This week’s headlines include: FDA advisers endorse 1st COVID-19 shots for kids under 5, World Bank approves $474 million loan to South Africa for COVID vaccines, Code Bio launches with $75M to reinvent genetic medicine for rare and not-so-rare diseases, WHO convenes experts to decide if monkeypox is an emergency, Moderna eyes fall vaccine season for omicron booster after phase 2/3 success, and BioNTech/Roche mRNA Therapy for Pancreatic Cancer Inspires Early Optimism.

Upcoming Conferences:

World Biopharm Forum 2022 – University of Oxford, UK – June 22-24, 2022

World Biopharm Forum 2022 is preparing for their upcoming conference Value Through Intensified Process II to be held at Lady Margaret Hall, University of Oxford, UK on June 22nd – 24th, 2022.  It will provide introductions, reviews, and a few case studies of exciting advances in the materials, equipment, technology and processes supporting bioprocess intensification.
https://www.subramanian.org.uk/conferences/

Save the Date! 2022 Virtual 3D Cell Culture Summit on June 30, 2022

3D cell culture is exploding. To capture the energy and excitement of this moment in research, Corning is bringing together industry leaders and innovators for a global virtual 3D Cell Culture Summit to share ideas and information, and to discuss the future of 3D applications, including spheroid, organoid and tissue models. Join industry leaders and key influencers for this can’t miss life sciences event.

This is a free event hosted by Corning Life Sciences. To register, please visit: https://cvent.me/58Wa1L

Podcasts:

Check out our podcast channel. We have over 50 great podcasts covering drug discovery, stem cell culture, upstream and downstream biomanufacturing and more! Click below to download from iTunes or Google play:

In Case You Missed It, Recent Articles on Cell Culture Dish and Downstream Column:

Using End-to-End Integrated Solutions to meet the High Demand for Viral Vector GMP Manufacturing

Animal cell culture is a central process for successful bioproduction of therapeutic proteins, viral vectors (for vaccines and gene therapies) and therapeutic cells (for cell-based therapies), which help prevent, treat and cure human disease. Optimized cellular nutrition is the key to unlocking cost-effective, highly efficient bioprocesses. Amino acids are essential nutrients and critical ingredients in cell culture media required for in vitro cell survival and proliferation because of their role as building blocks for proteins. Standard practice in biopharma is to add free amino acids to media formulations based on the nutritional requirements of the cell type of interest. However, the stability and solubility of certain free amino acids have quickly become limiting factors to higher productivity requirements as part of industrialized biomanufacturing (i.e., fed-batch or perfusion culture). Optimal media composition with the best ingredients at the right concentration helps drive productivity gains, streamline workflows, and reduce operating costs…

Gibco Cell Culture Basics Virtual Lab – A Great Example of the Benefits of Digital Training

Digital training tools are being popularized in the life sciences because of their accessibility, flexibility, and ability to foster learning across different competency levels and potentially in different geographical locations. The Gibco Cell Culture Basics Virtual Lab is a great example of this. Over the years, it has been a valuable resource for those new to cell culture or those seeking to improve their baseline skills. Due to popular demand, Gibco has recently re-launched the virtual lab with improvements and new features. The “renovated” lab builds on the original framework, offering scientists information on a wide range of cell culture topics and interactive activities within a virtual lab…

Extracellular Vesicles in Gene Therapy at this year’s ASGCT

The American Society of Gene and Cell Therapy (ASGCT) celebrated its 25^th year at their annual meeting at the Walter E. Washington Convention Center in Washington, DC last week and a full agenda is available on their site [ 1 ]. Originally designed for academic researchers to share their work, the annual meeting has grown to serve clinicians, bio-industry development, regulatory agencies, equipment manufacturers, patient advocates, and more. A truly remarkable number of activities and topics were addressed in the 4-day meeting. But from the scientific symposia and education sessions to the industry sponsored symposia and technology forums, it is really impossible to even begin to describe the topics and activities offered. There were over 6000 attendees participating in an excess of 80 unique sessions, covering many distinct cell gene technologies and addressing over 15 tissues, organs, and disease indications…

Increasing Single-Use Technology Operator Competency with New Training Methods

SUT training are cited as one of the top reasons for cGMP compliance inspection failures by drug manufacturers. Therefore, a concerted effort is needed in the biopharmaceutical industry to boost training competency across SUT operators. Recognizing the training gap, Pall Corporation and Lonza, embarked on a partnership to develop a standardized and comprehensive SUT training platform that combined the expertise of supplier and end-user. The program leverages a blend of digital media, virtual reality (VR) and in-person training tools to accommodate different learning styles. This variety of training tools helped to overcome the physical constraints of some types of single-use equipment to support the rapid and robust transfer of knowledge and skills required for consistent, strong performance…

Implementing Quality by Design for AAV Products

In this podcast, we talked with Parth Trivedi, Business Development Manager, Pall Corporation, about the importance of implementing a Quality by Design strategy for AAV product manufacturing and specific key steps for successful assessment…

Single-Use in Biotech – Addressing Opportunities and Challenges

Over the past decade, the growth and adoption of single-use technologies in biomanufacturing has been steep and sustained. What began primarily with single use bioreactors has expanded to include single-use components from early upstream processes through downstream and into commercial manufacturing. As of 2018, single-use systems were being used for about 85% of preclinical and clinical biomanufacturing and are increasingly being incorporated into commercial manufacturing¹. The benefits of single-use technologies have been well documented and include risk mitigation, increased efficiency, reduced personnel requirements, less validation time, and customizable solutions. The technology has also been found to reduce utility and water costs for cleaning and sterilization, as well as lower batch changeover time. This is particularly important for facilities that run smaller manufacturing lots, with several different products or are looking to maximize facility flexibility…

Full Adeno Associated Virus (AAV) Capsid Enrichment Using Mustang® Q Membrane

Adeno-associated virus (AAV) vectors are becoming increasingly important gene delivery platforms for the development of gene therapies for several reasons. They are relatively safe with low immunogenicity, and demonstrate high gene transfer efficiency and stable long-term expression in both dividing and quiescent cells. As well, a number of AAV serotypes exist that differ in their tissue tropism, or the types of cells they infect, making AAV a very useful system for transducing specific cell types. Typically, AAV production occurs within a packaging cell line, such as HEK293T cells, where plasmid containing the gene of interest (GOI) are assembled into a protein capsid shell. A current challenge in manufacturing is that the encapsidation efficiency for AAV is highly variable, which produces a heterogeneous mixture of AAV particles that contain the GOI (full) and particles without any genome (empty) or with fragmented, non-functional DNA (partial). Because only the full AAV capsids can exert a therapeutic effect in vivo, the other capsids are viewed as a process impurity and removing them is a necessary step in downstream purification process to achieve a consistent drug product. Existing methods to purify full AAV capsids like analytical ultracentrifugation (AUC) are effective yet have limitations in throughput and cost for a manufacturing environment, which has driven the development of more scalable methodologies for AAV purification…

Single-use Mixers – Ensuring the Customization, Scalability and Supply Required for Success

In this podcast, we talked with Dennis Hodgson and Phil Sanders from Agilitech about the benefits of single-use mixers, dealing with supply chain concerns, ensuring scalability, and tailoring a mixer to meet specific process needs…

Increasing Liquid Chromatography Efficiency with Fully-controlled Binary Blending

In this podcast, we spoke with Cory Hinz, Engineering Manager at Asahi Kasei Bioprocess about the different methods that are available for liquid chromatography mobile phase solutions and the benefits of inline blending. Cory also describes how to implement binary blending feeding of a liquid chromatography process using inline blending…

Agilitech Single-Use Tangential Flow Filtration (TFF) System for Biologics Manufacturing – A Case Study

Manufacturers in the biopharmaceutical industry are facing ever-expanding pipelines and the pressures to increase productivity and long-term sustainability while also rising to meet the challenges of new products with specialized processing requirements. This has led to increasing adoption of single use equipment into manufacturing workflows over traditional stainless-steel systems. While single use technology (SUT) designs have advanced for processes in both upstream and downstream unit operations, there are limitations to how tailored these units can be to fit the needs of a specific production process. Manufacturers are often required to adapt their process to what is commercially available resulting in technology solutions that may be suboptimal or unsustainable to meet future product demand. Those seeking more customization to a find ‘better fit’ are faced with piecemealing different components from different vendors into a contiguous system to achieve their production goals. Better engineering solutions are needed to create fit-for-purpose systems from the beginning that are cost-effective and scalable rather than the industry practice of reconfiguring standard systems. Addressing these gaps in process design requires thorough understanding of bioprocess requirements and equipment capabilities to build efficient processes to meet current production needs and ensure readiness to support future product growth…

White Paper:

Cell and Gene Therapy Catapult and Advanced Instruments collaborated on a project to see how modulating osmolality in the early stages of AAV upstream manufacturing can result in improved outcomes such as overall yield, quality, reduced manufacturing costs, and improved efficiencies. Download the paper now at Improving Titer, Quality and Efficiency of AAV Manufacturing and Production by Optimizing Osmolality.

Headlines:

“FDA advisers endorse 1st COVID-19 shots for kids under 5,” The Associated Press

The first COVID-19 shots for U.S. infants, toddlers and preschoolers moved a step closer Wednesday. The Food and Drug Administration’s vaccine advisers gave a thumbs-up to vaccines from Moderna and Pfizer for the littlest kids. The outside experts voted unanimously that the benefits of the shots outweigh any risks for children under 5 — that’s roughly 18 million youngsters. They are the last age group in the U.S. without access to COVID-19 vaccines and many parents have been anxious to protect their little children…

“World Bank approves $474 million loan to South Africa for COVID vaccines,” Reuters

The World Bank has approved a loan of 454.4 million euros ($474.4 million) to help South Africa fund COVID-19 vaccine purchases, the bank and South Africa’s National Treasury said in a statement. South Africa has recorded the most coronavirus cases and deaths on the African continent, with over 3.9 million confirmed cases and more than 101,000 deaths. It initially struggled to secure vaccines due to limited supplies and protracted negotiations, but it is now well-supplied with doses…

“Code Bio launches with $75M to reinvent genetic medicine for rare and not-so-rare diseases,” FierceBiotech

Duchenne muscular dystrophy (DMD) and Type 1 diabetes couldn’t be more different: One is fairly rare and degenerative, while we hear about the other life-altering condition all the time. A new biotech venture is launching with $75 million to try to improve on treatment for both, and it’s taking a few Big Pharmas along for the ride. Code Biotherapeutics has wrapped an upsized and oversubscribed series A financing thanks to Northpond Ventures, which led the round. Other investors include Amgen Ventures, UCB Ventures, Takeda Ventures, 4BIO Capital and CureDuchenne Ventures…

“WHO convenes experts to decide if monkeypox is an emergency,” CNBC

The World Health Organization will convene an emergency committee of experts to determine if the expanding monkeypox outbreak that has mysteriously spread outside Africa should be considered a global health emergency. WHO Director-General Tedros Adhanom Ghebreyesus said Tuesday he decided to convene the emergency committee on June 23 because the virus has shown “unusual” recent behavior by spreading in countries well beyond parts of Africa where it is endemic…

“Moderna eyes fall vaccine season for omicron booster after phase 2/3 success,” FierceBiotech

Moderna hopes that the successful readout from a phase 2/3 trial of its omicron-targeting COVID-19 vaccine will mean the booster shot is ready for people’s arms by late summer. In the 437-person trial, a 50 microgram booster shot of the vaccine, dubbed mRNA-1273.214, met all pre-specified endpoints including superior neutralizing antibody response against the omicron variant one month after administration when compared to Moderna’s approved Spikevax. The booster shot also increased neutralizing geometric mean titers against omicron approximately 8-fold above baseline, Moderna said in a June 8 announcement…

“BioNTech/Roche mRNA Therapy for Pancreatic Cancer Inspires Early Optimism,” BioSpace

BioNTech is best known for its mRNA COVID-19 vaccine developed with Pfizer, but a major part of the company’s pipeline is focused on mRNA cancer therapeutics. At the American Society of Clinical Oncology (ASCO)’s annual meeting this weekend, the company and Dr. Vinod Balachandran, M.D. of Memorial Sloan Kettering Cancer Center, presented preliminary Phase I data of its BNT122, which it is evaluating with Genentech (Roche) in pancreatic cancer…