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Pharm Tech Group sits down with Bernard Sagaert, CEO of etherna, to examine the role of mRNA in the treatment of autoimmune diseases.

Exploring the Potential of mRNA (BIO 2024)

Messenger RNA (mRNA) technology was propelled into the limelight during the COVID-19 pandemic. In fact, the pandemic, which saw mass vaccinations using mRNA-based vaccines and financial support for their production, had a significantly positive effect on the mRNA market that is predicted to grow to approximately $40 billion by 2033 (1).

Since the pandemic, much work has been done to explore other potential therapeutic applications of mRNA. One such area is autoimmune diseases, where etherna, a Belgian mRNA technologies company, has been working on a tolerizing mRNA-lipid nanoparticle (LNP) platform that restores immune homeostasis (2). “So together with [Hasselt University], we want to further develop our platform and make sure that we have all the data to show that this platform can be used in an autoimmune setting, and specifically that RNA and LNPs can be used in that setting,” notes Bernard Sagaert, CEO of etherna.

“Everybody knows that the RNA-LNP platforms have proven their use in vaccines,” Sagaert says. “The approach in autoimmune diseases is similar yet very different. You could call it an inverse vaccination, where the depletion of the disease-causing autoreactive T effector cells and the amplification of the regulatory T cells is what we’re looking for, and RNA-LNP technologies have the potential of doing that in different ways.”

Watch the video for the full interview. etherna will be exhibiting at the 2024 BIO International Convention in San Diego, Calif., from June 3–6.

. mRNA Therapeutics Market (by Application: Oncology, Respiratory Diseases, Infectious Diseases, Others; by Type: Prophylactic Vaccines, Therapeutic Vaccines; by End User: Hospitals and Clinics, Research Organizations, Other)—Global Industry Analysis, Size, Share, Growth, Trends, Regional Outlook, and Forecast 2024–2033

. Market Report, May 2024.
2. etherna. etherna and Hasselt University Announce Autoimmune mRNA Therapeutics Research Collaboration. Press Release. May 21, 2024.

Bernard comes with extensive operational and strategic experience in the pharmaceutical and life sciences industry. He has expertise in manufacturing and distribution, and has an extensive operational background, both in companies and as consultant. He is a pharmacist from the University of Leuven, (Belgium) and has obtained the qualification of Qualified Person. He was a former QA and Supply Chain Director at Mylan in Belgium, and VP QA EMEAA at Sterigenics, a Sotera company.

Videos

Exploring the Potential of mRNA (BIO 2024) 

Pharm Tech Group chats with Dr. Monika Paulė, CEO and co-founder of Caszyme about the evolution of CRISPR.

Seeking Excellence with CRISPR (BIO 2024)

Clustered regularly interspaced short palindromic repeats (CRISPR) is a novel gene-editing approach that has seen rapid commercial success with the approval of exagamglogene autotemcel (Casgevy) occurring within a span of only 11 years after its invention (1). For Dr. Monika Paulė, the advent of CRISPR came at a time when the industry and academia were “ready for something new and groundbreaking.”

Paulė is the CEO and co-founder of Caszyme—a CRISPR gene-editing platform company based in Lithuania. “We develop novel CRISPR tools, and work with other companies in their gene editing, portfolio development from discovery, research, development, and everything that's related to proteins engineering and making the CRISPR-Cas9 proteins, the best tools for the applications the companies are looking for,” she says.

However, CRISPR is not without its challenges, Paulė remarks. More tools are required to allow the technique to be more widely applicable, she explains. “We really need to find the right tool, the right technique for the right application [which] is still a little bit challenging,” Paulė adds.

“To improve the safety and efficacy of CRISPR, first of all, we have to seek excellence in CRISPR field at each and every step of CRISPR technology development and application,” summarizes Paulė. “So, we have to start from the very beginning, from very early research, and really find and develop the best tools, which are safe, which are specific enough, and which are effective enough.”

Watch the video for the full interview. Caszyme will be attending the 2024 BIO International Convention in San Diego, Calif., from June 3–6.

1. Sheridan, C. The World’s First CRISPR Therapy is Approved: Who Will Receive It? 

Dr. Monika Paulė, CEO and co-founder of Caszyme

 is an accomplished leader with over 20 years of experience in high-tech business and has a PhD in Business Management. In 2017, Monika co-founded 

, a biotechnology company that leverages CRISPR technology to empower a world that’s healthier, equitable, and more sustainable, where she acts as chief executive officer and board member.

With Monika's leadership, Caszyme has gained recognition as a renowned expert in CRISPR gene editing field among partners worldwide. By leveraging deep expertise in discovery and development of CRISPR solutions, alongside the company’s dynamic platform and strategic adaptation of complementary technologies, Caszyme is unlocking new possibilities for companies across multiple industries, from healthcare to agriculture.

Additionally, Monika serves as a vice president and board member at the life sciences association “LithuaniaBIO” and is a founder of many social initiatives such as “Women in Biotech” and “Gene Editing Summit”. Monika is an engaging speaker and moderator with experience at multiple international live events, broadcasts, and podcasts.

The PharmTech Group spoke with Stacey Treichler

Sustainability Goals Impacting Biopharma (BIO 2024)

Advanced therapies, such as monoclonal antibodies (mAbs) have transformed the pharma industry, according to Stacey Treichler, senior director of Marketing and Strategy for Purolite at Ecolab. Various other innovations are also making their impact.

“We're seeing the increasing approvals of cell and gene therapies. And that's really exciting, because they're treating rare disease, degenerative disease, other hereditary diseases that were previously untreatable. But even within the antibody space, there has been quite a bit of innovation and disruption. So, we even see newer antibody modalities [such as] antibody fusions, bispecifics, multispecific, and antibody drug conjugates. And what's really exciting about those is they're also addressing unmet needs for things [such as] relapsed refractory cancer and other complex disorders,” Treichler states in an interview with the PharmTech Group prior to the BIO 2024 convention.

When it comes to saving costs, process intensification is allowing manufacturers to make more product with less footprint, according to Treichler. “And that's improving cost of goods for each gram of drug product manufactured. This can be applied across the traditional modalities, but also the more advanced modalities.”

The industry is also being impacted by the push for sustainability in biopharmaceutical development and manufacturing, with companies factoring sustainability into their strategies. “One of the things that we would advise is, don't just think about your own internal operations, but think about the operations of your suppliers, because that's really an extension of your footprint,” says Treichler. “A key area that pharma companies can focus on is water usage. Moving cooling, heating, and treating water across any facility requires energy. And by reducing water usage, companies can lower their energy consumption and greenhouse gas.”

Watch the video for the full interview. Purolite/Ecolab will be showcasing its products and services at the 2024 BIO International Convention in San Diego, Calif., from June 3–6.

The PharmTech Group spoke with Stacey Treichler, senior director of Marketing and Strategy for Purolite at Ecolab, about the impact of advanced therapies, new innovations, and sustainability on the biopharmaceutical industry.

Pharmaceutical Technology spoke with Jeff Clement, PCI

Robotics in Sterile Processing

Using robotics in sterile fill/finish operations provides “hands-off” aseptic manufacturing, says Jeff Clement, executive director of Technical Sales for Development and Manufacturing Group at PCI Pharma Services, in an interview with 

® “Most of the robotic instruments don't even have a glove board. So, there's no human interaction with the equipment, which is great for contamination. You know, one source of contamination for a RABs [restricted access barriers] type of line would be the human operators, and with a robotic manufacturing system that takes that completely out of the equation.”

Companies already performing aseptic fill/finish are doing environmental monitoring and therefore have ISO 5 suites, according to Clement. “But with an isolator technology or a robotic system, which would incorporate isolator technology, basically, you can do that in an ISO 7 suite. So, actually, your classification is a little bit lower, so your environmental monitoring is lower. And basically, you can validate that the lower classification, which could be a quicker way to put in aseptic manufacturing.”

When adding robotic isolator technology to a new facility, modular systems are available that would require supporting services, such as HVAC systems, says Clement, “but with the lower classification required for your operating space for isolator and robotic technology,” he adds. “A lot of times, you can do that a little bit quicker and more economically than perhaps you could in the past [in comparison to] setting up a RAB system in an ISO 5 environment.”

PCI Pharma Services will be showcasing its products and services at the 2024 BIO International Convention in San Diego, Calif., from June 3–6.

Pharmaceutical Technology® spoke with Jeff Clement, executive director of Technical Sales for Development and Manufacturing Group at PCI Pharma Services, about the challenges and benefits of incorporating robotics into aseptic manufacturing.

Communication between the manufacturing plant and retail stores. | Image Credit: © Cagkan - © Cagkan - stock.adobe.com

On May 30, 2024, Enzene Biosciences, a contract development and manufacturing organization (CDMO), announced it is launching its Drug Discovery Division at the BIO 2024 International Convention, happening from June 3–6 in San Diego, Cali. The new division will expand the CDMO’s services to the biotech industry. It will also complement the company’s EnzeneX-equipped biologics manufacturing facility that is expected to open in summer 2024 in New Jersey.

The new division will be located at the company’s Pune, India, facility and have a state-of-the-art discovery laboratory. Services provided will feature end-to-end integrated discovery services, including antibody target validation, discovery, and engineering; reagent production ranging from custom peptides and proteins to advanced modalities such as plasmids; RNA and exosomes; and multi-platforms assays services.

The $50 million New Jersey facility, which was announced in June 2023, is expected to open in July 2024 and will be Enzene’s first site in the United States with a fully connected continuous manufacturing platform, according to the company. Capacity is available for the production of complex proteins with standard modalities via bothChinese hamster ovaryand mammalian cell lines.

“There is a global shortage of biology services, and our clients are increasingly looking to collaborate with us much earlier in the discovery phase to expedite their progress to IND [investigational new drug application] and beyond. We anticipate even greater demand for these services in the coming year as the resurgence in biotech funding leads to more targets entering discovery—with contract R&D empowering them to advance more targets simultaneously. For our big pharma partners, in addition to running the integrated programs, we provide the flexibility to easily outsource non-core activities. In particular, we have seen strong demand for high quality reagents and assay services that play a crucial role across drug discovery and development,” commented Himanshu Gadgil, CEO of Enzene Biosciences, in a company press release (1).

 in June of 2023 by Choose New Jersey, a nonprofit economic development organization for the state of New Jersey.

“The strong relationship between New Jersey and India has gained increasing momentum since we opened the New Jersey India Center to cultivate economic opportunities during our trip to the country in 2019,” said NJ Governor Phil Murphy, in a press release (2). “I am proud to welcome Enzene Biosciences and their expanding global presence to New Jersey’s flourishing life sciences industry. New Jersey’s favorable business environment, skilled workforce, strategic location, supportive ecosystem for growth, and deep cultural ties are why Indian companies thrive in our state.”

In a January 2024 press release (3), Himanshu Gadgil, CEO, Enzene Biosciences, stated, “While the [US] is home to most of the novel molecules that are the principle ingredients in creating many life-saving drugs, most of them have been developed by small biotech firms and start-ups that struggle to find clinical-phase capacity. Recognizing this gap and driven by our fundamental mission of creating access equity across the global healthcare ecosystem, Enzene Inc. has been launched in the US to establish a state-of-the-art manufacturing plant to cater to small and mid-sized biotech companies. We have successfully identified, leased, and set up our first manufacturing facility in the [US] in record time. We remain confident that our proprietary EnzeneX technology platform and proven track record in achieving cost-effective biologics manufacturing will see us emerge as a partner of choice for biotech companies as we support them in bringing promising molecules to market.”

Enzene Biosciences. Enzene Launches Discovery Services Division at BIO 2024. Press Release. May 30, 2024.

Choose New Jersey. Enzene Biosciences Expanding to New Jersey with $50M Investment in Bio Manufacturing Plant. Press Release. June 15, 2023. https://www.pharmtech.com/view/enzene-biosciences-invests-50-million-into-new-jersey-biomanufacturing-facility https://www.choosenj.com/news/enzene-expanding-to-new-jersey/

Enzene Biosciences. Enzene Biosciences Launches Its First Manufacturing Base in the US. Press Release. Jan. 8, 2024.

Articles

The company’s new division will provide end-to-end integrated discovery services.

Enzene to Launch Drug Discovery Division at BIO 2024

Mary (Nora) Disis, MD, University of Washington

Pablo Guasp, PhD, Memorial Sloan Kettering Cancer Center (MSK)

American Society of Gene and Cell Therapy’s (ASGCT’s) 27th Annual Meeting

 in Baltimore, Md., which occurred May 7–11, 2024, Pablo Guasp, PhD, MSK asked himself the question, “What can we use to teach the immune system how to recognize cancer cells?” Replying, “… the neoantigen that arise from the accumulation of mutations in cancer cells can be good candidates … while a limitation is that neoantigens usually impose a restriction towards personalized treatments; therefore, we need a delivery platform that allows for modularity where we can include and exclude neoantigens based on the mutations of each individual patient. Also, we need a platform that allows us to do this in a timely manner, so we can treat patients soon after they are diagnosed. We believe that nucleic acid [developments] have been a breakthrough in this field, and in particular mRNA [messenger RNA] is showing very promising results”. Guasp then considered which patients benefit the most from this approach, stating, “If we look at vaccines against infectious agents we see that vaccines are always given in a prophylactic setting before the patient develop[s] the disease. But, paradoxically, cancer vaccines historically have been tested in patients with advanced and metastatic disease, and we think that this may not be the best scenario. We think that cancer vaccines may work best in patient with minimal disease, such as patients that had their tumors surgically resected” he averred. 

MSK partnered with BioNTech and Genentech to launch the first clinical trial of personalized mRNA cancer vaccines in pancreatic cancer. The results of this trial were published in 2023 (1). In only nine weeks, patients were receiving their own individualized cancer vaccine. This happened in such a timely manner, “because of a huge collaborative effort between all the members involved in the teams, but also because mRNA allows for this quick modularity, and this speed that I was mentioning before,” Guasp said.

During those nine weeks, the patient received a single dose of anti programmed cell death ligand 1 immunotherapy with atezolizumab and then received eight priming doses of the vaccine, followed by a standard-of-care chemotherapy and a single vaccine boost dose. “We identified specific responses elicited by the vaccine in half of the patients that receive[d] the vaccine. Not only that, but response to the vaccine correlate[s] with a delay recurrence time. … We know that this is a small clinical trial with a small number of patients, but the results are promising” Guasp added.

1. Rojas, L. A.; Sethna, Z.; Soares, K. C.; et al. Personalized RNA Neoantigen Vaccines Stimulate T Cells in Pancreatic Cancer. 

, 144–150. DOI: 10.1038/s41586-023-06063-y

In an ASGCT Panel on nucleic acid- and cell-based vaccines in oncology, work on a personalized mRNA vaccine was highlighted.

Highlighting Personalized mRNA-Based Vaccines for Pancreatic Cancer (ASGCT 2024)

Regeneron’s Bari Kowal gave insight into how the biopharma industry views the application of AI in R&D.

Exploring the use of AI in R&D at the US Pharma and Biotech Summit

® at the US Pharma and Biotech Summit, Regeneron's Bari Kowal, senior vice-president, head Development Operations & Portfolio Management, talked about the implementation of artificial intelligence (AI) in the R&D space. For example. Regeneron, Kowal explained, has been using AI for quite some time. Though not a new technology, she observed, AI has allowed the company to leverage many large language models and advanced searches for the past few years. Because of this, the company has built up collections of data in different areas, such as in genomics, protein databases, or for operational uses, including patient populations, study-level data, information about investigators, and quality of documents, among others.

“[Much] of our work is crossing over multiple facets of the drug development lifecycle early on in the discovery space, looking at how we manufacture and improving those processes, but also really looking at how we execute trials and reach out to the patients create diversity in the populations,” Kowal said. “We utilize some of the genomic data for finding the targets and more personalized medicine. [There are many] ways that we're leveraging AI.”

However, she emphasizes that the real value of AI is the ability to use it for existing data, especially in the search for optimizing operational efficiency. “Looking in the future, how do we use this to help our decision making and really better predict molecules that will have a tremendous effect? The biggest benefit that I can see, at least today, is that operational efficiency. If we can streamline drug development, from start to finish, our mission is to bring important medicines to patients at the end of the day, and so the time it takes today for drugs to be developed is too long. It's not measured in years, it's measured in decades,” she remarked. “If we can go from the research space to the development space in [a] few years, that is so much better and more beneficial to the patients that are waiting for those treatments.”

While speaking on a panel at the summit about diverging schools of thought on how AI should be used in R&D, Kowal observed that there were not necessarily divergent views by the panelists on how to use AI, but rather that the panelists had like-minded views on the topic. “I think we all believe that AI can be transformative. I think we believe that, ultimately, we're scratching the surface of what AI can do, but we also understand the human component and the context of human intelligence that you have to apply,” she stated.

 occurred on May 16, 2024 in New York, NY.

Bari Kowal, senior vice-president, head Development Operations & Portfolio Management, Regeneron, discussed the utility of AI and how it can benefit R&D work.

A Discussion on Using AI in R&D at the US Pharma and Biotech Summit

Interview with Ken Keller, president and CEO, Daiichi Sankyo, at US Pharma and Biotech Summit

Ken Keller, CEO of Daiichi Sankyo sat with 

® at the US Pharma and Biotech Summit to discuss how to effectively create productive partnerships in the bio/pharmaceutical industry. He noted that a key component is for prospective partners to have a shared commitment to the therapeutic area that is the focus of a drug development program. “If it's strategically important to your partner, then it usually works out very, very well,” Keller said.

Relevant experience in the therapeutic area is also important. “Specifically, we work in oncology. So, if we're looking at a drug that has potential in breast cancer, we'll be looking for someone that's showing success in oncology in breast cancer very recently.”

Challenges involved in creating partnerships for developing therapies include creating a clinical data plan and committing to developing a stable supply chain going forward, Keller points out. “[A clinical data plan] is really the roadmap for whatever asset you have, that looks out five or 10 years, because sometimes it takes that long to complete the trials and the challenges, you've got to design them to deliver the evidence that not only prescribers will find attractive, but also payers.”

“I think it starts with the CEO and the C suite, doing something that only they can do. So as a leader, you've got to set the expectations for how bold you want that team to be,” says Keller. “The most important thing when two companies decide that they're going to do something together, is to really find a cohesive relationship, where you both believe that you can do more together than separately.”

Ken Keller, president and CEO, Daiichi Sankyo, discusses the foundation of partnership and takes a look at the future of biotech partnerships at the US Pharma and Biotech Summit.

A Look at the Future of Bio/Pharma Partnerships at the US Pharma and Biotech Summit

Melissa Green, head of global marketing for the healthcare division at TekniPlex, shares highlights from the company's presentation at INTERPHEX 2024. 

TekniPlex at INTERPHEX 2024: Advances in Materials Science

 sat down with Melissa Green, head of global marketing, healthcare division, at TekniPlex, to discuss the company's offerings and recent additions to its capabilities. 

TekniPlex is a global, integrated company that provides materials science and manufacturing technology solutions for healthcare, including the medical device, pharmaceutical, and diagnostics industries. 

During the show, TekniPlex demonstrated its expertise to support the burgeoning biologics market, including solutions for upstream and downstream processing and final fill operations, as well as single-use bioprocessing applications. 

“As the biologics sector continues its steady growth, so does the need for high-quality materials and components suppliers who can also be innovative solutions partners," said Chris Qualters, CEO of TekniPlex Healthcare, in a 

. “With our expanding capacities and innovative mindset, TekniPlex Healthcare is poised and ready to respond to the evolving needs of the sector, providing value-added solutions that help bring life-changing therapies to market.”

During the interview, Green also spoke to the advantages of the company's 

 in late 2023, which elevated the company to a full-service contract development manufacturing organization for medical materials science, specifically within the minimally invasive and interventional therapy devices space. 

Learn more about what TekniPlex has to offer by watching the video interview above. 

TekniPlex recently acquired Seisa Medical, which has expanded the company's services into that of a CDMO. 

How TekniPlex is Expanding to Support Innovative Solutions in Materials Science

Video interview with Adam Covitt, President, Federal Equipment Company

Federal Equipment Company: Supporting CDMO Operations Through Training, Parts, and Equipment

At a recent Drug, Chemical & Associated Technologies (DCAT) conference, 

spoke with Adam Covitt, President of Federal Equipment Company, to hear the latest in the CDMO landscape. Learn more about: 

An introduction/re-introduction to Federal Equipment Company.

Challenges and opportunities in the 2024 CDMO landscape.

How Federal Equipment Company can assist CMDO success with its Techceuticals and PharmParts business brands.

Adam Covitt, President of Federal Equipment Company, discusses how the company enables CDMO successes with its Techceuticals and PharmParts business brands. 

Latest Conference Articles

Exploring the Potential of mRNA (BIO 2024)

Seeking Excellence with CRISPR (BIO 2024)

Sustainability Goals Impacting Biopharma (BIO 2024)

Robotics in Sterile Processing

Enzene to Launch Drug Discovery Division at BIO 2024

Highlighting Personalized mRNA-Based Vaccines for Pancreatic Cancer (ASGCT 2024)

A Discussion on Using AI in R&D at the US Pharma and Biotech Summit

A Look at the Future of Bio/Pharma Partnerships at the US Pharma and Biotech Summit

How TekniPlex is Expanding to Support Innovative Solutions in Materials Science

Federal Equipment Company: Supporting CDMO Operations Through Training, Parts, and Equipment