Feliza Mirasol is the science editor for 

Feliza Mirasol

Frontier Scientific Solutions, a temperature-controlled storage and transportation company that exclusively serves the life sciences industry, announced on Nov. 13, 2024 that it has established state-of-the-art current good manufacturing practice (CGMP) facilities at Wilmington International Airport (ILM) in North Carolina, United States, and Shannon Airport (SNN) in County Clare, Ireland. The strategic locations will enable the company to create the first life sciences-dedicated direct global gateway, according to a company press release. Supported by a $1.5 billion commitment from an investor, the gateway is central to Frontier Scientific Solutions’ goal to create streamlined shipping lanes—starting with a route between North America and Europe—specifically for time- and temperature-sensitive pharmaceutical and biopharmaceutical products.

These facilities are supported by the $1.5 billion funding from GID, a real estate investor, owner, operator, and developer. Frontier Scientific Solutions expects that the facilities in both locations will bring further financial and supply chain management benefits through designation as Free Trade Zones (FTZ). The company unveiled plans at ILM for the scale and impact of its global gateway model. The company expects that this model will enable companies in the life sciences industry to transport time- and temperature-sensitive materials in significantly shorter timeframes. Benefits include fewer touchpoints, less risk, and more sustainable operations.

"We are thrilled to announce this transformative partnership with the airports at Wilmington and Shannon, enabled by GID's investment,” said Steve Uebele, CEO of Frontier Scientific Solutions, in the company press release. “Our initiative disrupts the life science supply chain market—promoting zero product losses, increasing sustainability, and directly saving lives—and it marks the first step in Frontier's goal to become the market-leading solutions provider."

The funding will be used for the construction of CGMP-compliant, temperature-controlled storage facilities housed at Shannon and within the FTZ at Wilmington. Having FTZ designation will facilitate streamlined customs and FDA processing, duty deferral, and operational efficiencies, all of which are critical for the efficient and secure distribution of life sciences products across international borders, the company stated in the release.

"GID’s commitment underscores our dedication to developing cutting-edge infrastructure that meets the specialized needs of the life sciences industry,” said Greg Bates, president and CEO of GID, in the release. “The new facilities in Wilmington and Shannon will create an unparalleled logistics network between the US and Europe, empowering companies to deliver critical healthcare products with unmatched speed and precision."

Based on live case studies conducted by Frontier Scientific Solutions, it was consistently demonstrated that a reduction of days to weeks in the transportation and waiting times for temperature-sensitive shipments were achieved with its gateway model. “Providing the industry with direct distribution makes this global gateway model faster, reducing exposure to temperature excursions, risk from unnecessary touchpoints, and carbon emissions. It will set a new benchmark for quality, speed, and environmental responsibility,” the company stated in the press release.

The facilities at ILM and SNN will be linked by a direct lane dedicated exclusively to life sciences materials. Flight operations are slated to begin early in 2025. Frontier Scientific Solutions also offers a fast direct-ocean lane from North Carolina to Cork, Ireland, and can achieve delivery in 12 days via this route. This new infrastructure will allow the company to disrupt the market by enabling faster deliveries while reducing reduced risk.

In addition to supply chain logistics, the biopharma industry is seeking ways to minimize risks in providing temperature-sensitive molecules to patients, such as 

innovating product formulation and drug delivery systems

Prioritizing Formulation Strategies for Temperature-Sensitive Biotherapeutics

Pharmaceutical Technology Trends in Formulation

Articles

Frontier Scientific Solutions has unveiled plans to use a $1.5 billion commitment from a major investor to create a global free trade zone gateway to facilitate the life sciences supply chain using Wilmington International Airport in North Carolina.

Frontier Scientific Solutions Plans a Global Free Trade Zone Gateway to Supply Life Sciences Industry on Back of $1.5 Billion Investment

Avantor, a provider of mission-critical products and services to the life sciences and advanced technology industries, announced on Nov. 14, 2024 that it has opened its new flagship Bridgewater Innovation Center in Bridgewater, NJ.  Spanning 60,000 square feet, the state-of-the-art R&D facility joins the company’s network of 13 research and innovation centers globally.

The expanded facility doubles the company’s previous laboratory and pilot plant capacity and was purpose-designed to enable collaborative work. The center houses spaces for upstream and downstream process development, dedicated analytical testing labs, and a viral vector laboratory. It also houses an expanded pilot plant that supports scale-up simulations, which is intended to enable rapid customization across the entire bioprocessing workflow, according to a company press release.

“Our new Bridgewater Innovation Center is twice the size of our previous facility and adds capacity, talent, and capabilities to support the growing demands for monoclonal antibodies [mAbs], cell and gene therapy [CGT], and mRNA [messenger RNA] workflows,” said Michael Stubblefield, president and CEO of Avantor, in the press release. “Working side-by-side with customers in our network of innovation centers across the globe enables us to power science at every step to deliver life-changing therapies faster and more cost effectively.”

The center gives Avantor the capabilities to optimize and accelerate biomanufacturing processes at scale. Advantages include faster problem solving, streamlined knowledge exchange, and strategic co-innovation. The facility is staffed with PhD scientists, bioengineers, biologists, and process engineers, which enhances capacity and expertise to support customers' needs. As stated in the press release, “The facility also supports Avantor’s sustainability goals, reinforcing the [c]ompany’s commitment to environmental responsibility.”

The center is aimed at helping biopharmaceutical companies accelerate the development of vital therapies across multiple modalities, including mAbs, CGTs, and protein/peptide therapeutics. Expansive collaboration spaces built throughout the facility allow scientists, biologists, and bioengineers to work side-by-side with biopharma manufacturers. These collaborations leverage the company’s advanced products, real-time analytical data, and process knowledge to optimize processes and materials. Having this co-development environment saves crucial time and resources, according to the company in the press release.

® (1), Benoit Gourdier, executive vice-president and head, Bioscience Production Segment, Avantor, noted how the pace of innovation in biopharma has been posing challenges, particularly in relation to which platform technology to use. “This is something where we’re seeing a fast evolution. From that perspective, at Avantor we are agnostic because we can provide product, solutions, technologies, platforms, support, and this innovation center is a good example. No matter the modality, we have a solution for the players,” Gourdier said in the interview.

Nandu Deorkar, senior vice-president, Bioscience Production Research & Development, Avantor, also spoke in the interview and pointed out that, because of the shift to personalized medicine manufacturing, there will be more distributed manufacturing. “If you look at the cell and gene therapy [space], [patients] will be treated on a personal basis, so there will be more distributed manufacturing on a local basis; so how do we support this geographically?” Deorkar said in the interview (1).

Meanwhile, the anticipated flood of new biotherapeutic modalities onto the market in the near future aimed at precision treatment is expected to open up many new opportunities for service providers, Gourdier said in the interview (1).

Avantor Sees Future of Opportunities with Growing Development of Next-Gen Biotherapeutics

Avantor's newly launched expanded Innovation Center in Bridgewater, NJ, is designed for integrated workflows and features purpose-built collaboration spaces. 

Newly Opened Innovation Center in New Jersey Gives Avantor Advantage in Solving Life Sciences Challenges

In preparation for the launch of its new innovation center in Bridgewater, NJ, Avantor executives discussed a future full of opportunities for service providers, which will come from a growing number of next-generation biotherapeutics in the development pipeline that are anticipated to reach the market in the near future.

“The first thing [that comes to mind] is lots of opportunities, because this is really going back to the base of innovation,” said Benoit Gourdier, executive vice-president and head, Bioscience Production Segment, Avantor, in an interview with 

® at a press event held at the Bridgewater facility on Nov. 13. 2024.

Where once the biopharma industry was dominated by monoclonal antibodies (mAbs), the industry can now expect to see a wave of newer, more innovative therapies aimed at achieving precision treatment. “Starting 25-30 years ago, it was really mAbs, mAbs, mAbs, and traditional vaccines,” Gourdier said, adding, “We grew up in this environment. Now we have this diverse portfolio of modalities, so [that will provide] lots of opportunities to go after, to learn.”

The challenges that Gourdier anticipates in the future could likely revolve around chemistry, fluid handling, meeting high purity in a regulated market, among others, but Gourdier is confident that Avantor will be well prepared to meet these challenges and to offer the appropriate support as a service provider.

Nandu Deorkar, senior vice-president, Bioscience Production Research & Development, Avantor, added that, because of the shift to personalized medicine manufacturing, there will be more distributed manufacturing. “If you look at the cell and gene therapy [space], [patients] will be treated on a personal basis, so there will be more distributed manufacturing on a local basis; so how do we support this geographically?” Deorkar said in the interview.

Deorkar also added, “Some of these therapies have 48 hours to 72 hours injection requirement after manufacturing, so [not all] the manufacturing can be done [in one place].”

Gourdier, meanwhile, pointed out that, in addition to the expectation of a different manufacturing and supply chain scenario for next-gen biotherapeutics, the industry suffered from supply chain disruptions due to the COVID-19 pandemic, which are still ongoing in the post-COVID environment. Regionalization has become more important, he noted. “[Developers] want global partners with regional focus,” he stated.

Other factors that have disrupted the pace of development for these next-gen biotherapeutics has been a drop in funding as a direct result of the COVID-19 pandemic, Gourdier added. “Most of the big players are okay,” he observed, “but for smaller players, the amount of money available for them has decreased significantly. We are just [coming] back [from that]. Now we are in modest recovery from that (i.e., the funding) perspective.”

Meanwhile, the pace of innovation has itself been posing challenges, particularly in relation to which platform technology to use. “This is something where we’re seeing a fast evolution. From that perspective, at Avantor we are agnostic because we can provide product, solutions, technologies, platforms, support, and this innovation center is a good example. No matter the modality, we have a solution for the players,” Gourdier stated.

Avantor’s new Bridgewater Innovation Center is set to launch on Nov. 14. It has been designed as a state-of-the-art research and development facility and joins the company’s network of 13 research and innovation centers globally.

Executives at Avantor talk about the future of the biopharmaceutical industry and the anticipated impact that a wave of next-generation biotherapeutics will bring.

Ginkgo Bioworks, a provider of a cell programming platform and biosecurity, announced on Nov. 12, 2024 that it has completed the first milestone of a previously announced partnership (1) with Merck, known as MSD outside of the United States and Canada. Their collaboration aims to improve biologics manufacturing, and, according to the terms of their agreement formed in August 2023, Ginkgo will receive a research milestone payment of $9 million in cash for completing this milestone.

Under their partnership, Ginkgo uses its expertise and capabilities in cell engineering, ultra-high-throughput multiplexed screening, protein characterization, and process optimization to improve biologics production efficiency and increase yields. The company is eligible to receive up to $490 million in aggregate of upfront research fees, research milestone fees, option license payments, and commercial milestone payments (1).

"We're excited to achieve the first milestone of our collaboration with Merck and continue our work to improve biologic manufacturing production," said Jason Kelly, CEO and co-founder, Ginkgo Bioworks, in a company press release. "It's very meaningful when our team can drive impactful and technically innovative work like this, especially when we're working shoulder-to-shoulder with counterparts like the team at Merck.”

The two companies will now move on to the second stage of the partnership, which involves focusing on the continued improvement of the biologics production process.

“As we go into the next phase of this project, we'll continue to deploy the strain engineering, high-throughput screening, and process development capabilities as well as the assets and know-how that make Ginkgo a unique and powerful partner to top pharmaceutical companies," Kelly said in the release.

The August 2023 partnership deal is the second collaboration between Ginkgo and Merck. In October 2022, the companies entered into a biocatalysis project to improve Merck's API manufacturing (2). Under the biocatalysis deal, Ginkgo earned an upfront research and development fee and was eligible for success-based research and development milestone payments. Ginkgo was also eligible to earn commercial milestone payments for each of a specified number of biocatalysis targets. These targets have the potential to total, in the aggregate, up to $144 million, according to a company press release issued at that time.

"At Merck we are always seeking new and innovative ways to optimize process efficiency while maintaining product integrity," said Michael Kress, PhD, senior vice-president, development sciences and clinical supply, Merck Research Laboratories in an Aug. 7, 2023 press release (1). "We are pleased to build upon our relationship with the Ginkgo Bioworks team."

1. Ginkgo Bioworks. Ginkgo Bioworks Announces Collaboration with Merck to Improve Biologic Manufacturing. Press Release. Aug. 7, 2023.
2. Ginkgo Bioworks. Ginkgo Bioworks Announces Collaboration with Merck to Improve Active Pharmaceutical Ingredient Manufacturing. Press Release. Oct. 11, 2022.

Under an ongoing partnership with Merck, known as MSD outside of the United States and Canada, Ginkgo Bioworks has achieved the first milestone in a project aimed at improving biologics production.

Merck and Ginkgo Bioworks’ Collaboration to Improve Biologics Manufacturing Has Reached Its First Milestone

TrakCel, a provider of integrated cell and gene therapy (CGT) orchestration solutions, announced on Nov. 12, 2024 that some manufacturers have selected its orchestration platform, OCELLOS, to support the management of five out of seven autologous and matched allogeneic cell therapy products. These products are approved or are expected to be approved in 2024. TrakCel’s technologies enable the precise management, control, and tracking of CGTs.

An IT portal, such as OCELLOS, is needed to successfully navigate the CGT supply chain, as manufacturers require tools to help launch these FDA-approved products and ensure that approved treatment centers (ATCs) can administer the CGT safely and effectively, according to a company press release.

“We’re committed to driving forward patient access to ground-breaking CGTs and are delighted to be supporting a growing number of manufacturers as they navigate the CGT supply chain and successfully launch their product,” said Fiona Withey, PhD, CEO, TrakCel, in the press release. “Commercializing CGT products is a complex and challenging process that requires close collaboration between manufacturers and ATCs. However, as more products are approved, ATCs are concerned this could lead to further proliferation of custom-made orchestration platforms, creating an additional burden on their staff that could potentially hinder the success of future new product launches.”

A list of CGTs approaching regulatory approval by the end of 2024 are listed in a presentation given by The Alliance for Regenerative Medicine (ARM) in August 2024 (1). OCELLOS is a Salesforce-powered solution that was specifically designed for the CGT industry, according to TrackCel, which stated in its press release that the platform aims to deliver a unique therapy process by connecting base modules and configurable forms with real-time visibility showing the progress of each personalized therapy through the supply chain. This workflow is intended to support efficiency in the supply chain and ease collaboration with ATCs.

“Working with our Industry Advancement Board (IAB), we recognize that there is a growing need to standardize on one platform to help foster a consistent approach to capabilities and communication processes, training, workflows, and use of terminology,” Withey stated in the press release. “OCELLOS is a proven technology ready to drive industry-wide standardization and ensure life-enhancing therapies reach the patients that so desperately need them.”

TrackCel also pointed out in its release that it sees continued investment in custom-built IT portals, even as CGT manufacturers are increasingly using OCELLOS. The company stated that these IT portals help facilitate product ordering and patient enrollment as well as efficient orchestration of supply chain activities, including manufacturing and logistics.

“As more CGT products are approved … an increasing number of custom-built platforms could overwhelm clinical site staff and be extremely expensive for each manufacturer to build and support, potentially limiting access to the therapy, and impacting patient safety,” TrackCel warned in its press release.

1. ARM. Sector Snapshot: The Emerging Value of Cell and Gene Therapy. 

alliancerm.org/sector-snapshot-august-2024

Manufacturers of five autologous or matched allogenic cell therapy products have selected TrakCel's  IT platform, OCELLOS, to orchestrate the administration of these therapies, which are approved or expected to be approved in 2024.

TrakCel’s OCELLOS Platform Selected to Support Treatment Management of Five Upcoming Cell Therapy Products

On Nov. 6, 2024, Johnson & Johnson (J&J) announced that the Medicines and Healthcare products Regulatory Agency (MHRA) of the United Kingdom has granted marketing authorization for Balversa (erdafitinib) to be used as a monotherapy in treating adults with unresectable or metastatic urothelial carcinoma (UC), which is the most common form of bladder cancer (1,2). This approved indication specifically covers eligible patients harboring susceptible fibroblast growth factor receptor 3 (FGFR3) genetic alterations, and who have previously received at least one line of therapy containing a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor in the unresectable or metastatic treatment setting.

MHRA’s authorization is based on results from Cohort 1 of a Phase III study (THOR). The study was randomized, open-label, and spanned multiple clinical treatment centers. The study evaluated the efficacy and safety of erdafitinib (n=136) versus chemotherapy (n=130) in patients with advanced or metastatic UC with select FGFR alterations who have progressed on or after one or two prior treatments, where at least one of those treatments included a PD-1 or PD-L1 inhibitor. The study’s primary endpoint was overall survival (OS), and secondary endpoints were progression-free survival (PFS), objective response rate (ORR), and duration of response (DOR), according to a company press release.

“Patients living with this advanced stage of bladder cancer, and whose tumors harbor FGFR3 alterations, require access to innovative precision therapies that can target the specific characteristics of their disease,” said Professor Alison Birtle, consultant oncologist and honorary clinical professor, Lancashire Teaching Hospitals National Health Service (NHS) Foundation Trust, in the press release. “Unfortunately, until now, there have been limited treatment options available for this group of patients, which may affect not only their prognosis, but their wellbeing and quality of life. Today’s authorization of erdafitinib, a targeted therapy that has been shown to significantly improve overall and progression-free survival for patients with FGFR3 alterations, will come as welcome news to eligible patients, and highlights the importance of integrating biomarker testing into the treatment pathway for people with urothelial cancer, so that genetic alterations such as FGFR3 can be detected as early as possible.”

Erdafitinib is administered orally as a once-daily, FGFR kinase inhibitor. It works by inhibiting the activity of FGFR3 alterations in cancer cells and has demonstrated an ability to extend overall survival compared to chemotherapy in the second-line setting (3).

In June 2023, the Phase III study (THOR) was stopped based on the recommendation of an independent data safety monitoring committee, following a review of efficacy and safety data of the study at interim analysis (3). All patients who were randomized to chemotherapy (docetaxel or vinflunine) were offered an opportunity to receive erdafitinib as crossover therapy, and results at the data cut-off timepoint showed that median OS of over one year was achieved in those patients receiving erdafitinib. These results marked a significant improvement compared to the patients in the chemotherapy arm (12.1 months vs. 7.8 months; hazard ratio, 0.64; 95% confidence interval, 0.44 to 0.93; P=0.005) (1).

Moreover, treatment with erdafitinib showed an improvement in median PFS compared to chemotherapy of 5.6 months versus 2.7 months and a confirmed ORR of 48 out of 136 patients (35.3%) versus 11 out of 130 patients (8.5%) (1).

“We are delighted that the MHRA has recognized the value that erdafitinib could bring to eligible patients with metastatic urothelial cancer,” said John Fleming, country medical director, Johnson & Johnson Innovative Medicine UK, in the release. “This milestone reflects J&J’s long-standing dedication to getting in front of cancer and delivering the most innovative precision therapies to patients in need. We look forward to progressing with HTA [health technology assessment] submissions for erdafitinib in the coming months, with the view to enabling eligible patients to access erdafitinib through the NHS as soon as possible.”

In the UK, approximately 10,500 people are diagnosed with bladder cancer each year, equating to 29 people every day (4). Of those diagnosed with advanced or metastatic bladder cancer, roughly 20% have FGFR3 alterations, which can drive the growth of cancer cells (5).

1. MHRA. Electronic Medicines Compendium, Balversa Summary of Product Characteristics. 

 (accessed November 6, 2024).
2. Leslie, S.W.; Soon-Sutton, T. L.; Aeddula, N. R. Bladder Cancer. In 

; StatPearls Publishing, Aug. 15, 2024.
3. Loriot, Y.; Matsubara, N.; Park, S. H.; et al. Erdafitinib or Chemotherapy in Advanced or Metastatic Urothelial Carcinoma. 

 (21),1961–1971.
4. Cancer Research UK. Bladder Cancer Statistics. 

cancerresearchuk.org/health-professional/cancer-statistics/statistics-bycancer-type/bladder-cancer

 (accessed Nov. 6, 2024).
5. Xiao, J. F.; Caliri, A. W.; Duex, J. E.; Theodorescu, D. Targetable Pathways in Advanced Bladder Cancer: FGFR Signaling. 

Balversa (erdafitinib) marks the first and only bladder cancer therapy to target FGFR3 alterations, with a demonstrated increase in overall survival from 7.8 months to 12.1 months. 