Behind the Headlines Episode 27

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Small Molecules, APIs, and Excipients—Trends, Challenges, and Opportunities

Drug Digest September 2025

Artificial intelligence, machine learning, and novel instruments are helping drug developers.

Enhancing Bioanalysis and Drug Development with Advanced Tools and Technologies

J.P. Morgan Report Lists Biologics, Obesity, China

Top Trends Reshaping Pharma R&D

Feliza Mirasol is the science editor for 

On May 15, 2023, BiOneCure Therapeutics, a US-based clinical-stage biopharmaceutical company focused on developing antibody-drug conjugates (ADCs), announced that it has entered into a strategic collaboration with China- based Nanjing Leads Biolabs, a clinical-stage biopharmaceutical company focused on second-generation immuno-oncology therapeutics, to develop a range of innovative ADCs for treating solid tumors.

The companies intend to leverage their complementary technologies in the collaboration, which they expect will strengthen their R&D and product development capabilities. BiOneCure’s technology includes several proprietary ADC payload-linker platforms. Its first product, BIO-106, is an anti-Trop-2 ADC that is currently in a Phase I clinical study. Leads Biolabs’ portfolio holds more than 20 novel mono- or bispecific antibody drug projects to fulfill unmet medical needs, according to a company press release.

"We are excited to collaborate with Leads Biolabs to develop ADC drugs," said Haifeng Bao, CEO of BiOneCure, in the press release. "Through this collaboration, we aim to accelerate innovation and development in the ADC field and provide better treatment options for patients."

"We believe that BiOneCure has leading ADC technology platforms, and [its] ADC molecule is in the clinical stage," said Xiaoqiang Kang, founder and chairman of Leads Biolabs, in the release. "Leads Biolabs is a clinical-stage antibody new drug company, with multiple antibody technology platforms. We are convinced that this collaboration will greatly expand the innovative R&D capabilities of both parties."

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Under the collaboration, BiOneCure Therapeutics and Nanjing Leads Biolabs intend to develop a range of innovative antibody-drug conjugates for treating solid tumors.

BiOneCure Therapeutics and Nanjing Leads Biolabs Partner to Develop ADCs for Solid Tumors

Synthego, a US-based provider of genome engineering solutions, has opened its new good manufacturing practice (GMP) synthesis facility. According to a May 11, 2023 company press release, the 18,000-ft

 facility will support the growing demand for the development of high-quality, clustered regularly interspaced short palindromic repeats (CRISPR)-enabled 

 therapeutics. Services at the new facility will also include investigational new drug-, new drug application-, and biologics license application-enabling clinical studies.

The new facility features multiple multiplexed manufacturing lines, fully automated controls, and an integrated manufacturing execution system. The company will have the capability to produce a range of oligonucleotides, including synthetic single guide RNAs. The facility also includes dedicated process development, analytical testing, and quality control areas, which will ensure that all aspects of the manufacturing process are closely controlled. The facility meets the requirements of both domestic and international regulatory authorities including FDA, the European Medicines Agency, the Medicines and Healthcare products Regulatory Agency, and Health Canada, according to the press release.

“This facility will be essential in supporting the burgeoning field of clinical phase, first-in-human, CRISPR-enabled medicines both for more common genetic diseases but also for rare diseases affecting children for whom other treatments do not exist,” said Adrian Thrasher, a clinician and professor of Pediatric Immunology at University College London Great Ormond Street Institute of Child Health, in the press release. Thrasher’s laboratory is developing new CRISPR-enabled medicines for children with rare genetic diseases.

“As we move into this new generation of cell and gene therapies, it will be crucial that there are strong, reliable manufacturing capabilities that exist for the necessary components of the CRISPR technology—such as the guide RNA—in order to accelerate the path to clinic and treat patients," Thrasher said in the release.

“Synthego pioneered the mass production of guide RNAs for discovery research that has enabled many of the CRISPR-based cell and gene therapy trials happening today. Having been a key leader in providing the technology for researchers and clinicians, it follows that they are now expanding their capacity to support this research as it translates into the clinic,” said Don Kohn, distinguished professor and clinician of Pediatric Hematology and Oncology at University of California, Los Angeles. Kohn’s laboratory pioneered genetic therapies for immunocompromised children.

Synthego has opened its new GMP-compliant RNA manufacturing facility in Redwood City, Calif., for CRISPR-enabled genomics therapeutics.

Synthego Launches New RNA Manufacturing Facility

Data from a Phase I clinical trial of BioNTech and Roche’s personalized cancer vaccine candidate, adjuvant autogene cevumeran, was published in 

 on May 10, 2023. The study evaluated the vaccine’s ability to fight pancreatic ductal adenocarcinoma (PDAC), an aggressive form of pancreatic cancer that the study noted is the third leading cause of death from cancer in the United States and lethal in approximately 88% of patients.

The treatment is an individualized neoantigen vaccine based on uridine messenger RNA (mRNA)–lipoplex nanoparticles, which was developed using mRNA neoantigen vaccines in real time from surgically resected PDAC tumors. When treated with adjuvant autogene cevumeran, in combination with atezolizumab and mFOLFIRINOX, eight of the 16 patients developed T cells that could potentially fight PDAC. Vaccine-expanded T cells were durable, persisting up to two years after injection.

“High-magnitude vaccine-induced T-cell responses, the focus of our immune response analysis that included a new method to track vaccine-expanded clones, correlated with delayed PDAC recurrence,” reads the study. “Despite the limited sample size, these early results warrant larger studies of individualized mRNA neoantigen vaccines in PDAC.”

The data, published in the scientific journal Nature, demonstrated that half of patients developed T cells that could potentially fight pancreatic ductal adenocarcinoma.

BioNTech and Roche Publish Personalized Vaccine Data

Agilent released an announcement of its Agilent 8697 Headspace Sampler -XL Tray, which features 120-vial capacity, in addition to their existing 48-vial capacity tray. In addition to the capacity increase, the new tray includes new instrument intelligence features that are designed for increased uptime and operator ease-of-use relative to its predecessor.

According to a press release from May 1, 2023, the device’s intelligence features are enabled through self-monitoring operations and guiding operators that use various troubleshooting and maintenance procedures. The system performs automated system checks, including leak checks, early maintenance feedback, and self-diagnostics. Additionally, the new tray can be run with alternate carrier gasses due to the separation of vial pressurization and the sample loop for added method flexibility.

“Labs that run headspace GC [gas chromatography] systems know that if they encounter problems during a sequence, and the vials are punctured, they may have to rerun or recollect samples,” said Mike Zhang, vice-president and general manager of Agilent’s Gas Phase Separations Division. “The 8697 and now the 8697 Headspace Sampler-XL Tray virtually eliminate that problem. The complete GC headspace system checks to ensure that the system and data quality are up to SOP [standard operating procedure] standards, avoiding recollection of samples and reruns.”

The Agilent 8697 Headspace Sampler-XL Tray has a 120-vial capacity.

Agilent Releases New Vial Sampler Tray Capacity

TriLink Biotechnologies launched its new cap analog, CleanCap M6, on May 9, 2023, which is expected to help developers and researchers maximize the impact of their messenger RNA (mRNA) therapeutics and vaccines while reducing overall manufacturing costs, according to a company press release. The introduction of CleanCap M6 technology is intended to improve mRNA potency with the highest protein expression of any CleanCap analog to date.

“TriLink’s portfolio of CleanCap mRNA capping technology is expected to be the new go-to capping method in the very near future—it is the future,” said Kate Broderick PhD, chief innovation officer at Maravai LifeSciences, in a press release. “What excites me most about this novel analog is its ability to make mRNA an even more powerful therapeutic than we have seen previously.”

The first CleanCap analog was launched in 2017, and since then, the capping technology has been incorporated in one of the first commercially approved COVID-19 vaccines.

“mRNA is clearly an emerging therapeutic modality where mRNA vaccines and therapeutics are expanding rapidly,” said Drew Burch, EVP of TriLink’s Nucleic Acid Products division, in a press release. “CleanCap M6, and the entire CleanCap portfolio, joins a strong roster of synthesized nucleic acids, NTPs, and more that we expect to continue to grow.”

TriLink Biotechnologies Introduces CleanCap M6 Analog

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

Oral solid dosage (OSD) forms are a mainstay for the pharmaceutical industry, providing a convenient, cost-effective solution to drug delivery. The outlook for the OSD market remains positive with estimates of growth reaching a compound annual rate of 6.4% between 2022 and 2032 (1).

If you enjoyed this article, do listen to the podcast interview from the Pharmaceutical Technology Group, featuring Andy Dumelow from I Holland, discussing tablet tooling maintenance, available 

Within this market, tablets account for the largest share, and the segment is forecast to continue to expand during the aforementioned analyzed period. The manufacture of tablets, although cost effective (particularly at scale), still requires specialist equipment and tools, which, if not maintained correctly, can cause defects in the finished product and ultimately harm the manufacturer’s bottom line.

Pharmaceutical Technology's May 2023 Trends in Manufacturing eBook

Keeping Up with Tablet Tooling Maintenance

Felicity Thomas, European/senior editor, Pharmaceutical Technology Group

When referring to this article, please cite it as Thomas, F. Keeping Up with Tablet Tooling Maintenance. 

Pharmaceutical Technology's Trends in Manufacturing eBook 

A good understanding of tablet tooling and how to properly maintain these key parts will maximize lifespan of tools.

Jeong Jin-hyeok is a lead specialist in drug product at Samsung Biologics.

To protect patient safety, drug developers and manufacturers must consider every possible route of contamination of the drug substance (DS) or drug product (DP) through all stages of production. Aseptic processes must be employed from the filling lines, containers, and stoppers to the air in the surrounding manufacturing space to maintain a sterile environment throughout fill/finish. This article explores the aseptic processing strategies biologics developers can integrate to safeguard DS and DP and examines the monitoring tactics helpful in maintaining a sterile environment.

Jeong Jin-hyeok is principal scientist in drug product at Samsung Biologics.

When referring to this article, please cite it as Jin-hyeok, J. Aseptic Processes Safeguard Biologics. 

Aseptic techniques must be practiced throughout all stages of biologics production.

Aseptic Processes Safeguard Biologics

Blake Willias is vice-president of Operations at G-CON Manufacturing.

Dennis Power is vice-president of Business Development at G-CON Manufacturing.

Cleanroom or classified environmental infrastructures have been designed and constructed for decades (1) and can be either on-site framed epoxy coated gypsum wall (stick-built), on-site assembled modular wall panels (modular), or off-site prefabricated cleanroom units (podular), the last of which is a relatively recent addition. All of these cleanroom material and design modalities have benefits and limitations that require careful review when making decisions on which approach to use for constructing current good manufacturing practice (CGMP) cleanroom infrastructure. Such decisions need to be made not only with the process application needs in mind, but also with the long-term perspective on future capability and capacity. This is especially important for multi-modal or multi-product processing streams and new therapies, which may not have a clearly defined process design or require process modifications along the way, and contract manufacturers, as their client base require process modalities change.

In all cases, the need for flexibility is real (2,3,4). However, flexibility must be defined, as it is often used in general terms (5). Some claim that standardization of cleanroom infrastructure designs, faster deliveries, and lower cost of goods sold, will impede or prevent flexibility. This generalization may be inaccurate, as smaller volumes require smaller process footprints, which can be reconfigured within the available standardized space (6). Such spaces may be even more flexible if units can be scaled rapidly.

To truly determine flexibility, one needs to dissect the different flexibility needs and the available solutions. Only then can an appropriate decision be made, which will not just satisfy the short-term requirements, but also show long-term benefits.

M.W. Jornitz is is Prinicipal Consultant at BioProcess Resources LLC. Sindey Blackstrom is vice-president of Business Management, Blake Williams vice-president of Operations, and Denniss Powers vice-president of Business Development, all at G-CON Manufacturing.

When referring to this article, please cite it as Jornitz, M.W. et al. The Quest for Cleanroom Flexibility. 

Contextualizing what type of flexibility is needed is paramount when considering cleanroom design.

The Quest for Cleanroom Flexibility

Jennifer Markarian is a contributing editor for 

Pharmaceutical manufacturing equipment companies and technology providers are exploring how artificial intelligence (AI) can benefit process control systems. Two Italian companies—pharmaceutical machinery manufacturer Luperini and technology start-up Eigengran—partnered to launch AI-enabled equipment for oral solid dosage drugs that they have called Cognitive Pharma Machinery.

Luperini signed a licensing agreement to use Eigengran’s cognitive control technology in December 2022, following 18 months of testing and development. The initial work focused on high-shear wet-granulation (HSWG). In one-pot HSWG equipment, such as that made by Luperini, the entire granulation process, including the drying step, is performed within one unit. This set-up was well-suited for the initial project of developing a cognitive control system using process analytical technology (PAT) sensors to monitor the progression of the granulation process over time.

AI Algorithms Find Use in Pharma Equipment

“Our technology is a key element in the application of PAT guidelines for oral drug production and will be a key step towards achieving Pharma 4.0 practice in the pharmaceutical manufacturing process,” said Marco Luperini, chief technology officer of Luperini, in an announcement of the technology (1). Luperini’s new test center in Chiesina Uzzanese, Italy houses pilot machines for evaluation to simulate conditions that may be encountered in real-world scenarios.

Jennifer Markarian is manufacturing reporter for 

When referring to this article, please cite it as Markarian, J. AI Algorithms Find Use in Pharma Equipment. 

Pharmaceutical Technology Trends in Manufacturing

Machine learning provides real-time data for process control of high-shear wet granulation.

Life Biosciences, a biotechnology company, and Forge Biologics, a genetic medicines manufacturing organization, announced on May 8, 2023 that they are in a manufacturing partnership to help advance Life Bio’s partial epigenetic reprogramming platform to further address aging-related diseases, according to a press release.

“Life Bio is an emerging leader in the development of novel therapies for aging-related diseases, and we are thrilled to serve as their cGMP manufacturing partner to help advance the manufacturing of AAV for their innovative cellular rejuvenation technology, which has the potential to benefit millions of aging patients worldwide,” said Timothy J. Miller, PhD, CEO, President, and Co-Founder of Forge, in a press release.

Forge will provide adeno-associated virus (AAV) process development, toxicology, cGMP manufacturing, and analytical services to Life Bio, which will occur at Forge’s gene therapy facility in Columbus, Ohio called the Hearth.

“We are delighted to be working with the Forge team, whose expertise in gene therapy manufacturing is unmatched,” said Jerry McLaughlin, Chief Executive Officer of Life Biosciences, in the press release. “We believe we’re on the cusp of revolutionizing medicine with our cellular rejuvenation capabilities across a range of aging-related diseases, including ophthalmic disorders that involve retinal ganglion cell dysfunction.We are confident our partnership with Forge will have a tremendous impact on our ability to enhance the speed and quality with which we can manufacture our therapeutic candidates as we progress toward the first human clinical trials and continue to develop treatments to reverse diseases of aging by restoring cells to a more youthful state.”

Featured Content

Behind the Headlines Episode 27

Ask the Expert

Drug Digest September 2025

Enhancing Bioanalysis and Drug Development with Advanced Tools and Technologies

Top Trends Reshaping Pharma R&D