is critical for ensuring that the drug is released at the correct rate and location in the body.  Made from thin membranes derived from the small intestines of sheep, SAPARIS capsules are an early form of specialized drug delivery technology. They were designed to allow for a slow dissolution rate, aiming to improve the timing of drug release within the body. This technology showcases the evolution of capsule materials from organic origins to today's synthetic and semi-synthetic materials used in capsule manufacturing. 

Simulating the Journey of Oral Medications: A Leap Towards Personalized Medicine  In this episode, we are joined by Deanna Mudie, a senior principal engineer at Lonza, and John DiBella, president of PBPK & Cheminformatics at Simulations Plus, to discuss new techniques in enhancing the bioavailability of drugs.  When you swallow a pill, have you ever pondered the intricate journey it undertakes to deliver its therapeutic effect? This voyage, crucial for the drug's effectiveness, is at the heart of pharmaceutical R&D's quest to enhance bioavailability - the proportion of the drug that enters circulation and reaches the target area.  By simulating how drugs interact with the body, scientists can optimize therapeutic outcomes by tailoring medications to the needs of individual patients. This approach promises a future where drugs are not only more effective but also safer, with reduced side effects. Listen as we delve into the cutting-edge world of Physiologically Based Pharmacokinetic (PBPK) modeling. These computer models integrate factors like gastrointestinal physiology and population characteristics, shedding light on how drugs behave in various body systems without the need for extensive patient testing.   Curious to Know More?  Join us in this conversation hosted by Martina Hestericová with Lonza's Deanna Mudie and Simulations Plus's John DiBella as they unveil the potential of PBPK modeling to revolutionize drug development and personalized medicine.     KEY TERMS IN CONTEXT:  In the context of pharmaceuticals, drug bioavailability refers to the proportion of a drug that enters the circulation when introduced into the body and is thereby able to have an active effect. It's a critical factor in determining the drug's effectiveness, as it measures how much of a drug in a dosage form (like a tablet or injection) becomes available at the target site of action.  PBPK modeling is a sophisticated computational modeling technique used to predict the absorption, distribution, metabolism, and excretion (ADME) of drugs within animals and humans. This approach aids in understanding a drug's bioavailability and supports the design of more effective and safer drug therapies.  Gastrointestinal Physiology refers to the study of the functions and processes of the digestive system or gastrointestinal (GI) tract. In the context of PBPK modeling, understanding gastrointestinal physiology is crucial for predicting how a drug is absorbed into the body, especially for orally administered medications. It includes factors like stomach acid levels, GI transit time, and the surface area available for absorption.  "In silico" refers to the use of computer simulations or digital analyses to conduct experiments or procedures virtually rather than in a laboratory or real-world setting. In silico tools in drug development include software and algorithms used for modeling and simulation, such as PBPK models, which allow researchers to predict how drugs interact with animals and humans, aiding in drug design, testing, and the customization of therapies for personalized medicine. 

Capsules for Targeted Therapy: A Game-Changer in Modern Medicine  In this episode we are joined by Vincent Jannin, Lonza's R&D Director, to explore Enprotect, the Award-Nominated Capsule Technology.  Imagine starting your day with a simple capsule that goes beyond simply dissolving in your stomach to reach the place in your body where it is needed most before releasing its medicine. That's just what Lonza's Enprotect enteric capsules do. They are designed to release medication directly into the small intestine, which represents a significant leap in pharmaceutical delivery. They improve patient compliance without increasing production costs and offer targeted delivery for specific therapies such as live biotherapeutic products. This targeted approach is crucial for treatments that require local delivery, for example for Crohn's disease, exocrine pancreatic insufficiency, or Clostridium difficile infection.    In this episode we hear from Vincent Jannin about how advances in polymer science have ushered in this new era of capsules capable of targeted drug delivery. This marvel of modern medicine combines the fields of chemistry, nanoscience, biology, and physics. The creation of a bilayer capsule—comprised of a structural layer for shape and a functional layer for targeted release—both required the development of new technologies and could itself serve as an enabling technology for future therapies.    Vincent Jannin and his team have published several peer-reviewed studies in open access scientific journals, which were mentioned in the podcast:  In Vivo Evaluation of a Gastro-Resistant Enprotect Capsule under Postprandial Conditions (https://www.mdpi.com/1999-4923/15/11/2576)  In Vivo Evaluation of a Gastro-Resistant HPMC-Based “Next Generation Enteric” Capsule (https://www.mdpi.com/1999-4923/14/10/1999)  In vitro evaluation of the gastrointestinal delivery of acid-sensitive pancrelipase in a next generation enteric capsule using an exocrine pancreatic insufficiency disease model (https://www.sciencedirect.com/science/article/pii/S0378517322009966)  Curious to Know More?  Join us this episode as we explore the journey from a simple capsule to a sophisticated drug delivery system and how this advancement reflects a remarkable fusion of science and innovation. Discover how the Enprotect technology not only offers hope for more effective treatments but also exemplifies the relentless pursuit of medical advancement for the benefit of patients everywhere.     KEY TERMS IN CONTEXT:  An enteric capsule is a type of capsule specifically designed to bypass the acidic environment of the stomach and release its contents into the small intestine. The term 'enteric' relates to the small intestine. These capsules are formulated to remain intact in the stomach and dissolve only when they reach the more neutral pH levels of the intestine, ensuring targeted drug delivery.  Enteric polymers are materials used in the construction of enteric capsules. They are chosen for their ability to withstand acidic conditions (like those in the stomach) and dissolve at higher pH levels like those found in the small intestine. HPMC Acetate Succinate is an example of an enteric polymer used for the outer layer of the capsule to ensure the treatment's proper dissolution and release in the intestine.  Live Biotherapeutics (LBPs) refer to live microorganisms used for therapeutic purposes. They are designed to interact with the human microbiome, particularly in the small intestine, and are sensitive to stomach environments. The protection LBPs need before their release in the desired intestinal location is facilitated by specialized capsules.  Fecal Material Transfer refers to a medical treatment involving the transfer of fecal matter from a healthy donor to a patient, often used for conditions like Clostridium difficile infections. The podcast highlighted the potential use of enteric capsules for the delivery of such treatments directly to the small intestine, thereby offering an alternative to more invasive procedures. 

Embark on a microscopic journey into particle identification — the unsung hero of pharmaceutical safety — and uncover how this vital process shields us from unseen threats in every single medication we take.

Season three so far, we've covered a lot!   In this summary episode, A View On host Martina Hestericova shares a secret and goes over a selection of the best ideas from season three so far.     The production team for A View On has managed to keep things moving along smoothly, so you most likely didn't even notice that Martina was away for four months. She's back! Listen in as Martina navigates us through a curated selection of the finest moments from the season: from cell culture to highly potent molecules, from inhalants to gene therapies, we've covered a lot this season.   Check out this summary episode and stay tuned for future topics such as antibody based therapies, health ingredients, and even forensic science. 

"A View On: CAR-T Cell Therapies" featuring Tamara Laskowski, Senior Director of Clinical Development in Personalized Medicine at Lonza, and Aya Jakobovits, CEO of Adicet Bio. Dive into the world of CAR-T cells, their therapeutic potential, and the future of cancer treatments.

Lonza experts Selene Araya and Charles Johnson discuss the manufacturing process, trends, and future of highly potent compounds (HPAPIs). They highlight Lonza's facilities and expertise in producing HPAPIs across various technologies and scales, ensuring advanced containment and addressing bioavailability challenges.

Ian Thomson from Ypsomed and Roman Mathias from Lonza discuss the market trends in injectable delivery devices, their manufacturing process, and their future in sustainable pharma. Injectable devices offer various benefits, including improved convenience, accuracy, and safety, allowing for precise dosing while reducing the need for hospital visits.     

Scaling Up Cell and Gene Therapies: Automation Is the Next Step   In this episode, we take a deep dive into manufacturing cell and gene therapies with Lonza expert Behnam Ahmadian Baghbaderani, executive director of Cell and Gene Therapy Process Development.   Cell and gene therapies have the potential to revolutionize the treatment of rare genetic diseases, cancer, and neurodegenerative disorders. These therapies involve extracting cells or genetic material from a patient or donor, altering them and then re-injecting them back into the patient to provide a highly personalized treatment. However, the manufacturing process for these therapies is complex and expensive. To increase the availability of these therapies, the industry is making strides in scaling up the manufacturing process to reduce costs.   According to Behnam Ahmadian Baghbaderani, executive director of Cell and Gene Therapy Process Development at Lonza, “It is important to incorporate innovative technologies and reduce the cost of goods and production in order to make these therapies widely accessible for a large number of patients.”  One essential way to achieve this is through automation: automated cell culture systems, including bioreactors, can be used to grow and expand cells in a controlled environment, which reduces the need for manual labor while increasing consistency and reproducibility. Simply put, scaling up the manufacturing process using automation makes these therapies more widely accessible to the large number of patients who need them.  Curious to Know More?  Listen to this episode of A View On Cell and Gene Therapies to explore how cell and gene therapies are manufactured. Get an inside look into the next steps for the industry from Lonza expert Behnam Ahmadian Baghbaderani.  

What If the Jab Were a Puff? A Look at Drug Delivery to the Lungs In this episode we explore the advantages of using inhalers for drug delivery with Lonza experts Kim Shepard and Matt Ferguson. In late 2022, China introduced the world's first Covid-19 vaccine to be inhaled into the lungs. The Chinese scientists who developed the vaccine tout its ability to directly stimulate the immune system's first line of defense – the lungs' mucous membrane. However, vaccines are just the tip of the inhaler iceberg. While we all are familiar with metered-dose inhalers, as well as nebulizers, for asthma, a whole range of therapeutic options for many diseases are becoming available through the technology known as dry-powder inhalers, or DPI. While the first commercially available DPIs appeared in the seventies, recent advances have opened the door to treatments for diabetes and even cancer. As with the Covid-19 vaccine, direct delivery to the lungs can be more efficient, but it also has the advantage of lowering toxicity by bypassing the liver altogether. Still, getting the correct amount of the molecule to the right part of the lung without unwanted immune responses is tricky business. Recently developed manufacturing techniques and new types of molecules make drug inhalers a continually evolving field full of potential advantages for patients. Curious to Know More? Listen to this episode of A View On Manufacturing Inhaled Drug Products to learn more about what it takes to develop effective treatments with insights from Lonza's Associate Director, R&D, Kim Shepard and Matt Ferguson, Lonza's Head of Respiratory Drug Delivery.

Safe Jabs Thanks to Horseshoe Crabs: Making Sure Your Injection is Free of Endotoxins Allen Burgenson, Lonza's expert for all things testing, speaks to us about the dangers of endotoxin contamination and the future of non-animal testing for it. “Before testing for endotoxins in the 1940s, a physician literally had to gauge the risk to your life because of something called injection fever,” explains Allen Burgenson. Luckily, we've come a long way since then. Thanks to advanced testing methods, one can rest assured today that any sort of injection or implant is completely free of dangerous endotoxins. Currently, the predominant mode is Limulus Amebocyte Lysate (LAL) testing, in which scientists harvest the bright blue blood of American Horseshoe Crabs and use the animal's primitive immune system to look for clotting reactions that would indicate the presence of any endotoxins. The horseshoe crabs, Burgenson explains, survive the extraction unscathed and are safely returned to the waters in less than 24 hours. However, in a continual attempt to remove animals from the testing pipeline, Lonza's recombinant factor C assay known as PyroGene could eventually replace LAL testing.

Developing Cell Culture Media For Growing Cells We are back! And in the first episode of our new season, we explore growing cells for therapeutic purposes with Lonza specialists Alexis Bossie, Director of Media R&D, and Tariq Haq, Senior Director of Global Media Marketing.        Lab-grown meat is having a moment—the FDA just declared one company's cultivated chicken safe to eat, and another type of lab poultry was just served for dinner to delegates at this year's COP27 climate conference in Egypt. Whether this piques your pallet's curiosity or turns your stomach, one thing is clear: growing meat in a lab for human consumption will take massive amounts of cell media. What is cell culture media? It is the medium in which cells grow in a lab, serving as both the cell's food and its shelter. The medium can take various forms, depending on which cell type is being grown and for what specific purpose. It is a careful recipe that balances the complex needs of cells in nutrients, energy, pH balance and saline percentage. Like our bodies, cell culture media is mostly water. However, crafting the right media is no simple matter: choosing the correct formula can make all the difference for the cell growth outcome, whether trying to stimulate virus production or make the tastiest animal-free cultivated cordon bleu on the planet. Curious to Know More? Listen to this episode of A View On to learn more about what it takes to grow cells in a lab. As a bonus for our listeners, at the end of the discussion, Alexis Bossie shares insights into the possibilities and obstacles of growing meat in the lab.   KEY TERMS IN CONTEXT: Cell culture media is the medium in which cells grow. It must meet all environmental conditions to keep a cell alive and flourishing. Either as a liquid or gel, synthetic or organic, the cell culture media's most important function is to deliver nutrients to cells and to wash away waste products. The osmotic balance in cell media is the salt and water balance needed to maintain proper cell functioning. An imbalance creates uneven flows of water between a cell and its media, resulting in a cell burst or cell shrinkage. A protein factory is a name given to batches of cells cultivated in a laboratory to produce high quantities of one or several types of proteins, often for therapeutic purposes. It must not be confused with the cell's own protein factory, aka the ribosome.   The metabolomics and the proteomics are the biological disciplines describing the metabolites and proteins in a cell. As these fields advance, so does the understanding of cultivating cells and developing the correct media for each type of desired growth and outcome.

Join us in celebrating the finale of our second season of A View On, the Lonza podcast. Over the past few months, we have brought you a series of insider conversations with our experts at Lonza, our partners, and leaders in the industry exploring the new pharma and biotechnology trends. We explored exciting topics, such as oncolytic viruses, the human microbiome, antibody-drug conjugates, nuances of early drug development, capsule manufacturing, and the use of artificial intelligence in the pharma industry. In the latest episode, the podcast host, Lonza's Martina Ribar Hestericová, recaps the highlights from this season and looks forward to later this year for what is coming up in the next season.   Interested to learn more? Visit our dedicated podcast site on www.lonza.com/a-view-on and don't forget to subscribe.

Artificial Intelligence and Life Sciences: The Dawning of a Digital Revolution in Pharma Manufacturing Dr. Loubna Bouarfa, CEO of OKRA.ai, and Stephan Rosenberger, Lonza's Head of Digital Transformation, discuss how AI is currently transforming the pharma industry. Machine learning is an essential subset of the vast field of Artificial Intelligence, in which computer programs aim to mimic human intelligence. Machine learning is at work in many of the algorithms that impact our daily lives, from suggesting new songs we might like to targeting ads. These algorithms learn from large data sets similarly to how a child learns during the early stages of development. As the algorithm matures from child to teenager to adult, it refines its own functioning by learning from its errors and through help from humans, much like we do. This powerful way of programming is making tsunamis across nearly all industries. It notably transforms the pharmaceutical world from drug discovery to production and even sales. Whether it is creating AI brains for companies to build digital workers to help their human employees or streamlining the factory with ultra-reliable predictive maintenance, the machine learning and artificial intelligence revolution is well underway. Curious to Know More? Listen to the conversation between A View On host Martina Hestericová and two world specialists about the present and future applications of AI and Machine Learning in the pharmaceutical industry.   KEY TERMS IN CONTEXT: Artificial Intelligence is a field of computer science where simulations of human intelligence by computer processes are used to improve the performance of machines. Machine Learning is a subset within the field of AI that is inspired by the way humans learn. Computational programs in the form of algorithms continually evolve by "learning" or processing information through trial and error, often with the help of human intervention. The goal of machine learning is to create machines that are independent learners capable of solving problems without human involvement. An AI brain, or digital brain, is the term used by OKRA CEO Dr. Lubna Bouarfa to describe a form of AI that learns from several data sets to create a company-specific intelligence to aid decision-making and predictions. For Bouarfa, the company's product can be employed to solve many of the current bottlenecks and problems in Life Sciences and pharma. Synthetic route optimization is used to map out the best way that a scientist can synthesize a compound. With the help of machine learning and AI, this route can be even further optimized by harnessing vast data sets and predicting outcomes. It is only one of many avenues where AI can greatly improve the efficiency of existing technologies in pharma manufacturing. In edge computing the computational work happens outside of the cloud and closer to the actual data event, which allows for real-time processing. With bigger data sets needed to feed in-house AI systems, edge computing architecture holds many advantages for companies looking to harness the power of AI for functions such as automation and safety.

Where There's A Risk, There's A Way: De-risking Drug Development at the Earliest Stages Lonza's wide array of analytical tools and professional experience create a go-to solution for small biotechs looking to decrease risk in their drug development process. An evolving toolbox of technology and advanced scientific knowledge is fueling the growth of a wide range of next-generation drugs in today's pipelines. These novel but complex products, while offering the ability to treat previously unmet medical needs across the globe, also present many challenges. This is often due to their unique profiles that require bespoke development and manufacturing processes as opposed to using well-known platform approaches, adding even more risk to a space fraught with uncertainty. This increasingly competitive market leaves little room for error or delay. Therefore, selecting and optimizing the right lead candidate becomes critical, as this allows you to de-risk your drug development process and maximize your chances of success. The largest cause of failure during drug development is most often related to safety and efficacy, so it is important to have processes in place that can identify potential issues as early as possible. Simple, cost-effective in silico and in vitro assessments can help look at potential developability challenges  in the earliest stages and allow for modifications to the drug candidate and its process development to mitigate potential efficacy, safety or manufacturability risks. Many of the drugs currently in early development around the world are initially developed by small biotechs, companies that often require the support of service providers to assist and to accelerate the de-risking of their candidates This is where Lonza's Early Development experts step in. Today's guest is Raymond Donninger, Senir Director of Commercial Development for Lonza's Early Development Services in Cambridge. To start the de-risking process, the team can predict development issues very early, based on the candidate's sequence and structure. This knowledge allows for modifications to the drug candidate and its process development to mitigate risk early and increase the likelihood of a successful first-in-human study. The experts then also apply in vitro tools to look at developability challenges  and to mitigate potential efficacy, safety or manufacturability risks. Curious to Know More? We previously addressed the importance of immunogenicity in decreasing risk in drug production in Episode 5. To take an even deeper dive into the whole process, listen to the conversation between Martina Hestericová and Raymond Donninger, the Senior Director of Commercial Development for Lonza's Early Development Services.   KEY TERMS in Context: In silico immunogenicity and human cell in vitro assays are two essential ways to de-risk a molecule's development pathway . In silico tests run computer models to predict a molecule's interaction with the human immune system; in vitro testing assesses the molecule's interaction with human immune cells. The attrition of a drug candidate occurs when it reaches clinical trials but fails for one reason or another. According to Donninger, an attrition rate of nine out of ten candidates has remained stubbornly high over the years. Attrition happens when a molecule has therapeutic potential but safety, target engagement or developability (for example complex, uneconomic manufacturing processes)  issues prevent the product from reaching the market. The de-risking process aims to reduce attrition to improve the chances for viable and safe therapies to make it to market. According to Donninger, a T-cell epitope is a sequence within the protein that has the potential to allow the immune system to recognize it as being foreign and then mount an unwanted and potentially dangerous immune response. To learn more about de-risking and immunogenicity, listen to this season's Episode Five.

Capsule Manufacturing: It's Not Only What's Inside That Counts The recent EU ban on Titanium Dioxide and changing customer habits are shaking up capsule production. Over the past few years, the coloring and manufacturing of pill capsules have undergone significant changes due to new EU regulations and customer demand for natural ingredients. And while originally invented to mask and protect the contents inside a capsule, research suggests that the color of a tablet or pill can affect how patients feel about their medication. Until recently, manufacturers have primarily used Titanium Dioxide (TiO2) to create white capsules due to its efficiency in protecting the active ingredients from UV rays. However, this year an EU-wide ban on TiO2 has forced the industry to move towards alternatives that work as well, or better, than TiO2. To add to the colorant shake-up, many people are actively avoiding unnatural ingredients in their food and nutritional supplements, which has created a new demand for plant-based capsule colorants. Anticipating these changes and solving the technological challenges in a timely manner are key to a successful long-term strategy for capsule manufacturing. Curious to Know More? Listen to the conversation between A View On host Martina Hestericová and Ljiljana Palangetic, Lonza's Associate Director of Hard Capsules R&D, about the challenges and solutions in current capsule manufacturing.   KEY TERMS IN CONTEXT: Pharmaceutical capsules can be either hard or soft. Soft-shelled capsules are one unique mold that encapsulates the contents, whereas the more widely-used hard-shelled capsules—such as the ones produced by Lonza—are two molded telescopic pieces of capsule: a smaller one contains the active ingredients, and a larger one encloses the capsule. Titanium dioxide (TiO2) is a widely-used pigment in capsule manufacturing, as well as in food, paint and sunscreen. Considered completely inorganic and nontoxic from a chemical point of view, it is labeled as an unnatural ingredient for ingestion, and carries the E number E171. Earlier this year, the European Food Safety Authority (EFSA) announced a six-month phasing-out ban of the colorant over concerns about nano-sized particles of TiO2 accumulating in the body. The full ban takes effect in August. The dip molding process is the manufacturing process for capsules. The final shape of the two pieces that make the capsules is defined by specifically designed molds, which are dipped in a bath of liquid formulation to pick up material that will, after the drying process, give the final capsule form, shape and composition.

Antibody-drug Conjugates: Next-Generation of Targeted Cancer Treatments Iwan Bertholjotti and Laurence Bonnafoux from Lonza give an insider look at how these promising treatments make it from development to commercialization. Chemotherapy is the first-line treatment for most types of cancer. However, one of the major challenges with this approach is that it targets both cancer and healthy cells, with patients suffering severe side effects. A new class of therapies, called antibody-drug conjugates, or ADCs, can target tumors much more precisely by harnessing the power of antibodies. The antibody can bind specific types of tumor cells, delivering a fatal blow to the cancer cells while sparing healthy cells. These promising new drugs have seen a significant uptick in FDA approvals in recent years, pointing towards a trend that could transform the way many diseases are treated. While numerous companies succeed in developing promising ADCs, manufacturing such complex and highly potent treatments presents unique challenges. The intricacy of scaling up the manufacturing of ADCs leads many companies to outsource their production, and Lonza currently fabricates the majority of ADC therapeutics in the world. For the companies that choose to work with Lonza, the collaboration simplifies the process and streamlines the supply chain. Decades of collective experience in fabricating ADCs means that the drugs make it from discovery to approval in less time, improving patients' lives through more effective, targeted treatments with fewer side effects. Curious to Know More? Listen to the conversation between A View On host Martina Hestericová and two of Lonza's experts on ADC manufacturing—Lonza's senior director of Commercial Development of bioconjugates, Iwan Betholjotti, and Lawrence Bonnafoux, Lonza's Head of MSAT BioConjugates.   KEY TERMS IN CONTEXT: Bioconjugates are a class of biopharmaceuticals developed by attaching two molecules together, of which at least one is a biomolecule. Examples of bioconjugates include antibody-drug conjugates (ADCs), PEGylated proteins, and vaccine conjugates. Antibody-drug conjugates consist of three parts: an antibody, a cytotoxic drug and the linker that covalently binds these two together. This approach combines the targeted delivery of the antibody with the cancer-killing power of the cytotoxic drug that would be too potent to be used on its own. A cytotoxic drug is a drug that contains a molecule toxic to cells, leading to cellular death. Used in traditional chemotherapy, these molecules attack both healthy and cancerous cells. When linked to an ADC antibody, they target only the tumor.  Targeted delivery of a cytotoxin is when a cell-killing toxin is delivered to a specific type of cell, such as tumor cells. This specificity allows for effective cancer treatment with fewer unwanted side effects for the patient. Scaling up production for bioconjugates involves moving from manufacturing small batches for clinical trials to large batches up to five kilograms for commercial production. This major challenge for companies is essential for the successful commercialization of ADCs.

Putting Manufacturing First: Codiak Moves Swiftly into Clinical Trials with Exosome-based Treatments  Sriram Sathyanarayanan, Codiak's CSO, and Linda Bain, their CFO, share how the company moved into clinical trials in under 6 years.   Exosomes, extremely small vesicles shed by all cell types, promise to become a viable delivery system for treatments of many diseases. But until recently, manufacturing them at a commercially viable scale has been unfeasible. That is why in 2015 the company Codiak took a two-pronged approach to developing exosome-based treatments: prioritizing both the engineering and manufacturing tracks from the outset. A mere six years after the company was launched, this approach has proven effective, with two promising studies in the clinic for tumor treatments, while most other developers are still at the starting blocks. The exosome field today holds the kind of promise that antibody and protein-based therapeutics did in the 1980s—the potential to improve both treatments and patient well-being is great. With a low risk of immunotoxicity, leveraging their natural abilities and engineering them to deliver targeted therapies could open new therapeutic pathways to previously undruggable targets. Yet as recently as only a couple of years ago, manufacturing exosomes was limited to small batches. Codiak has successfully increased production to thousand-liter batches, permitting clinical studies and greatly improving the prospective of widespread use. In collaboration with Lonza, Codiak is doubling down on their advantage with a recently established Center of Excellence for exosome manufacturing in Massachusetts. To learn more about Codiak's pipeline of therapeutic candidates with a potential to transform patients' lives, visit: https://www.codiakbio.com/pipeline-programs/pipeline Curious to Know More? Listen to Martina Hestericová's conversation with Sriram Sathyanarayanan and Linda Bain as they discuss the advantages of exosome treatments, how they are developed and why the company's early manufacturing strategy is paying off.   KEY TERMS IN CONTEXT:   Exosomes are nano-sized delivery vehicles generated by all eukaryotic cells. They are roughly between 30 and 120 nanometers large and originate when endosomes, or intercellular vesicles, are released into the blood, milk or tissue. Exosomes then become messengers and surrogates for the original cell. Their surface markers represent a location code and spread through the extracellular space in the body to communicate with other cells and deliver packages. Commercial exosome manufacturing is the scaling-up process of moving from small-batch exosome production that uses ultracentrifuges to large-scale production that in many ways resembles the processes already used to manufacture antibody and protein-based therapies.   The exosome's lumen is the interior volume of the exosome where, through biological engineering, the therapeutic molecule can be placed. The molecule can also be on the surface of the exosome, allowing for two alternative payload capacities, depending on the target. Immunosafety and immunotoxicity refer to how potentially safe or toxic the immune system's reaction to a molecule may be. Since exosomes already have a history of low immunotoxicity – think of blood transfers – their immunosafety is already proven to be very high.    

In this reposted episode from December 2020, we're exploring how a better understanding of exosomes is leading to new treatments and diagnostic technologies with Uwe Gottschalk.  According to Uwe , the exosome revolution is already in full march. As researchers begin to identify how these cell-generated, nano-sized delivery drones function in the human body, novel drug delivery prospects are emerging, including applications for cancer, neurodegenerative diseases and spinal cord injury recovery. Perhaps even more exciting is the role exosomes will play in diagnostic applications in the near future, wherein a liquid biopsy, based on a blood sample, would detect cancer or other diseases both more easily and in a more timely fashion than traditional biopsies. One of the many challenges is the ongoing task of defining the manufacturing protocols and processes for this new biotechnological paradigm. Even so, the field is abuzz with new discoveries, trials and general optimism about the potential of these microscopic extracellular delivery vehicles. Curious to Know More? Listen to our special, in-house episode of the podcast "A View On" and tune in next time as we are exploring the manufacturing challenges of exosome-based therapies with Codiak Biosciences. 

De-risking Drug Development: Early Testing for Toxicity Saves Time and Resources   Yvette Stallwood, head of Lonza's Early Development Services, talks about patient safety and other advantages of early testing for immunogenicity in the drug development pipeline.   In the high-stakes drug discovery game, from IND filings all the way up through the clinical trial phase, regulatory authorities are now expecting developers to have an immunogenicity risk strategy in place. “It really is essential that drug developers assess the immunogenicity risk as early as possible in the pipeline, as not only can it impact the functionality of the drug, but it can also be a significant safety risk for the patient,” explains Yvette Stallwood, whose work at Lonza's Early Development Services (EDS) is helping small and large biotech companies reduce risk with a “Right First Time” approach when developing drug therapies. Drug candidates often fail during clinical trials due to their toxicity to patients, which is evident, for example, in an immunogenic reaction—where the drug triggers an unwanted immune response known as immunotoxicity. This can result in the loss of years of work and funding. Stallwood and her team encourage their clients to begin with in-silico testing, where up to a thousand digital models of potential immunoresponses can be predicted.  Once the digital models show a candidate to have a low risk of toxicity, the EDS team then moves to human donor cell assays—with the advantage of screening up to fifty different immunotypes. The ideal time to assess immunosafety and immunotoxicity is well before deciding on a molecule as a lead drug candidate. By understanding as much as possible about the potential product through early testing, biotech companies are better equipped to take the correct path to regulatory approval with a drug that is ultimately safer for patients. Curious to Know More? Listen to the conversation between A View On host Martina Hestericová and Lonza's head of EDS Yvette Stallwood as they discuss de-risking the drug development process.   KEY TERMS IN CONTEXT: Anti-drug antibody (ADA) response happens when the patient's immune system generates antibodies to remove and clear the drug from the body. This can impact the effectiveness of the drug molecule as well as be dangerous for the patient. Immunogenicity testing is the process by which one can test for the body's immune response to a drug. With in-silico testing, the screening can be done quickly for a large swath of different immune system typologies before moving on to animal models or, preferably, human cell assays. Immunosafety and immunotoxicity refer to how potentially safe or toxic the immune system's reaction to a molecule may be. They deserve the utmost consideration when developing a leading drug candidate. De-risking is an EDS drug development strategy to ensure clients select the right drug candidate at the approval phase. De-risking avoids costly clinical trial failure through extensive immunotoxicity testing early in the process. In-silico testing uses computer models to test a molecule's reaction within an organism, such as a human immune system. The advantage is that they are quick and can test in hundreds and thousands of models. Since they are limited in their nature, they are only a first step. Once a drug candidate is selected as low risk using in-silico testing, further testing is needed using animal models or human cells assays. Human cell assays, in the context of drug development de-risking, are in-vitro tests that use actual human immune cells to test immune system responses to drug candidates. Although they are more costly and time-consuming than in-silico testing, the precision they offer is essential to establish the appropriate data for selecting lead drug candidates.

Host and Health: Tailoring Personalized Medicine Using The Unique Microbiome Fingerprint  Professor Eran Elinav from the Weizmann Institute of Science discusses how the interaction between the microbiome and its host is transforming personalized medicine.   “I believe that in the next five to ten years, exploiting the potential of the microbiome will be central to personalized and precision medicine,” explains Eran Elinav. His research into this second genome in the human body at the Weizmann Institute of Science in Isreal is shedding light on how these trillions of cells function and interact with their host. The individualized data from the unique microbiome fingerprint can be harnessed to tailor nutritional therapies to improve metabolic functions in the treatment of, for example, obesity and type 2 diabetes—with a wide range of further potential applications. And even small molecules found within the microbiome could themselves be developed into drugs. The future hope lies in the inherent therapeutic translatability of these insights from host-microbiome interaction research into treating the whole spectrum of metabolic diseases.   Curious to Know More? Listen to the conversation between Lonza's Martina Hestericová and Weizmann Institute of Science Professor and researcher Eran Elinav in this special episode of the "A View On" podcast. KEY TERMS IN CONTEXT: Genome: All of the genetic information of an organism. When speaking about the microbiome, it refers to an entirely different organism that is comprised of its own genetic makeup from the host—the interaction between the two genomes is the subject of study known as host-microbiome interaction. Microbiome: The extremely diverse ecosystem of hundreds, sometimes thousands of different species of microbes found in and on the human body. Microbial biodiversity is key to a healthy microbiome and a poor microbiome is linked to diseases such as inflammatory bowel disease, cancer and possibly some central nervous disorders. Therapeutic translatability: The ability to translate or apply basic research into therapies for the benefit of humans. As we understand more how the complex microbiome works, Professor Elinav asserts that these insights translate directly into ways to manipulate it and improve health. Personalised or Precision Medicine: A general trend to adapt treatments to individuals instead of a one-size-fits-all approach. In the context of host-microbiome research, as the microbiome is unique to each individual, it could hold the keys to specialized treatments by harnessing the individualized data.

Bridging Business and Biotechnology: Kodiak Sciences Is Increasing Treatment Efficacy for Retinal Diseases Victor Perlroth, MD, the Chairman and CEO of Kodiak Sciences, discusses how the company's ABC platform medicines are designed to treat the leading causes of blindness.   Age-related macular degeneration (AMD) is one of the leading causes of blindness in adults worldwide. This disease deteriorates the macula, a miraculous little spot on your retina that allows for precise vision in good light. Although several treatments exist for macular deterioration, they require frequent trips to the doctor's office for uncomfortable but quick and routine injections directly into the eye. The required frequency of the treatments means that most patients miss appointments, leading to undertreatment of the disease and permanent vision loss. In a manufacturing collaboration with Lonza, Kodiak is designing novel antibody-biopolymer conjugate (or ABC) medicines with the same efficacy and safety with much longer durability, allowing patients to visit the doctor on a realistic schedule over the long term. By focusing on business implementation alongside formidable biotech R&D, Kodiak Sciences is on track to bring together the necessary clinical and manufacturing elements for an FDA filing in 2023.   Curious to Know More? In this most recent episode of “A View On,” Lonza's Martina Hestericová is joined by Victor Perlroth, MD, the Chairman and CEO of Kodiak Sciences, to talk about the recent developments in AMD treatment research.    KEY TERMS:   Age-related macular degeneration (AMD) is a common degenerative disease of the retina. There are two types of AMD: Dry AMD occurs when the formation of debris (drusen) on the retina causes the macula to deteriorate over time. Patients sometimes experience vision loss and frequently experience substantial functional limitations, including vision fluctuations, loss of peripheral vision, and reduced night vision. Wet AMD is an advanced form of AMD. While wet AMD represents only 10% of the number of cases of AMD overall, it is responsible for 90% of AMD-related cases of severe vision loss. Wet AMD occurs when the growth of abnormal blood vessels underneath the macula leads to leakage of fluid and blood, which leads to visual distortion, acute vision loss, and total blindness if left untreated. Vascular endothelial growth factor (VEGF) is a sub-family of factors that stimulate the growth of blood vessels. In the case of AMD, these VEGF are overexpressed, creating leaking in the macula. This leakiness causes fluid to exit from blood vessels, causing swelling – or edema – of the retina and loss of vision. An antibody biopolymer conjugate (ABC) is Kodiak Science's proprietary platform for designing and developing drugs into the retina. The antibody in the KSI-301 molecule inhibits VEGF, while the biopolymer is comprised of phosphorylcholine, which creates a sort of “water cloak” around the antibody to increase its effectiveness. Phosphorylcholine is a natural component of the cell membrane of all the cells in our body, with remarkable properties. It attracts and binds water in a very strong – even permanent – way, creating what is known as “structured water,” which then impacts all biological interactions in the local area.

Safe Jabs Thanks to Horseshoe Crabs: Making Sure Your Injection is Free of Endotoxins Allen Burgenson, Lonza's expert for all things testing, speaks to us about the dangers of endotoxin contamination and the future of non-animal testing for it.   “Before testing for endotoxins in the 1940s, a physician literally had to gauge the risk to your life because of something called injection fever,” explains Allen Burgenson. Luckily, we've come a long way since then. Thanks to advanced testing methods, one can rest assured today that any sort of injection or implant is completely free of dangerous endotoxins. Currently, the predominant mode is Limulus Amebocyte Lysate (LAL) testing, in which scientists harvest the bright blue blood of American Horseshoe Crabs and use the animal's primitive immune system to look for clotting reactions that would indicate the presence of any endotoxins. The horseshoe crabs, Burgenson explains, survive the extraction unscathed and are safely returned to the waters in less than 24 hours. However, in a continual attempt to remove animals from the testing pipeline, Lonza's recombinant factor C assay known as PyroGene could eventually replace LAL testing. Curious to Know More? In Episode 2 of the new season of the podcast A View On, host Martina Ribarhestericova speaks with Lonza expert Allen Burgenson to discuss his close bond with the American Horseshoe Crab and the history of testing for endotoxin contamination.    KEY TERMS: Endotoxins are parts of bacterial membranes that could lead to a harmful reaction – or even death – if they enter a patient's bloodstream or spinal fluid. Surprisingly, we have kilograms of endotoxins in our stomachs, but even little more than a nanogram in the bloodstream could be deadly. Bacterial endotoxin tests, or BETs, is the general name for all assays used to detect endotoxins. Rabbit pyrogen tests are BETs that were developed in the 1940s using rabbits as test subjects. To ascertain the endotoxic danger to humans, the scientist observes a rabbit's reaction to an injection over a period of 3 hours. The European Pharmacopoeia Commission decided in June 2021 to completely replace the rabbit pyrogen test (RPT) within approximately 5 years. Limulus Amebocyte Lysate (LAL) is an aqueous extract of blood cells (amoebocytes) from the horseshoe crab, Limulus Polyphemus, that enables batch testing of vaccines and other drugs for endotoxins. The crab's extracted blood is a surprising blue color due to the crabs' copper-based Hamasyan. The obtained LAL is an opaque white-colored liquid that clots in the presence of any toxicity. PyroGene recombinant factor C is an animal-free way to test for endotoxins. It was initially developed at the National University of Singapore by Lin Deng and her husband Bo Ho to save money on testing at their relatively small lab. Lonza collaborated with Deng and Ho to become the first company to offer the test on a commercial scale.

A Welcome Virus: Cracking the Viral Code for the Battle Against Cancer Chairman and founder of PsiVac, Prof. Ghassan Alusi, and the chief operating officer, Imad Mardini, discuss how the company's proprietary oncolytic virus platform offers new hope for cancer patients. During a time when everyone actively fears viruses (especially THE virus) and their mutations, it is only cancer cells that have cause to worry about oncolytic viruses, and rightly so. These mutated viruses are administered directly into a tumor. Once inside, they crack open the tumor's cells in a process known as lysing that provokes a strong response from the body's immune system, which has, until then, ignored the cancerous cells. What's more, the therapy's attack doesn't stop at a single tumor. The replicating and lysing viruses release previously hidden tumor-associated antigens (TAAs) that alert the immune system about cancer cells to attack all over the body. The body's own immune system then goes on to destroy previously unrecognized tumors far removed from the initial injection point. The biotech company PsiVac advances this technology even further by creating a treatment platform that transforms the adenovirus, aka the common cold, into an especially powerful oncolytic virus. A precision modification in the virus's DNA improves its efficiency against cancer cells while making it harmless to other cells, rendering the treatment at once more effective and safer for patients. “Now that the technologies of other forms of immunotherapy are gaining ground, and as cancer remains a major cause of mortality, we now understand there is a huge need for oncolytic viral therapy,” says Prof. Alusi, whose company has planned to start Phase 1 clinical trials later this year. Unlike other immunotherapies, such as patient-centric CAR T-cell therapy, oncolytic viruses can be made in relatively large quantities once their efficacy and safety have been proved.   Curious to Know More? In the first episode of the new season of the podcast A View On, host Martina Ribar Hestericová discusses the current state of oncolytic viruses and their promising applications with Prof. Ghassan Alusi and Imad Mardini.    KEY TERMS: Cell lysing is the process of breaking down a cell's membrane, destroying the cell and releasing its contents into the body. If an oncolytic virus lyses a cell, it releases replicated versions of itself as well as antigens helpful in the immune system's fight against a tumor. Tumor-associated antigens (TAAs) are released once a cancer cell is lysed, setting the previously dormant immune system into action. The release of TAAs means that the tumor is no longer successfully hiding from the immune system, and the body can begin to fight the disease by its own means. The cytotoxicity of a virus is the extent to which a virus attacks and destroys cells, often an undesirable event. However, with oncolytic viruses, this cytotoxicity works in a patient's favor, thanks to gene editing, by being specifically designed to attack cancer cells. An agnostic oncolytic virus targets not only one or a group of cancers but is effective against all malignant solid palpable tumors. PsiVac's modified adenovirus has proven agnostic so far, making it a powerful weapon in the fight against cancer.

It’s the season finale for A View On, the Lonza podcast. Over the past 12 months of our initial run, we have brought you a series of conversations exploring the new pharma and biotechnology trends. We spoke to Lonza and the industry experts and discussed exciting topics, such as exosomes, stem cells, drug product testing, bioprinting, and much more. In the latest episode, the podcast host, Lonza’s Martina Ribar Hestericová, recaps the highlights from this season and looks forward to later this year for what is coming up in the next season. Interested to learn more? Visit our dedicated podcast site on www.lonza.com/a-view-on and don't forget to subscribe!

Devouring Bacteria: How Phage Therapy Is Shaping Antibacterial Treatments of the Future In this episode we speak with the CEO of BiomX, Jonathan Solomon, about producing and using phages to test and treat various diseases and conditions. Until very recently, treating a condition such as acne with an army of microscopic bacteria-destroyers known as phages—bacterial viruses that target and kill specific bacteria—would have seemed highly unlikely. However new research linking acne to an imbalance in the skin’s microbiome has opened the door to innovative treatment approaches. That’s where the biotech company BiomX comes in. Uniting powerful computational science with the inherent capacity of phages to destroy specific bacteria, BiomX creates natural and synthetic phage therapies for some of the most troublesome bacteria-related health issues: acne, atopic dermatitis, cystic fibrosis, inflammatory bowel disease (IBD) and even colorectal cancer. For acne the company has developed a successful cocktail of three different phages to treat the condition, with phase 2 testing close on the horizon. BiomX’s developments in phage therapy promise to change the way we treat imbalances in our microbiome, with potential health benefits for large swaths of the population. Curious to Know More? To learn more about BiomX, listen to the conversation with Jonathan Solomon on this episode of A View On: Phage Therapy. KEY TERMS: Bacteriophage (also known as a phage): A virus that attacks and devours only bacteria (‘phagein’ in Ancient Greek means to devour). Bacteriophages are bacteria-specific, which is both an advantage and disadvantage in manufacturing treatments. Fun fact: taken altogether, bacteriophages are the most numerous entity on the planet. Phage cocktail: Since a phage targets and destroys only one type of bacteria, treatments for complex ailments necessitate a mixture, or cocktail, of different phages to be effective. Phage fermentation: Although destructive, unwanted phages can grow during fermentation processes for wine-making and milk production, fermentation is nevertheless the optimal way to produce phages for therapeutic uses. Computational (science): Computer modelling of the phage and its potential interaction with specific bacteria (known as in silico testing) allows researchers to develop phage cocktails more efficiently and with a greater chance of success.

Delivering Personalized Therapies: Streamlining the Supply Chain for a New Generation of Treatments In this episode, we speak with Amy DuRoss, Co-Founder and Chief Executive Officer of Vineti, about the challenges facing "just-in-time" manufacturing and delivery of personalized therapies—and the solutions her digital startup provides.   According to Amy DuRoss, the COVID-19 vaccine distribution has exposed existing deficiencies in the entire pharmaceutical supply chain. This situation echoes, albeit on a far smaller scale, the distribution complexities of delivering cell and gene therapies (CGT). Unlike more traditional treatments, CGT requires the extraction of live cells from a patient or donor to then be delivered to a manufacturer and make it back to the patient in a timely manner. Her company Vineti "introduces a new level of fidelity, control, and transparency" into the personalized drug delivery process, streamlining CGT distribution through a novel digital orchestration platform. Based on an astute understanding of the behavior of care providers, specialized couriers and CGT manufacturers, her company has developed a software infrastructure that supports this exponentially complex delivery process. By facilitating Good Manufacturing Principles for all required stakeholders in the advanced therapy process, the Vineti platform ensures regulatory compliance and maintains both Chain of Identity and Chain of Custody from cell collection to manufacturer and back to the patient.   Curious to Know More? Take a listen to this episode of A View On: Streamlining Cell and Gene Therapy Manufacturing to learn more about Vineti. At the end of the discussion, as a bonus for our listeners, Amy DuRoss offers insight into some of the difficulties encountered in the COVID vaccine rollout, and how it parallels the complexity of the supply chain for CGT.   KEY TERMS:   Personalized medicine and therapies, such as cell and gene therapies (CGT), treat patients on a much more individualized basis. They require an unprecedented level of automation and navigation because the materials used to prepare the end product are raw cells originating from a donor or a patient. Good Manufacturing Principles (or Practices), GMP for short, are practices that ensure adherence to the guidelines put forth by regulatory agencies. They apply to any manufacturing industry but reach an unparalleled level of complexity in CGT production due to the implication of health care providers in the cell extraction and donor matching processes. A digital orchestration platform such as Vineti's uses the power of data management and user experience design to organize and execute an effective supply chain system in the face of exceptional complexity and strict regulations. Chains of Identity and Custody in the pharmaceutical supply chain are the cornerstones of regulatory compliance, whereby each step in the process and each individual involved is transparently identifiable in order to reduce the risk of contamination and to eliminate fraud. With CGT production, the process of extracting the raw materials for the treatment directly from the patient or donor multiplies the Chains of Identity and Custody, exponentially increasing the supply chain complexity.

Exosomes Know Where To Go: Using the Body's Own Cell-to-Cell Communication Network for Diagnostics and Drug Delivery We speak with Uwe Gottschalk, the Chief Scientific Officer of Lonza, about how a better understanding of exosomes is leading to new treatments and diagnostic technologies.   According to Uwe Gottschalk, the exosome revolution is already in full march. As researchers begin to identify how these cell-generated, nano-sized delivery drones function in the human body, novel drug delivery prospects are emerging, including applications for cancer, neurodegenerative diseases and spinal cord injury recovery. Perhaps even more exciting is the role exosomes will play in diagnostic applications in the near future, wherein a liquid biopsy, based on a blood sample, would detect cancer or other diseases both more easily and in a more timely fashion than traditional biopsies. One of the many challenges is the ongoing task of defining the manufacturing protocols and processes for this new biotechnological paradigm. Even so, the field is abuzz with new discoveries, trials and general optimism about the potential of these microscopic extracellular delivery vehicles. Curious to Know More? Lonza's Chief Scientific Officer gives us his expert insight into exosome research and application in this special, in-house episode of the podcast "A View On."   KEY TERMS: Exosomes are nano-sized delivery vehicles generated by all eukaryotic cells. They are between roughly 30 and 120 nanometers large and originate when endosomes, or intercellular vesicles, are released into the blood, milk or tissue. They then become messengers and surrogates for the original cell. Their surface markers represent a location code and spread through the extracellular space in the body to communicate with other cells and deliver packages. Extracellular vesicles (EV) are particles released from cells that cannot replicate but otherwise behave like the surrogate cells from which they originate. While there is some overlapping in definition between exosomes and EVs—all exosomes are extracellular vesicles but not vice-versa—exosomes are defined by their size (30 to 120 nm) and their biogenetic origin. Liquid biopsy: As with a traditional biopsy, a liquid biopsy is a test to diagnose and monitor diseases that uses a blood sample instead of a tissue sample. As a liquid biopsy is not restricted to one tissue or part of the body, the test is less invasive, cheaper and even more precise. Messenger RNA (mRNA) as a cancer biomarker:  Recent research has proven that mRNA from a blood test can be analyzed to find cancerous and pre-cancerous tumors throughout the body. As exosomes transport and stabilize the otherwise highly unstable mRNA, they could be targets for early detection and treatment in the near future. 

Bugs as Drugs: Harnessing the Therapeutic Potential of the Microbiome Lukas Schüpbach, the CEO of Bacthera, and Gemma Henderson, Bacthera’s Head of Project and Portfolio Management, speak to Lonza about creating pharmaceuticals from the human microbiome.   The human microbiome, comprised of trillions of bacteria, fungi, viruses, and archaea, is unique to each individual and develops over the course of lifetime, stabilizing once we reach adulthood. Despite the widespread understanding that this microbiome is a key component to our health, there are currently no commercially available live biotherapeutic products (LBPs). There is, however, an increasing amount of scientific evidence that using live biotherapeutic products to promote a vigorous microbiome can improve general physical health and positively impact quality of life by targeting diseases such as obesity, diabetes, inflammatory bowel syndrome and cancer. The biopharma company Bacthera is manufacturing and testing these difficult-to-produce anaerobic bacteria treatments that could improve metabolic functions and have anti-inflammatory effects. Alongside manufacturing, Bacthera is meeting the challenging delivery process to harness the therapeutic potential of the microbiome through easily administered, encapsulated pills. Curious to Know More? Listen to the conversation between Lonza and Bacthera in this episode of the podcast "A View On."   KEY TERMS: Microbiome: The extremely diverse ecosystem of hundreds, sometimes thousands of different species of microbes found in and on the human body. Microbial biodiversity is key to a healthy microbiome. A poor microbiome is linked to diseases such as inflammatory bowel disease, cancer and possibly some central nervous disorders. Live biotherapeutic products: These pharmaceutical products, LBPs for short, are unique because their active substance is actually a living organism. that has been identified as showing promise in treating one or sometimes several diseases. Enclosed process: The manufacturing of LBPs necessitates special equipment and expertise since many of the microorganisms are anaerobes and spore-forming organisms. To ensure a robust process with high yields, the manufacturing must be entirely enclosed so that these strains are not exposed to oxygen. Entrinsic strict delivery: As some microbes would not make it to the intestine by way of stomach acids, Bacthera has access to a proprietary technology that encapsulates the microbe to ensure targeted and precise delivery.  

Since the arrival of Crispr in 2009, gene editing has made its way into labs. Its groundbreaking importance was acknowledged through the 2020 Nobel Prize in Chemistry. Expanding gene editing for medical treatments, Cellectis focuses its 20 years of experience on developing cancer immunotherapies. Curious to Know More? Cellectis CEO André Choulika explains the technology of allogenic gene editing they invented in this episode of the podcast "A View On."

In 2016 Lonza established its Drug Product Services (DPS) to realize this supply chain model for its partners and clients. Since then the DPS has grown to a workforce of over 250 experienced experts focused on safety, efficacy and quality. To ensure that Lonza’s pharmaceuticals perform as expected in real-world situations, the DPS team simulates the entire administration process until certain the patient will receive the correct dose of the highest quality. 

Cannabinoids are emerging as a treatment option for autoimmune and other immune-related diseases thanks to their modifications as synthetic derivatives. Emerald Health Pharmaceuticals has widened the potential application of cannabinoids by designing cannabidiol and cannabigerol derivatives that have a greater effect on the endocannabinoid system and can interact with receptors and pathways from other biosystems. Alain Rolland, COO of EHP, talks about using cannabinoids for unmet medical needs.

Mesoblast CEO Dr. Silviu Itescu speaks to Lonza about the company’s advanced portfolio of anti-inflammatory allogeneic cellular medicines including remestemcel-L, which is currently being evaluated in a U.S. Phase 3 randomized controlled trial for acute respiratory distress syndrome (ARDS), the principal cause of mortality due to COVID-19 infection.

One of the limitations of cancer therapy is off-target activity, which often has devastating effects on patients’ life quality. A novel strategy pursued by Cybrexa Therapeutics uses treatments specifically targeting solid tumors by taking advantage of one of their universal biomarkers – acidity. By using cell-penetrating peptides bearing an anticancer cargo load, their platform brings the treatment directly inside tumors, leaving healthy cells alone and minimizing bystander killings.

Scientists have been printing cells for decades, but with the arrival of 3D bioprinters, getting printed tissue models to behave like living tissue has proved elusive. That is why angiogenesis and vascularization are two holy grails of 3D bioprinting. Allevi is not stopping at Earth-bound breakthroughs. The US company has also secured funding for simultaneous bioprinting experiments on the International Space Station.