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Dosage Forms: Roundtable on Oral Solid Dosage Trends

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From Theory to Reality: Automating Process Development

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The Importance of Appropriate Supplier Oversight

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Patrick Lavery is an Editor for the MJH Life Sciences brands 

. Previously at MJH, he filled the same role for 

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The Belgium-based contract development and manufacturing organization (CDMO) Ardena announced on Oct. 29, 2024 that it was making a “strategic investment” in two sites in the Netherlands, one of the countries in which the company maintains good manufacturing practice (GMP) facilities, along with Belgium, Spain, and Sweden (1). Ardena said the improvements were being made with an eye toward client demand for cutting-edge services in the field of bioanalytics.

Namely, the expansion of Ardena’s footprint in the Netherlands will include a brand-new bioanalytical laboratory at its Pivot Park facility, located in the city of Oss, as well as added capacity and new, good laboratory practice (GLP) capabilities at its Bioanalytical Center of Excellence in Assen, according to a press release (1). The enhancements in the two locations are meant to complement each other, the company said.

“This expansion allows us to quickly scale our operations, leverage an additional pool of talented professionals, and bring enhanced efficiency and value to our clients, from discovery to late-stage clinical phases,” Jeremie Trochu, Ardena CEO, said in the press release.

Ardena previously expanded its headquarters in Gent, Belgium, in 2018, at which point the company was operating in that country as well as the Netherlands, Sweden, and Latvia (2). At the time, Ardena said it was seeing an increase in organic growth of more than 20% per year, crediting its merger and acquisition strategy. Shifting forward to 2024, that strategy has most recently included the company acquiring an advanced drug product manufacturing facility in the United States from Catalent (1).

There was also, in 2021, the acquisition of Idifarma, a manufacturer of niche and highly potent generic and innovative drugs based in Pamplona, Spain, from Suanfarma (3). Ardena said its offerings are designed to serve clients from small innovators in the biotech realm to large pharma companies across all stages of drug development, and with respect to small molecules, therapeutic peptides and proteins, antibodies and antibody-drug conjugates, nanoparticles, oligonucleotides, messenger RNA (mRNA), and cell and gene therapies (1).

“Together, these investments demonstrate Ardena's focus on growth and innovation, reinforcing our strategy to assist clients navigate through an ever-increasing complex drug development process and bring life-changing therapies to patients worldwide faster,” Trochu said in the Oct. 29 press release.

The Oss laboratory, which Ardena said will be functional by the first quarter of 2025, covers 3000 sq ft for both small and large molecule analytical testing, complete with an automated technology platform, fluorometric detection assays, and liquid chromatography—tandem mass spectrometry (LC–MS/MS) capabilities (1). By contrast, although the Assen facility will also have its LC–MS/MS capacity increased and will add new automation systems, its focus will be more on immunochemistry, flow cytometry, and quantitative polymerase chain reaction platforms, according to Ardena.

1. Ardena. Ardena Expands Bioanalytical Services in Europe. Press Release. Oct. 29, 2024.
2. Ardena. Ardena Cements Growth and Expansion with New HQ. Press Release. Nov. 8, 2018.
3. Ardena. Ardena Acquires Idifarma from Suanfarma. Press Release. Sept. 1, 2021.

Articles

A new laboratory will be built in Oss, while additional capacity and capabilities will be added to the Bioanalytical Center of Excellence in Assen.

Ardena Expands Bioanalytical Services in the Netherlands

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to highlight best practices for preparing for 2025.

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Alec McChesney (BD Director, SCORR Marketing) discusses goal setting and alignment between life sciences marketing and business development teams.

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Feliza Mirasol is the science editor for 

ViaNautis Bio (ViaNautis), a UK-based biotechnology company specializing in delivering targeted genetic nanomedicines, announced on Oct. 28, 2024 that it has entered a collaboration agreement with Eli Lilly and Company (Lilly) in which Lilly will use ViaNautis’ proprietary polyNaut platform to develop precision therapeutics carrying genetic cargos. The collaboration aims to precisely deliver these therapeutics to prioritized tissues to address significant unmet medical needs.

Under the agreement, Lilly will pay ViaNautis an initial upfront payment with the short-term potential of making additional payments, including research-based milestones. ViaNautis is eligible to receive further milestones and royalties contingent upon specific cargo-loaded polyNaut-based products entering clinical development and reaching and reaching commercialization.

“This collaboration with Lilly is a testament to our polyNaut platform, which is also driving a highly differentiated emerging pipeline for severe life-limiting diseases. Combining our scientific know-how to precisely deliver genetic nanomedicines targeting difficult-to-reach sites, with Lilly’s extensive drug development and commercialization expertise will accelerate the creation of novel therapies to improve patient outcomes,” said Francesca Crawford, PhD, co-founder of ViaNautis, in a company press release.

The company’s proprietary polyNaut nanovesicles are designed to precisely target specific tissues and cell types to deliver genetic materials. The technology aims to improve the current delivery of genetic medicines and hold the potential to transform treatment for various diseases, according to the company in the press release.

“I am very excited about our collaboration with Lilly to develop novel genetic medicines for patients in need. I want to thank [Crawford] and the team for their hard work in getting to this key milestone, and I look forward to working with Lilly to accelerate our shared vision of delivering targeted genetic medicines,” said Adi Hoess, MD, PhD, chief executive officer of ViaNautis, in the release. The company is currently working on a pipeline of therapeutics in the central nervous system and cystic fibrosis disease fields (1).

Nanovesicles are being researched as alternatives to extracellular vesicles (EVs) for various applications, including drug delivery. As mediators of intercellular communication, EVs have gained growing interest from researchers for their potential use in the field of regenerative medicines or as a drug delivery vehicle. However, because EVs are derived from stem cells or progenitor cells, their use is limited primarily due to a lack of efficient and scalable production methods (2).

As a result, researchers are turning to nanovesicles, particularly biofabricated cell-derived nanovesicles, as an alternative to EVs that are offer scalable, efficient, and cost-effective production (2). Meanwhile, further research has been underway to develop and test simplified and rapid production methods for cell-derived nanovesicles that offer advantages over traditional methods in terms of yield and cost-effectiveness (3), which are anticipated to offer new approaches to therapeutic drug delivery.

 (accessed Oct. 30, 2024).
2. Ilahibaks, N. F.; Lei, Z.; Mol, E. A.; et al. Biofabrication of Cell-Derived Nanovesicles: A Potential Alternative to Extracellular Vesicles for Regenerative Medicine. 

 (12),1509. DOI: 10.3390/cells8121509
3. Goh, W. J.; Zou, S.; Ong, W. Y.; et al. Bioinspired Cell-Derived Nanovesicles versus Exosomes as Drug Delivery Systems: a Cost-Effective Alternative. 

The partnership will use ViaNautis’ proprietary polyNaut technology platform to develop genetic medicines that can precisely target tissue types.

Lilly and ViaNautis Bio Partner on Development of Novel Therapeutics Delivering Genetic Cargo

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Abzena, an end-to-end contract development and manufacturing organization, is expanding its quality control testing capabilities at its San Diego, Calif., biologics development and current good manufacturing practice site, the company announced on Oct. 30, 2024. The expansion is part of the company’s strategy to meet market demand for quality testing. According to Abzena, the additional laboratory space will enhance its existing analytical toolkit for antibodies with microbiology release testing. The expansion will also improve scalability with advanced materials separation.

The standalone microbiology lab offers separate product testing from other areas of the facility, in alignment with industry and regulatory standards. Product microbiological testing is segregated from utility and environmental microbiological testing in order to minimize cross-contamination. Production capacity is optimized by the separation of high volumes of clinical materials, which results in yields up to 99%, according to the company. “This design methodology offers superior operational efficiency and improved scalability while minimizing overall ecological impact,” the company stated in a press release (1).

“This announcement comes at a very timely moment with World Quality Month quickly approaching. As an organization focused on quality excellence, increasing our QC [quality control] footprint is a testament to our commitment to continuous improvement and innovation,” said Troy Wright, senior vice-president and global head of Quality at Abzena, in the release. “This expansion provides even greater sample integrity in the management of our microbiological testing capabilities. We are now positioned to deliver faster results and better support for our customer network, ultimately providing quality and compliant products to patients around the world.”

In other Abzena news, the company announced on Sept. 26, 2024 that its mammalian cell line development platform, AbZelectPRO, reduces timelines by more than 30% and improves overall protein production levels. The platform can bridge from DNA to research cell bank in 10 weeks and produces up to 8 g/L of high-performing titres before process development, according to the company (2).

Abzena recently attended CPHI Milan in October 2024, and 

caught up with Nicolas Camper, senior director—Bioconjugation Chemistry at Abzena, to talk about advancements in conjugation and linker technologies in antibody-drug conjugate (ADC) development and manufacturing (3).

“There have been a lot of new developments in terms of conjugation and linker technologies. There is a definitive move towards site-specific conjugation technologies and away from stochastic conjugation approaches that allows [us] to prepare better-defined antibiotic-[drug] conjugates,” Camper said in the interview.

Another new development that has been observed over the past few years is that linkers are becoming more sophisticated. “Quite often, when you conjugate cytotoxic drugs, they are quite hydrophobic, and that can impact the performance of your ADC. So, there has been a lot of effort put into developing more hydrophilic linkers,” Camper noted.

Abzena. Abzena Announces Major Expansion in Microbiology Laboratory Space in Suppor of Biologic Manufacturing. Press Release. Oct. 30, 2024.

Abzena. AbZelectPRO Enables Biopharma Customers to Reach their Research Cell Bank in 10 weeks with 8g/L. Press Release. Sept. 26, 2024.

Mirasol, F. CPHI Milan 2024: New Developments in ADC Technologies. 

The company will be expanding its quality control testing capabilities at its San Diego, Calif. biologics development and manufacturing site.

Abzena Expands Microbiology Laboratory Space

Dosage forms, in particular oral solid dose, continue to evolve as new technologies enter the market, and as customer tastes and demands change. In this episode of the Drug Solutions podcast, Patrick Lavery, editor for 

®, surveys attendees from the recent CPHI Milan gathering about how fast the market is expanding, how it's evolved in recent years and even decades, and what drugs are most conducive to OSD formulations.

 is vice president, drug product division, 

 Her CPHI interview can be viewed in full 

 is head of global pharmaceutical sciences, 

 His CPHI interview can be viewed in full 

 is head of product implementation, oral solid dose, Recipharm. His CPHI interview can be viewed in full 

 is vice president and chief innovation officer, Colorcon. His CPHI interview can be viewed in full 

 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 podcast episode, we discuss trends in dosage forms, specifically oral solid dosage, as viewed by five industry experts who recently exhibited at CPHI Milan.

Drug Solutions Podcast: Dosage Forms: Roundtable on Oral Solid Dosage Trends

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

Marianna Tcherpakov, PhD, director of Business Development, CMC Services, at Frontage Laboratories, spoke on the CDMO’s role in tackling complex technical challenges to enable startup biotechs solve scale up problems.

BIO 2024: CDMOs Focus on Complex Technical Challenges to Boost Startup Biotechs

Contract development and manufacturing organization (CDMO) Frontage Laboratories founder Song Li, PhD, who currently holds the position of executive chairman, board of directors, at the company, first set up a testing laboratory in Thorofare, NJ, with a focus on analytical and bioanalytical testing that quickly established a reputation for expertise. That foundation of expertise seems to have stayed fairly well intact, and science—and scientists—driving projects forward appear to be the key selling point for this CDMO, which says of itself, “our scientific knowledge base, technical expertise and reputation for high quality services have been integral to our ability in turning our services into your solutions” (1). In speaking with the company’s staff at BIO 2024, Pharmaceutical Technology Group’s Chris Spivey discerned that the practical upshot for the company has been that Frontage Laboratories often attracts the more vexing or technically challenging projects, and as a result, the company’s teams are now well situated to deal with the more difficult of perplexing projects for which others may not be as well prepared. As an example of the company’s preparedness, it states on the resources section of its website that it uses a next-generation sequencing (NGS)-based method, called the Frontage PGx NGS Test, to rapidly determine the genotype of 300+ genes involved in absorption, distribution, metabolism, and excretion (2).

Talking with Spivey at BIO, Marianna Tcherpakov, PhD, director of Business Development, CMC Services, Frontage Laboratories, at BIO, outlined the general approach the company takes when working with clients. She emphasized that the company’s approach is always customizable and personalized to not only the science the client requires but also to their specific strengths and weaknesses as an organization. “We look at your project as our own product. We therefore offer fast delivery, fast responses to client questions, almost as if we are holding hands … Many come from smaller startups that need a lot of guidance. [This] can be technical, it might be regulatory, but it all relies on our very strong scientific base,” Tcherpakov said. “We are passionate about technology,” she added.

Many of the PhD staff at Frontage Laboratories have come from other companies in the industry, and Tcherpakov pointed out that these past experiences and learnings are often brough to bear on the current project challenge. “We understand how stressful it can be for the smaller biotech, and even middle-sized [biotechs], when you have a lot of deadlines. You have clinical trials submissions coming up, and you need to be on top of everything, and fast,” she said.

Touting their scientific backbone Frontage Laboratories advertises itself as “more than a CRO”, in that it is also a “full global pharmaceutical development organization. Frontage Laboratories offers a comprehensive suite of drug discovery and development services including chemistry, DMPK [drug metabolism and pharmacokinetics], preclinical safety/toxicology, and clinical trial services with strategic and regulatory support to effectively bring new lead candidates through the IND [investigational new drug] stage to Phase II” (3).

BIO 2024 was held on June 3–6 in San Diego, Calif.

1. Frontage Laboratories. Services & Solutions. 

 (accessed August 21, 2024).
2. Frontage Laboratories. Resource Library. 

 (accessed August 21, 2024).
3. Frontage Laboratories. About Frontage. 

Marianna Tcherpakov, PhD, is the director of Business Development, CMC Services, at Frontage Laboratories. Image courtesy of Frontage Laboratories.

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The World Health Organization (WHO) announced on Oct. 29, 2024 that it has activated the Global Health Emergency Corp (GHEC) in collaboration with Member States and other partners in response to mpox outbreaks (1). This is the first time GHEC, which was established by WHO in 2023 as a response to COVID-19, has been activated to support countries dealing with mpox. Leaders from affected and other countries met on October 22 as part of the GHEC activation to discuss effect control measures, best practices, and the coordination of efforts.

Mpox was declared as a public health emergency by WHO in August of 2024 (2). “The emergence of a new clade of mpox, its rapid spread in eastern DRC [Democratic Republic of the Congo], and the reporting of cases in several neighboring countries are very worrying. On top of outbreaks of other mpox clades in DRC and other countries in Africa, it’s clear that a coordinated international response is needed to stop these outbreaks and save lives,” WHO Director-General Tedros Adhanom Ghebreyesus, PhD, said at the time of the announcement (2).

“WHO and partners are supporting the government of the Democratic Republic of the Congo and other countries to implement an integrated approach to case detection, contact tracing, targeted vaccination, clinical and home care, infection prevention and control, community engagement and mobilization, and specialized logistical support,” said Mike Ryan, MD, executive director of WHO’s Health Emergencies Programme, in the October 29 press release (1). “The GHEC enhances the ability of the many effective responders at national and regional levels to collaborate and ensure the success on the ground in interrupting transmission and reducing suffering.”

GHEC’s objective is to strengthen the response to health emergencies and provide a platform in which countries and health networks can collaborate the response, including deployment of experts and leaders. GHEC is assessing workforce capacities in eight countries including DRC and Burundi, which have been the most impacted by the mpox outbreak. There have been 22 areas identified by GHEC that are in need of epidemiology, surveillance, laboratory capacity, infection prevention, risk communication, and community engagement. WHO has deployed 55 experts to the impacted countries, as of October 17, that include WHO staff through the Global Outbreak Alert and Response Network (GOARN) and the African Volunteers Health Corps. GOARN is also mapping partner support on a bilateral basis to impacted countries and the coordination structure in the region, including providing experts, supplies, financial support, and strengthening capacity.

“By mobilizing trained professionals from within the continent, we ensure that responses are not only timely but also contextually relevant,” said Abdou Salam Gueye, MD, PhD, regional emergency director for the WHO Regional Office for Africa, in the press release (1). “The dedication and expertise of these responders are essential in saving lives and building resilient health systems capable of withstanding future threats.”

In other mpox news, a report based on outcomes of a workshop held on Oct. 2, 2024 by the International Coalition of Medicines Regulatory Authorities was announced by the European Medicines Agency on Oct. 25, 2024. The current epidemiological status and the availability of mpox vaccines in African countries was discussed during the workshop. Discussion at the workshop was focused on the prioritization of treatments for children because children are the most vulnerable group in the DRC and have the highest fatality ratio (3).

WHO. WHO and Partners Activate Global Health Emergency Corps for the First Time in Response to Mpox Outbreak. Press Release. Oct. 29, 2024.

WHO. WHO Director-General Declares Mpox Outbreak a Public Health Emergency of International Concern. Press Release. Aug. 14, 2024.

EMA. Fostering Regulatory Collaboration to Improve Access to Mpox Medicines. Press Release. Oct. 25, 2024.

WHO and its partners are providing support to countries dealing with mpox outbreaks.

WHO Activates Global Health Emergency Corp in Response to Mpox

AAPS PharmSci 360 2024: Big Data and Variation in Excipient Production

As part of its AAPS PharmSci 360 2024 coverage, 

 spoke with Pauline Janssen, global technical lead and product development manager at DFE Pharma, about her presentation at the conference, “Using Big Data Analysis to Better Characterize Excipient Variability,” delivered on Tuesday, Oct. 22, 2024.

Janssen said there is inevitable variation in all production processes, including excipient production, and big data sets can help—but especially when those analyzing the data are subject matter experts.

“We believe it's important to understand, what is the consistency? Which [data can] you look at [for] specific parameters, for example, inconsistency documents where you can see what is the typical variation that can be expected in a certain parameter?” Janssen said in the interview. “But a challenge when you want to evaluate multiple excipients, maybe multiple parameters, is actually all the potential interrelationships between those parameters. If you want to do a full evaluation of all these parameters and maybe perform a full design of experiments, you would require a huge amount of resources. And actually, this is also one of the key concepts in the QBD [quality by design] approach that allows [us] to identify what is the typical variation and what is the relevant variation in a large data set.”

Click the above video to watch the full interview.

AAPS PharmSci 360 was held from Oct. 20–23, 2024 in Salt Lake City, Utah. Find the rest of our coverage 

, and more information can be found on DFE Pharma's 

Pauline Janssen of DFE Pharma gave a presentation on the utility of analyzing big data sets to better understand and diagnose variability in the production process.

Monte Rosa Therapeutics, a Boston, Mass.-headquartered clinical-stage biotechnology company specializing in novel molecular glue degrader (MGD)-based medicines, announced on Oct. 28, 2024,  that it entered into a global exclusive development and commercialization license agreement with Novartis. The agreement is worth potentially up to $2.1 billion and aims to advance vav guanine nucleotide exchange factor 1 (VAV1) MGDs, including MRT-6160, a drug candidate being evaluated in an ongoing Phase I single ascending dose (SAD)/multiple ascending dose (MAD) healthy volunteer study for immune-mediated conditions.

Under the agreement, Novartis will pay Monte Rosa $150 million up front in exchange for exclusive worldwide rights to develop, manufacture, and commercialize MRT-6160 and other VAV1 MGDs. Monte Rosa is eligible to receive up to $2.1 billion in development, regulatory, and sales milestones, starting with the initiation of Phase II clinical studies of MRT-6160. Monte Rosa is also eligible to receive tiered royalties on ex-US net sales.

Novartis will be responsible for all clinical development and commercialization of MRT-6160, starting at the Phase II clinical phase. Monte Rosa, meanwhile, will remain responsible for completing the ongoing Phase I clinical study for the drug candidate. In addition, Monte Rosa will co-fund any Phase III clinical development. The company will also share any profits and losses associated with the manufacturing and commercialization of MRT-6160 in the United States.

“We are thrilled to announce this agreement with Novartis, a key player in immune-mediated conditions, and we are excited about the transformative potential it provides for Monte Rosa and MRT-6160. We expect this will accelerate and broaden the scope of clinical development of MRT-6160 to advance this unique, orally bioavailable modality while retaining substantial value for Monte Rosa. We believe the transaction validates our unique and industry leading QuEEN discovery engine, and it further increases our conviction to rationally design and develop highly selective and safe MGDs for undruggable targets, including in the areas of immunology and inflammation, metabolism, and genetic diseases,” said Markus Warmuth, MD, chief executive officer, Monte Rosa Therapeutics, in a company press release. “The financial resources provided by this agreement are expected to extend our operational runway, enable us to advance our pipeline to potential value-creating milestones and anticipated proof-of-concept readouts, and further leverage our QuEEN discovery engine.”

MRT-6160 is a potent, highly selective, and orally bioavailable investigational degrader of VAV1, which is known as a key signaling protein found downstream of both T- and B-cell receptors. According to preclinical studies, MRT-6160 demonstrated deep degradation of VAV1, which resulted in a significant decrease in cytokines linked to immune-mediated conditions. It also showed no detectable effects on other proteins. The candidate has shown promising activity in preclinical models of multiple immune-mediated conditions (1,2).

"Novartis has had a long-standing interest in molecular glue degraders, which offer the potential to tackle challenging biological targets. We are excited about their application in immunology and the early progress we have seen by Monte Rosa in this space and with MRT-6160. We look forward to advancing MRT-6160 and learning more about its potential to provide a new therapeutic option for people living with a range of immune-mediated conditions,” said Fiona Marshall, president, Biomedical Research, Novartis, in the press release. “Novartis is committed to bringing forward new therapeutic options for these patients, and we are happy to be working with Monte Rosa to harness the potential of this approach to address unmet medical needs.”

1. Cartwright, A.; Desai, F.; Vora, S.; et al. MRT-6160, a VAV1-Directed Molecular Glue Degrader, Reduces Joint Inflammation and Autoantibody Production in a Collagen-Induced Arthritis Autoimmune Disease Model. Poster presented at Digestive Disease Week 2024, Washington, DC, May 21, 2024.
2.Cartwright, A.; Vora, S.; Gyger, L.; et al. MRT-6160, a VAV1-Directed Molecular Glue Degrader, Inhibits Disease Progression in a T-cell Transfer Mediated Colitis Model Concomitant with Reduced Calprotectin Expression. Poster presented at EULAR 2024—Annual European Congress of Rheumatology, Vienna, Austria, June 14, 2024.

The collaboration aims to advance clinical development of a Phase I drug candidate, MRT-6160, and to explore other therapeutic opportunities across multiple indications.

Novartis and Monte Rosa Therapeutics Form $2.1 Billion Global License Agreement for MGD-based Therapeutics

Emerging therapies and new modalities, such as cell and gene therapies (CGTs), have developed fast in the past decade. This ever-evolving development of not only new formulations but also new delivery methods has created an ever-complicated industry. Technology transfer is a critical step in drug manufacturing, specifically when contract manufacturers are involved.

 an efficient technology process ensures transferrable processes. “A well-defined and robust technology transfer process involves a systematic approach to transferring manufacturing processes, protocols, and knowledge from the research and development phase to the clinical and potentially commercial manufacturing setting” (1). “This process ensures the developed cell and/or gene therapy can be produced consistently, efficiently, and in compliance with regulatory requirements. The same requirements exist for transferring analytical methods to contract development and manufacturing organizations (CDMOs) or equivalent” (1).

“Tech transfer becomes more complex with emerging therapies and new modalities, particularly in method transfer, as these therapies are often derived from living cells, making consistency challenging to maintain,” says Satish Shetty, senior director, Product Development & Project Management, Halo Pharma (part of the Noramco Group). “Additionally, the manufacturing processes are highly intricate and rely on specific environmental and process conditions, which further complicates the transfer.”

Ahmed Youssef, senior manager USP, Process Development at Ascend, points to timing as being key for technology transfer with emerging therapies. “It takes an innovator around 12 to 24 months to make a product ready, tech transfer it, [and] complete the safety studies for it,” says Youssef. “But there could be other solutions that could be offered in the background that could make this more efficient, faster, and more attractive to product developers.”

Planning for technology transfer is essential, according to Hewitt 

 A smooth transfer requires careful evaluation, strategic decision making, and planning (1). Risk assessments are also crucial at this stage. “By proactively addressing these risks and implementing mitigation strategies during the initial assessment and planning stage, organizations can enhance the efficiency, reliability, and safety of the transfers” (1).

“A continuous feedback loop also encourages ongoing learning, incorporating insights from manufacturing teams into refining protocols. Regulatory alignment ensures compliance and adherence to guidelines. In essence, successful knowledge transfer ensures that the innovation harnessed in research is translated effectively, leading to consistent, high-quality outcomes in CGT manufacturing and accelerating the availability of transformative treatments to patients in need” (1).

Patient-centric therapies that might require small-batch manufacturing require flexible approaches to technology transfer, according to Andréa Simard, manager, Technical Services, Halo Pharma (part of the Noramco Group). This is particularly true for CDMOs that usually perform large-scale manufacturing. “Equipment train must be adjustable, and in-process control strategies should ensure process capability while meeting clinical or commercial needs,” Simard says. “Quality must be designed into the product through good knowledge of input materials and a robust manufacturing process, so that endpoint confirmation does not require extensive sampling and testing, which can greatly reduce the units available for the patients.”

However, Youssef remarks that tech transfer is a similar process no matter what batch size is involved. “Normally, when you tech transfer, you need to take risk-based approaches [and] appropriate measures to be able to tech transfer [a] process successfully and properly,” he says.

Brianna Kempfer, project manager at Sterling Pharma Solutions agrees. “The process of tech transfer is much the same, no matter the scale; a CDMO must evaluate the information provided: assessing any potential hazards, the scientific feasibility, and qualify the necessary synthetic and analytical methods. Through laboratory scale work, chemistry can be optimised and demonstrated to understand which parameters of the process are the most critical when scaling up and manufacturing.”

The smaller scale of patient-centric medicine production offers options for process development such as reagent and solvent choice, according to Kempfer. “Because of the smaller volumes of manufacturing, the costs of goods are usually higher, and so development of an efficient process that minimiszs potential losses in purification and isolation steps is crucial.”

One must understand technologies involved in formulation, as well, stresses Simard. “For example, formulation must consider patient-specific factors such as taste masking for bitter drugs or ease of administration for pediatric and geriatric populations,” she says. “Additionally, specialty packaging plays a crucial role in simplifying complex dosing regimens, making it easier for patients to adhere to treatment plans.”

Many bio/pharma outsourcing organizations have been moving to become full-service, end-to-end CDMOs. But does working with a contractor who offers services from every step of a drug’s lifecycle improve technology transfer. It depends, asserts Youssef. “If you mean by end to end, the whole process, from the start of plasmid manufacturing till commercial manufacturing of the batches and then the fill and finish and release, understand that the advantage would be that you're running all of that under one MSA [master supply agreement], one big MSA. But know that, not all CDMOs are expert and skilled in each unit operation when they offer the end-to-end process,” says Youssef.

Shetty points out that there are several reasons why tech transfer may be less complicated with an end-to-end service provider. “[A] unified sending unit (SU) and receiving unit (RU) enable seamless knowledge transfer. Tech transfers are often complicated by development knowledge getting lost through site transfers, and delays from having to set up project logistics at a new CDMO, so having both teams in the same organization is a solid advantage.”

End-to-end service providers often provide consistency in the equipment used between the different phases of development and manufacture, notes Shetty. There is also often the same project manager and core team involved, as well as the same excipient grades and reduced supply lead times. End-to-end providers also feature “fewer knowledge gaps and better identification of potential scale-up issues,” according to Shetty. “Consolidating R&D and supply chain expertise minimizes logistical delays and fosters smoother transitions, troubleshooting, and overall customer experience,” Shetty concludes.

“When scaling up between clinical phases and commercial launch, it is common for a process to move between sites, and potentially between countries, within an end-to-end provider, so this results in the need for a tech transfer. Whether this is an internal or external transfer, there is little difference in the process that needs to be undertaken,” says Kempfer.

When it comes to sponsors and contract organizations working together to ensure a secure tech transfer, Youssef specifies that the CDMO should shoulder the responsibility. “A good CDMO [should] have structured communication, because communication is key … when you're talking to your business partner. We always build that in a more collaborative environment,” says Youssef. “[A CDMO needs] to protect the IP [intellectual property] rights of their clients, their partners, as well as good documentation practices, in addition to a solid IT department. All of that gives the assurance to the security of the data and the security of the transfer information between the CDMO and their partner.”

Simard stresses that communication is key for secure tech transfer. “The first step is to define success criteria and core values, ensuring both parties have a mutual understanding of each other's capabilities, limitations, and the project's critical milestones. When issues arise, they should be treated with speed and transparency in an atmosphere fostering collaboration rather than finger-pointing, so that solutions and a path forward may be found as a team,” Simard says.

Expectations, milestones, goals, and needed processes should be communicated and understood, says Kempfer. “The more information that can be shared at the start will reduce the potential of unexpected problems; however, having a transparent relationship between parties, where communication is open will allow progress to be monitored, and any issues that do arise to be shared and understood as quickly as possible. Having a key point of contact within the CDMO who can liaise with all of the key members of the project team across multiple disciplines, allows a customer to gain direct access to progress and ensure that communication is as efficient as possible,” Kempfer states.

Trust and transparency should be established, confirms Shetty. Differences in facilities and excipients should be understood, and process documentation should be simplified. “[One should] ensure regular communication between subject matter experts, rather than relying solely on project managers,” Shetty summarizes. “[Sponsors and contractors should] proactively work as a team to identify and resolve potential issues [and] focus on a common goal that benefits both parties.”



1. Hewitt, M.; Bellot, L.; Anderson, C. Best Practice Tech Transfer Methods for CGTs. 

Emerging therapies, patient-centric medicines, and the ever-changing world of bio/pharmaceuticals complicate technology transfer.

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