Pharmaceutical Technology Is Now PharmTech: A Sharper Name for a Smarter Industry

Navigating FDA Shakeups and Obesity Drug Wars

Behind the Headlines Episode 28

Ask the Expert

Closing the Gap in Modern Process Control and Automation for QC/QA

Drug Digest December 2025

How It Helps Avoid Facility and Equipment Problems

A “Plan, Prevent, Prove” Approach

Expertise on using Hybrid LC-MS versus LBA for bioanalytical analysis.

Bioanalytical Methods to Optimize Drug Development: Hybrid LC-MS vs LBA

Selecting the right bioanalytical method to support qualitative and quantitative analysis requires flexibility and expertise from a CRO equipped to adapt to industry advancements.

Understand when to use LC-MS, LBA or both.

Learn why keeping LBA and Hybrid LC-MS in the forefront of your studies can result in big benefits.

Understand the best method to optimize your drug development outcomes.

See the entire 45-minute deep-dive broken into an 11-part video series, 

Videos

Drs Dawn Dufield and Dominic Warrino, KCAS Bioanalytical and Biomarker Services, share expertise on using Hybrid LC-MS versus LBA for bioanalytical analysis.

Feliza Mirasol is the science editor for 

Editor's note: this story was originally published in BioPharmInternational.com.

Roche has entered into an agreement to sell its large-scale biologics manufacturing site in Vacaville, Calif., to Lonza for $1.2 billion, Lonza announced on March 20, 2024. Lonza plans to invest approximately CHF 500 million (US$557 million) to upgrade the facility and enhance capabilities at the site for next-generation mammalian biologics.

The deal is expected to close in the second half of 2024, subject to customary closing conditions. The Vacaville site will be integrated into Lonza’s Biologics division upon closing and will be included in Lonza’s network of existing mammalian manufacturing sites in Visp, Switzerland; Slough, United Kingdom; Tuas, Singapore; Portsmouth, NH; and Porriño, Spain (1).

“The Vacaville site is a highly valuable strategic acquisition that will make capacity immediately available for our customers and unlock future growth for our Biologics division. It will support us in providing a commercialization path to existing customers and incremental large-scale commercial capacity to our partners,” said Jean-Christophe Hyvert, president, Biologics, Lonza, in a company press release.

Lonza will supply the products currently manufactured by Roche at the Vacaville site in the medium term as it phases out those products over time to serve alternative customers. With a total bioreactor capacity of approximately 330,000 L, Lonza expects the Vacaville acquisition to significantly increase its large-scale biologics manufacturing capacity, allowing it to meet demand for commercial mammalian contract manufacturing from customers with existing commercial products. The facility will also serve molecules within Lonza’s network that are currently on the path to commercialization.

The facility’s capacity makes it one of the largest biologics manufacturing sites in the world by volume, according to the Lonza company press release. Because demand for commercial-scale capacity for biologics is expected to remain high across the contract development and manufacturing organization industry as the result of anticipated approvals for innovative new therapies, the acquisition puts Lonza in a position to offer immediate access to significant new capacity in the United States. The move also creates a significant West Coast commercial manufacturing presence, which complements Lonza’s existing biologics site on the East Coast (Portsmouth, NH). The US sites also complement Lonza’s manufacturing network across Europe and Asia.

“Demand for biologics manufacturing capacity by volume is projected to reach nearly 4400 kL, a five-year growth rate of nearly 11.5% per year (just over 2500 kL in 2022). Global biologics manufacturing capacity will increase to nearly 8400 kL by 2027 from nearly 6500 kL in 2023,” according to the 2023 CPHI annual report (2).

“We have deep and long-standing industrial expertise in delivering commercial-scale manufacturing services for our customers’ therapies. In combining this with the strong legacy of the Vacaville facility, its highly skilled colleague community, and its proven track record on quality, we are excited to take our leading large-scale mammalian offering to its next chapter of growth,” Hyvert said in the press release.

Under the transaction, Lonza will offer positions to the approximately 750 Genentech employees working at the Vacaville facility.

1. Lonza. Mammalian Biopharmaceutical Services. 

 (accessed March 25, 2024).
2. Ecker, D. M.; Seymour, P. Supply and Demand Trends: 2022-2027 Mammalian Biomanufacturing Industry Overview. In 

Articles

The Vacaville, Calif., site acquisition gives Lonza one of the largest biologics manufacturing sites for mammalian cell-based therapies worldwide.

Roche Sells Large-Scale Biologics Manufacturing Site in US to Lonza for $1.2 Billion

Podcast will give an overview of the latest analytical tools and their applications in the drug development market, covering topics such as the current implementation of capillary electrophoresis, the impact of emerging therapies on innovation, and the ethical and regulatory considerations in ongoing drug development.

Dr. Jan Bekker, Vice President of Business Development, Commercial and Technical Operations at BioCina.

Jan Bekker, Vice President of Business Development, Commercial and Technical Operations at BioCina, boasts over 20 years of experience in the biopharmaceutical field, with notable tenures at Pfizer, Hospira, Centre for Marine Bioproducts Development, Stellenbosch University, and the Queensland University of Technology. With a background enriched by a Ph.D. from Stellenbosch University and an MBA from the University of New South Wales, Jan tailors programs spanning pre-clinical to commercial manufacturing phases, enhancing BioCina's business development through his comprehensive industry insight and technical expertise.

 presents the Drug Solutions podcast, where the editors will chat with industry experts from across the pharmaceutical and biopharmaceutical supply chain. Join us as experts share insights into your biggest questions—from the technologies, to strategies, to regulations related to the development and manufacture of drug products.

Podcasts

In this episode of the Drug Solutions Podcast, Jan Bekker, Vice President of Business Development, Commercial and Technical Operations at BioCina, discusses the latest analytical tools and their applications in the drug development market.

Drug Solutions Podcast: Applying Appropriate Analytics to Drug Development

Text sign showing Industry News. Business photo text delivering news to the general public or a target public | Image Credit: © Artur - © Artur - stock.adobe.com

Endo International announced on March 12, 2024 that one of its operating companies, Par Pharmaceutical, is voluntarily recalling one lot of Treprostinil Injection 20 mg/20 mL (1 mg/mL) because of the potential presence of silicone particulates in the particle solution (1).

Treprostinil Injection is a prostacyclin vasodilator used to treatpulmonary arterial hypertension. It is formulated for subcutaneous or intravenous infusion. According to the company, administration of an injectable product containing particulate matter may cause irritation or swelling. More seriously, if the particulates reach the blood vessels, they can travel to organs and block blood vessels, which can cause a stroke or death.

The recalled product was distributed in 20-mL multidose vials as sterile solutions in water for injection. Vials are individually packaged in cartons under NDC #42023-206-01. The impacted lot, 57014, was distributed nationwide to wholesalers and hospitals from June 16, 2022 through Oct. 17, 2022 and has an expiration date of 04/2024.

Written notification about the recall has been sent to wholesale accounts and hospitals that received the affected lot. The company stated in a press release that, “Wholesale distributors and hospital pharmacies that have the product being recalled should immediately discontinue use and stop distribution immediately. If you have further distributed the recalled product, please notify your accounts or any additional locations which may have received the recalled product.”

The company states that it has not received any reports of adverse events related to the recall as of the date of the press release. Adverse reactions may be reported to FDA via it’s MedWatch Adverse Event Reporting program.

There are regulatory guidelines in place to mitigate the problem of particulates. According to the FDA guidance document, 

Inspection of Injectable Products for Visible Particulates

 patient safety can be impacted by visible particles in injectable products (2). The guidance states that companies should develop and implement a “holistic, risk-based approach” to controlling these particulates and preventing contamination. The guidance document states, “The clinical manifestations of adverse events caused by particulate contamination vary and may depend on the route of administration (e.g., intravascular, intravisceral, intramuscular), patient population, and nature or class of the particulates themselves (e.g., physical size or shape, quantity, chemical reactivity to certain cells or tissues, immunogenicity, infectivity, carcinogenicity). Particulates in intravascular or intravisceral injections generally can cause more adverse events than those in subcutaneous or intramuscular injections.”

Risk assessments should be conducted during product development, according to the FDA guidance document, and any visible particulates that may pose a risk should be identified. “Manufacturers should also consider the potential sources of particulates, appropriate analytical methods to monitor them, and mitigation strategies to prevent their presence in the final product,” the guidance document states.

Visual inspection of the product should be part of a program to ensure products are free of contaminates, according to the agency. “During injectable product development, manufacturers should establish procedures for inspecting the product, statistical sampling plan(s), acceptance/rejection criteria, and procedures for evaluating inspection results. Inspection procedures carried over from another site or another product may not always be suitable for a new product,” the agency states in the guidance document.

FDA. Par Pharmaceutical Issues Voluntary Nationwide Recall of One Lot of Treprostinil Injection Due to Potential for Silicone Particulates in the Product Solution. Press Release. March 12, 2024.

Draft Guidance for Industry,Inspection of Injectable Products for Visible Particulates

The company is recalling one lot of the product because of the potential presence of silicone particulates.

Par Pharmaceutical Recalls Treprostinil Injection 20 mg/20 mL (1 mg/mL)

Hand shaking which print screen on wooden cube block in front of human icon for business deal and agreement concept | Image Credit: © Dilok – © Dilok - stock.adobe.com

Editor's note: this story was originally published on 

On March 14, 2024 AstraZeneca announced its acquisition of Amolyt Pharma, a clinical-stage biotechnology company focused on developing novel treatments for rare endocrine diseases, in a deal worth potentially $1.05 billion. The transaction includes an $800 million upfront payment and a potential milestone payout of $250 million. With this acquisition, AstraZeneca will obtain eneboparatide (AZP-3601), Amolyt’s Phase III investigational therapeutic peptide that operates with a new mechanism of action intended to treat hypoparathyroidism.

Hypoparathyroidism is characterized by a deficiency in a patient’s parathyroid hormone (PTH) production, leading to severe dysregulation of calcium and phosphate. Patients suffer from life-altering symptoms and complications, including chronic kidney disease. An estimated 115,000 people in the United States and 107,000 people in the European Union, approximately 80% of whom are women, are affected by the disease, making it one of the largest rare diseases being studied. 

Amolyt’s clinical pipeline also includes other therapeutic peptides under development to treat rare endocrine diseases. Among them is AZP-3813, a peptide growth hormone receptor antagonist in Phase I clinical development. AZP-3813 is intended for the potential treatment of acromegaly. With these peptides, especially eneboparatide, AstraZeneca aims to increase its rare disease pipeline, especially in bone metabolism and rare endocrinology.

“Chronic hypoparathyroid patients face a significant need for an alternative to current supportive therapies, which do not address the underlying hormone deficiency. As leaders in rare disease, Alexion is uniquely positioned to drive the late-stage development and global commercialization of eneboparatide, which has the potential to lessen the often debilitating impact of low parathyroid hormone and avoid the risks of high-dose calcium supplementation. We believe this program, together with Amolyt’s talented team, expertise and earlier pipeline, will enable our expansion into rare endocrinology,” said Marc Dunoyer, CEO, Alexion, AstraZeneca Rare Disease, in a company press release.

This move follows other recent acquisitions that AstraZeneca has been making. In January, 

© reported on the pharma company’s acquisitions of Icosavax (1) and Gracell (2), both of which were acquired for over a billion dollars. Icosavax’s acquition, which was finalized in February 2024 (3), marked another addition to AstraZeneca’s growing pipelines. In addition to these transactions, AstraZeneca also recently poured $828 million into its Liverpool and Cambridge manufacturing sites in the United Kingdom. The investement is to be used in research, development, manufacturing, and expansion. With the strengthening of these facilities and the continued acquisition of promising drug candidates, AstraZeneca is expected to boost its positioning in the UK pharmaceutical market.

AstraZeneca to Acquire Icosavax in Deal Valued at Approximately $1.1 Billion

AstraZeneca Poised to Acquire Cell Therapy Developer, Gracell, in Deal Valued at $1.2 Billion

AstraZeneca Completes Acquisition of Icosavax

AstraZeneca’s acquisition of Amolyt Pharma includes eneboparatide, a Phase III therapeutic peptide for the treatment of hypoparathyroidism.

AstraZeneca to Acquire Amolyt Pharma in Deal Worth Potentially $1.05 Billion 

Belgium-based contract development and manufacturing organization (CDMO) White Raven and Cytiva announced on March 19, 2024, that White Raven is installing Cytiva's SA25 Aseptic Filling Workcell at its fill/finish site. This installation completes White Raven’s drug product platform and will increase its manufacturing efficiency. The site is expected to be good manufacturing practice (GMP)-compliant with the new system by the end of 2024.

SA25 is an integrated gloveless robotic isolator that eliminates the need for operator intervention, according to a company press release. The system is designed to provide an optimal aseptic process and comprises a standardized flexible filler that is capable of producing vials, syringes, and cartridges in batch sizes ranging from 100 to up to 20,000 units. The system has a fill-volume range of 0.2 mL – 50 mL. Because it is a closed robotic system, it reduces the risk of product contamination. It also allows for changeover between drug formats within a 45-minute timeframe.

Ludovic Brellier, president, Biotechnology Integrated Solutions and Business Operations, Cytiva. Image courtesy of Cytiva.

“White Raven recognizes that by replacing a human operator with a robotic gloveless isolator, they can remove the biggest risk for contamination—ensuring they are taking the right steps to protect customers’ high value drug products. The need for greater standardized solutions to improve this process is a trend we are seeing across the industry,” Ludovic Brellier, president of Biotechnology Integrated Solutions and Business Operations, Cytiva, says.

White Raven offers a range of formulation services adapted to clinical batches and is capable of working with mixing volumes as low as 50 mL and up to 20 L. The CDMO also offers GMP formulation and aseptic filling of injectable drugs for pre-clinical, clinical, and orphan drugs. White Raven employs single-use consumables over the full aseptic filling process to also reduce contamination risk.

The company is part of Cytiva’s aseptic filling user group, which is a group that works closely with Cytiva to collaborate on best practices, such as system validation and operations. White Raven expects the installed workcell to complete a site acceptance test by mid-2024, prepping the site for GMP readiness by end of the year.

Considerations for improving aseptic operations in the bio/pharma industry are an ongoing exercise for manufacturers. Automation technologies have been a major solution in this area. However, in one sense, automated aseptic processes can be seen as “often playing catch up to the new tools that are being developed,” according to Dan Strange, chief technology officer at Cellular Origins, in a 

 video interview (1). “These consumables and equipment … have been fundamentally designed for manual processes … and that's very hard to automate. It's … straightforward for a manual operator to automate, but it's hard for traditional automated systems to handle that,” Strange noted in the interview.

Drug Digest: Improving Aseptic Processing and Manufacturing Needs

CDMO White Raven aims to reduce contamination risk and gain the capability to handle multiple product formats with the installation of Cytiva’s SA25 Aseptic Filling Workcell.

White Raven to Increase Manufacturing Efficiency with Cytiva’s Aseptic Filling Workcell

Felicity Thomas is the former Associate Editorial Director of Pharmaceutical Technology, Pharmaceutical Technology Europe, BioPharm International.

Text sign showing Industry News. Business photo text delivering news to the general public or a target public | Image Credit: © Artur - stock.adobe.com

The government of the United Kingdom announced on March 20, 2024 that it is 

 that are considered highly addictive, dangerous, and pose a higher risk of accidental overdose (1). These substances will now be subjected to the strictest controls, in line with the controls placed on fentanyl.

As a result of these new government controls, if anyone is involved in the production or supply of the synthetic opioids, they will be subjected to the full force of the law. “We are highly alert to the threat from synthetic drugs and have been taking a range of preventative action, learning from experiences around the globe,” said James Cleverly, UK home secretary in a press release. “Our plan is working—the overall quantities of synthetic opioids reaching the UK remain lower than other countries, but we are not complacent.”

Synthetic opioids are a major contributor to preventable drug overdose deaths, particularly affecting the United States. However, Europe is also now fearing a crisis, as the availability of these drugs is increasing (2). Although opioids can be prescribed for pain relief, any regular non-medical use, prolonged use, misuse, or use without medical supervision can result in dependence on the drug and various related health problems (3).

Of the 15 synthetic opioids, 14 are nitazenes, which were developed by the pharmaceutical industry during the 1950s but were never approved for use as medicines and can cause fatal respiratory depression. Some nitazenes are known to be hundreds of times more potent than heroin, and these compounds are being detected in other products, meaning consumers may be using these substances without realizing (4).

“Synthetic opioids are significantly more toxic than heroin and have led to thousands of deaths overseas,” added Chris Philp, UK’s crime and policing minister. “We are determined to ensure these destructive and lethal drugs do not take hold in our communities in the UK.”

The opioids that have been made Class A drugs under the UK’s Misuse of Drugs Act 1971 are metonitazene; protonitazene; isotonitazene; butonitazene; flunitazene; metodesnitazene (metazene); etodesnitazene (etazene); N-pyrrolidino-etonitazene (etonitazepyne); N-piperidinyl-etonitazene (Etonitazepipne); N-pyrrolidino protonitazene; ethyleneoxynitazene; N-desethyl protonitazene; N-desethylisotonitazene; N-desethyl-etonitazene; and brorphine.

Additionally, the UK government has placed controls on five other substances on March 20, 2024. Of these substances, three are stimulants—diphenidine, ephenidine, and methoxyphenidine—that are now Class B drugs; one is a synthetic cannabinoid receptor agonist (cumyl-PeGaClone) that is also now a Class B drug; and the final one is a short-acting benzodiazepine (remimazolam), which is now a Class C drug.

1. UK Gov. More Synthetic Opioids Banned to Protect Communities. 

, News Story, March 20, 2024.
2. Bauer-Babef, C. Opioids on the Rise, Under Close Surveillance in Europe. 

, March 6, 2024.
3. WHO. Opioid Overdose. 

, Fact Sheet, Aug. 29, 2023.
4. Holland, A.; Copeland, C.S.; Shorter, G.W.; et al. Nitazenes—Heralding a Second Wave for the UK Drug-Related Death Crisis? 

More synthetic opioids have been placed under the strictest controls in the United Kingdom.

UK Government Bans 15 Synthetic Opioids

FDA has approved Lenmeldy (atidarsagene autotemcel) from Orchard Therapeutics, making it the first approved gene therapy for treating children with pre-symptomatic late infantile, pre-symptomatic early juvenile, or early symptomatic early juvenile metachromatic leukodystrophy (MLD) in the United States. Following the March 18, 2024, approval, AGC Biologics announced that it will be manufacturing the therapy at its Milan, Italy, site.

According to a FDA press release, MLD is a debilitating, rare genetic disease that affects the brain and nervous system caused by a deficiency of the arylsulfatase A (ARSA) enzyme. This deficiency leads to a buildup of sulfatides in the cells, which causes damage to the central and peripheral nervous system, leading to a loss of motor and cognitive function and early death. MLD is estimated to affect one in every 40,000 individuals in the US, and there is no cure for it. Currently, treatment typically focuses on supportive care and management of symptoms.

Peter Marks, director, FDA’s Center for Biologics Evaluation and Research (CBER). Image courtesy of FDA.

“This is the first FDA-approved treatment option for children who have this rare genetic disease,” said Peter Marks, director of FDA’s Center for Biologics Evaluation and Research (CBER), in the press release. “We remain committed to advancing scientific and regulatory principles that enable the efficient development and review of safe, effective and innovative products that have the potential to change patients’ lives.”

Lenmeldy, derived from the patient’s own hematopoietic stem cells (HSCs), is a one-time, individualized single-dose infusion. The patient’s HSCs are collected and genetically modified to include functional copies of the ARSA gene. The modified cells are then transplanted back into the patient where they engraft within the bone marrow and supply the body with ARSA enzyme-producing myeloid cells. The enzyme helps break down the build-up of sulfatides and may stop the progression of MLD. Prior to treatment with Lenmeldy, patients must undergo high-dose chemotherapy to removes cells from the bone marrow that can then be replaced with the modified HSCs, according to the FDA release.

“This approval represents important progress in the advancement and availability of effective treatments, including gene therapies, for rare diseases,” said Nicole Verdun, director of the Office of Therapeutic Products in CBER. 

Nicole Verdun, director, Office of Therapeutic Products, CBER. Image courtesy of FDA.

The application for Lenmeldy received priority review, orphan drug, rare pediatric disease, and regenerative medicine advanced therapy designations.

Following Lenmeldy’s approval, contract development and manufacturing organization (CDMO), AGC Biologics, announced that its Milan, Italy, site has received clearances from both US and European regulators and can now use approved commercial processes to supply patients with the gene therapy. The company will begin commercial manufacturing for the lentiviral vector and genetically modified cell drug product (DP) for Lenmeldy at the site.

“It is wonderful to see Orchard Therapeutics reach this goal. This accomplishment is a strong proof point of our commitment at AGC Biologics to collaborating directly with developers to meet rigorous regulatory standards and achieve the best possible results for our partners,” said Patricio Massera, CEO, AGC Biologics, in a March 18, 2024 company press release.

According to the company press release, the Milan site has become the only CDMO site to receive FDA clearance to commercially produce this gene therapy for US patients. AGC Biologics stated in the release that the FDA approval is “the culmination of a partnership that started in 2018 with Orchard Therapeutics, which also includes guiding the product through commercial approval by the European Commission (EC) in 2021 [under the European brand name Libmeldy] (1). AGC Biologics’ Milan site is uniquely connected to this product’s full lifecycle, as the facility and our scientific teams partnered with each company that owned this treatment’s IP [intellectual property] over the [p]ast 20 years to help advance it from research and development stages to this DP commercial milestone.”

Luca Alberici, general manager of AGC Biologics Milan, stated in the release, “We congratulate Orchard for reaching this important stage. Our team has had the privilege of supplying every clinical milestone for Lenmeldy and are glad to see it reach FDA approval. I am proud of the work of the Milan team.This demonstrates our unique ability to collaborate on technical processes to deliver groundbreaking treatments to patients worldwide. This approval makes our site one of the rare few in the viral vector and genetically modified cells field that has commercial manufacturing authorization from two of the world’s leading regulatory authorities."

"AGC Biologics has played a pivotal role as a strategic partner throughout the clinical development and commercial scale-up of atidarsagene autotemcel, which helped facilitate regulatory approvals in Europe and the US," added Nicoletta Loggia, chief technical officer of Orchard Therapeutics, in the company press release. "Utilizing our … commercial HSC gene therapy manufacturing platform, we have consistently met the demands for drug product production since the European launch in early 2021. This has enabled us to provide treatment to patients from three continents, including Europe on a commercial basis, the Middle East through treatment abroad programs, and the US, under compassionate use. With the recent US approval, we are excited to further expand access to this vital therapy for eligible children with early-onset MLD."

1. Orchard Therapeutics. Orchard Therapeutics Receives EC Approval for Libmeldy for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD). Press Release, Dec. 21, 2020.

FDA's approval makes Orchard Therapeutics' Lenmeldy (atidarsagene autotemcel) the first gene therapy approved to treat pediatric metachromatic leukodystrophy in the US.

FDA Greenlights Orchard Therapeutics' Gene Therapy for Children's MLD; AGC Biologics Selected for Manufacturing

On March 13, 2024, Japan=based Ricoh Company, a digital services company, and Ireland-based ERS Genomics announced that they have entered into a non-exclusive licensing agreement in the United States and Japan that gives Ricoh access to clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) genome editing technology patents managed by ERS Genomics.

CRISPR/Cas9 has been shown to be a breakthrough gene editing technology as it enables relatively easy modification of targeted genome sequences. It has become an essential tool in drug discovery research and has application across other fields, according to Ricoh in a company press release. The CRISPR technology is “

” that UK Medicines and Healthcare products Regulatory Agency’s (MHRA’s) approval of Casgevy in November 2023 “caused quite a stir in both the medical and pharmaceutical worlds” (1,2). Alongside FDA’s approval of Casgevy in December 2023, the regulatory authorities’ approvals mark a significant 

 in the biopharma industry (3,4). Casgevy is the first therapeutic based on CRISPR/Cas9 gene-editing technology to be approved by regulatory authorities anywhere in the world (4).

As part of Ricoh’s strategy with its recent moves, the company is aiming to predict the mechanism of action of drug candidates for patients with varied genetic backgrounds. The goal is to improve the speed and efficiency with which messenger RNA (mRNA) is designed; the company plans to achieve this goal by combining certain core technologies with the CRISPR/Cas9 genome editing technology. In 2022, the company completed the acquisition of Baltimore, Md.-based Elixirgen Scientific (5), whose core technologies enable the rapid and efficient differentiation of human induced pluripotent stem cells as well as the design, production, and management of mRNA.

By manipulating the genetic editing of cells to enhance or diminish specific functions, it thus becomes possible to create highly reliable disease models, including models for rare diseases. Ricoh expects that such an advancement will lead to shorter drug development timelines and increased success rates, according to the company press release. Meanwhile, following through on its acquisition of Elixirgen Scientific, Ricoh has been expanding the application areas for that company’s technology through its expertise in digital and artificial intelligence technologies.

New Medicines, Markets, and Manufacture: CRISPR for Sickle Cell Disease and β-thalassemia

 online, Nov. 30, 2023.
2. Vertex Pharmaceuticals. Vertex and CRISPR Therapeutics Announce Authorization of the First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY (exagamglogene autotemcel), by the United Kingdom MHRA for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia. Press Release, Nov. 16, 2023.
3. FDA. FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease. Press Release, Dec. 8, 2023.
4. Mirasol, F. 

Milestone Approval to Steady Stream, Gene Editing Revs Up

 (2), 16–19,25.
5. Ricoh. Ricoh to Strengthen Support for Drug Discovery Services Using mRNA. Press Release, May 17, 2022.

Ricoh aims to create novel disease models for R&D using ERS Genomics’ CRISPR/Cas9 genome editing technology.

Ricoh and ERS Genomics Enter License Agreement for CRISPR/Cas9 Technology

Chris Spivey is director of Industry Relations and Strategic Partnership, MJH Life Sciences.

Cellares CEO Fabian Gerlinghaus discusses the nuts and bolts of counterflow centrifugal elutriation and electroporation innovations and optimization.

Cellares Cell Shuttle is More Valuable Than the Sum of the Value of the Individual Parts

Standardizing workflows for system components and process steps is both a challenge and a goal for cell and gene therapies (CGT) scale up and commercialization. Counterflow centrifugal elutriation is employed for white blood cell enrichment and platelet removal. In addition, Cellares is using this technology for concentration steps and buffer exchanges. Gerlinghaus believes the key to appreciating the importance of their counterflow centralfugal elutriation feature is that it is but one of many technologies integrated into a fully-closed, fully-automated cartridge. The sum of all of those technologies put together in a closed and automated system is much more valuable than the sum of the parts; the whole system is worth more than the value of the individual technologies. Simply, automation solves the cost problem and closing the process solves the contamination problem. All of these technologies in and of themselves had to be at a similar efficacy level as technologies used in the current market, so Cellares generated a data set to show that that is the case. Gerlinghaus pointed out that in many cases, Cellares’ implementation of these technologies actually outperforms what's out there in the market; its electroporator achieves higher electroporation efficiency than many of the top electroporators used by professionals in the field. This can be seen as an underappreciated strength of Cellares’ technology platform.

Analytically speaking, it’s important to understand that the cell shuttle system handles purely the manufacturing of a cell type product; it does not handle release testing of the cell therapy products that come out of the platform. Gerlinghaus hinted, however, that there's a much bigger story coming soon. During the actual manufacturing process that is conducted on the cell shuttle, Cellares’ team does a lot of in-process testing. Cellares has integrated a fully automated biochemistry analyzer that can run up to 16 different assays on a tiny sample. It provides cell count, density, viability, and metabolite and nutrient data and it's fully integrated fluidically, mechanically, and from a software perspective. Gerlinghaus outlined the cell shuttle process: technicians decide ahead of time at which frequency they want the cell shuttle to take a sample and which assays they want that platform to run on the cell shuttle. Then, with the support of offline assays, the cell shuttle can take a sample, a human can pick it up, and the sample can be analyzed on any other third-party instrument. In future iterations of the cell shuttle, Cellares plans to go through an entire design-build test cycle roughly every year and a half. Moving forward, the company aims to integrate even more in-process analytical capabilities into the cell shuttle, promising that there is yet more to come.

Cellares CEO Fabian Gerlinghaus discusses the nuts and bolts of counterflow centrifugal elutriation and electroporation innovations and optimization, while hinting at big news on future release testing of products, as a product itself.

Featured Content

Pharmaceutical Technology Is Now PharmTech: A Sharper Name for a Smarter Industry

Behind the Headlines Episode 28

Ask the Expert

Drug Digest December 2025

A “Plan, Prevent, Prove” Approach