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

Chris Spivey

Behind the Headlines: Mpox Resurges; Eli Lilly Adds CGT Facilities; FDA Updates

This Behind the Headlines news roundup panel discussion covers news items such as the resurgence of mpox in Africa, “medical tourism” in India, Eli Lilly’s new Boston CGT facilities, and contract development and manufacturing organization (CDMO) projected growth rates for the 2023–2029 period, and the reasons behind them.

The panel also discussed at more length the surge, or more appropriately the revitalization of interest, in the antibody-drug conjugate (ADC) space. In August 2024, Merck said it will pay an initial $700 million to acquire full global rights to clinical-stage bispecific antibody CN201 from privately held biotechnology company Curon Biopharmaceutical. Why was there so much excitement about ADCs at the recent BIO Conference in San Diego

The panel also talked about FDA leadership, especially in regard to training how to solve common problems but also in how best to train staff generally. A 

 notice dated Aug. 8, 2024 announced FDA’s Fiscal Year 2025 for the Center for Drug Evaluation and Research (CDER) Office of Pharmaceutical Quality (OPQ) Experiential Learning Site Visit Program (ELSVP) (1). As per their press release, the ELSVP, “allows staff to observe commercial manufacturing and testing operations as well as pilot plants, with the goal of better understanding standard operations of the pharmaceutical industry and how those impact the developmental programs and commercial life cycles of drugs. It does not replace or supplement a regulatory inspection” (1).

Also, due to the rapid expansion of cell and gene therapies (CGT), FDA has launched the Cellular and Gene Therapies Interactive Site Tours Program. “This initiative allows FDA staff to visit industry sites, facilitating an exchange of regulatory and industry best practices. By gaining first-hand insight into the manufacturing process, the FDA aims to enhance its review capabilities and foster a deeper understanding of biotech operations. This collaboration between industry and the regulatory body is a crucial step towards streamlining the development of ATMPs [advanced therapy medicinal products], ultimately benefiting patients and advancing the field of biotechnology” (2).

As a final wrap-up, the panel looked at the idea that the dark days of 2023 investment are now squarely behind us, and that the dawn of stronger investment sentiment is coinciding with a broader adoption cycle of artificial intelligence and machine learning equipment and innovation. This presages well for a boom two-year cycle, which is about to provide unusually high levels of industry growth opportunities for nearly all stakeholders—ultimately passing on rewards to most patient populations, and especially those in the rare disease category.

Lavery, P. Drug Companies Encouraged by FDA to Participate in FY 2025 Site Visitation Program. 

https://www.pharmtech.com/view/drug-companies-encouraged-fda-participate-fy-2025-site-visitation-program

Manalac, T. FDA Eyes Site Tours of Cell and Gene Therapy Manufacturers. 

Dr. Laks Pernenkil Principal, US Life Sciences Product, Supply and Manufacturing Operations Practice Leader, Deloitte Consulting

Beth Willers, Principal and Owner, White Matter Communications

Benjamin McLeod, Director, Cell & Gene Therapy Segment, NanoMosaic Inc.

Chris Spivey, Director of Industry Relations and Strategic Partnership, MJH Life Sciences

Videos

This Behind the Headlines news roundup panel discussion covers news items such as the resurgence of mpox in Africa, “medical tourism” in India, Eli Lilly’s new Boston CGT facilities, and contract development and manufacturing organization (CDMO) projected growth rates for the 2023–2029 period, and the reasons behind them. 

Nadia Sellami, PhD, global segment lead for gene and cell therapy at PacBio interview

The Benefit of Long-Read Sequencing in Gene Therapies

At the American Society of Gene and Cell Therapy (ASGCT) conference in Baltimore, Md. in May 2024, 

® sat down with Nadia Sellami, PhD, global segment lead for gene and cell therapy at PacBio, about the benefits of long-read sequencing in cell and gene editing.

According to Sellami, long-read sequencing detects information that short-read sequencing cannot capture. “And in contrast to other long-read sequencing methods, it has higher accuracy while maintaining the same read length,” she says in this video interview. “So, for example, you can see long indels that you might get from gene editing. You can sequence the entire AAV [adeno-associated virus] molecule, ITR [inverted terminal repeat sequence] to ITR. You can sequence plasmids. You can sequence full length mRNA [messenger RNA] and so many more sequencing applications.”

DNA sequencing is important for the design, production, and quality of gene therapies, says Sellami. Click the video box above to view the full interview.

For more coverage from the 2024 ASGCT conference, visit PharmTech.com’s 

Highlighting Personalized mRNA-Based Vaccines for Pancreatic Cancer

Research Shows that HIC Can Effectively Separate Deaminated Capsids from Non-Deaminated Capsids

Using Receptor Architecture to Improve CAR-T Cell Sensitivity in Oncology

The Role of Bispecific Antibodies in Redirecting AAV Vectors

Nadia Sellami, PhD, global segment lead for gene and cell therapy at PacBio, talks about long-read sequencing for cell and gene editing in a video interview. 

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

Articles

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)

In this video, Chris Spivey, Director, Industry Relations and Strategic Partnerships, talks with industry experts about aspects of drug product stability.

Drug Digest: Is Our Understanding of Stability Changing

In this exclusive Drug Digest video interview, Chris Spivey, Director, Industry Relations and Strategic Partnerships, interviews Nitin Swarnaker, Pharma Applications Manager, BASF, and Geoff Carr, Former Director, Analytical Development, Thermo Fisher Scientific/Patheonabout various aspects of drug product stability testing. Stability studies involve a drug substance tested under storage conditions and assess its thermal stability and sensitivity to moisture. Over the last decade there has been some intermixing of supply chain storage and distribution systems, and traditional formulations taking these storage and delivery systems into account—continuous monitoring comes to mind. While long-term testing should be performed over a minimum of 12 months at 25 °C ± 2 °C/60% RH ± 5% RH or at 30 °C ± 2 °C/65% RH ± 5% RH. The intermediate and accelerated testing should cover a minimum of 6 months at 30 °C ± 2 °C/65% RH ± 5% RH. Accelerated Predictive Stability (APS) studies, carried out over a 3–4-week period and combining extreme temperatures and RH conditions (40–90 °C)/10–90% RH, have emerged as novel approaches to predict the long-term stability of pharmaceutical products in a more efficient and less time-consuming manner.

Nitin is a Formulation scientist with over 16 years of combined (academic research/training/industrial) experience, and a very strong track record of publications, primarily focused on various formulation aspects of small, peptide and protein molecules for oral and parenteral applications. He has vast experienced in solid, solution, suspension, semisolid products and novel drug delivery systems such as lipid- and polymer-based drug delivery systems (proliposomes and liposomes, liquid crystalline particles nanoparticles and microspheres) including their targeted drug delivery counterparts. He is well-versed in various characterization techniques used in formulation development, possesses a good understanding of various regulatory guidelines and cutting edge concepts and technologies.

Former Director, Analytical Development, Thermo Fisher Scientific/Patheon

Geoff Carr, PhD, has retired after over 45 years experience of working in the pharmaceutical industry and currently provides training and consultancy services to pharmaceutical companies with an emphasis on the application of good chemistry in the development of new pharmaceutical products. Previously, he was appointed as Manager, Analytical Development at Patheon Inc, Canada in 2000 and progressed to Director, Analytical Development, Thermofisher Scientific/Patheon until 2023. Prior to that, he held positions of Manager, Analytical Sciences at Celltech-Chiroscience, UK, Head of Analytical Development at Medgenix, Belgium and before that at Wyeth Research, UK. He originally started his career in pharmaceutical analysis at The British Pharmacopoeia Laboratory where he held several posts and was finally appointed Head of the Laboratory. His academic background is a PhD in synthetic organic chemistry awarded by University of London, UK. He has been a member of various British Pharmacopoeia Committees, a UK representative to Committees of the European Pharmacopoeia and Member of USP Committees including a Member of the 2015-2020 Cycle USP General Chapters – Physical Methods Expert Committee. He is the author of numerous publications including journal articles and book chapters as well as presentations and workshops at international conferences on various topics related to applications of analytical chemistry during the development of pharmaceutical products. This includes a 8 module distance learning course on Pharmaceutical Stability Testing.

This episode of Drug Digest is sponsored by:

Drug Digest is a tech talk video series with the 

® editors, who interview industry experts to discuss the emerging opportunities, obstacles, and advances in the pharmaceutical and biopharmaceutical industry for the research, development, formulation, analysis, upstream and downstream processing, manufacturing, supply chain, and packaging of drug products.

 Biologic Drug Development and Manufacturing

In this exclusive Drug Digest video interview, Chris Spivey, Director, Industry Relations and Strategic Partnerships, talks with industry experts about various aspects of drug product stability testing.

Cytiva's Eric Corti discusses the challenges with designing a new single-use mixing system that ensures leak-free fluid handling operations.

Meeting Upstream Bioprocessing Challenges with Innovative Engineering Design (INTERPHEX 2024)

With the recent launch of Cytiva’s new single-use magnetic mixer system, the company took to 

® at the show, Eric Corti, global product manager for the Mixer Portfolio at Cytiva, discussed the key characteristics, challenges, and future of design of such equipment.

“This is a large-scale mixer that is primarily targeting media and buffer preparation applications. It's solving some really unique challenges in that space, and that's really driven by the consumable,” Corti stated. He explained that, in talking with customers, the company came to understand that one of the main problems plaguing the industry for large-scale media and buffer preparation are leaks.

To address this problem, the company aimed to design consumables that could effectively protect against leaks. The consumables were thus designed with protective features, such as a carrying case that wraps around the outside of the film and a covering for the impeller for protection on the inside. The company’s work didn’t stop at the design alone, Corti remarked, but rather the company went a step further to simplify the process as well.

“We understand that, during installation, large-scale consumables can often be tricky to install, and if they're done incorrectly, this can compound and lead to leaks, so we wanted to design something that was really simple to install, made it ergonomic and intuitive for operators to place the consumable inside of the hardware, and overall just make that experience [much] simpler and take [up] [much] less time. [This] can cut down on these leaks occurring in the first place, and can save customers a [significant amount] of money and a lot of pain and trouble that comes when those leaks do occur,” Corti emphasized.

Corti also pointed out that the new mixing system does not require any special configuration of the facility. “The mixer here has been deliberately designed to have a footprint that is going to fit through double doors that are standard for most clean rooms, as well as fitting within typical cleanroom ceiling heights. It’s going to cut down [much] of the costs … typically associated with modifying facilities just to get large-scale equipment in the door,” he explained.

Furthermore, Corti predicts that the future of single-use mixing systems design is leading more toward higher quality and increased quality assurance. Building on those protective capabilities in the design, Corti stated that many end users are asking for things such as point-of-view sleep testing and the ability to confirm even further that, once the consumables are installed in the system, there is a guarantee that there will be no leaks.

INTERPHEX 2024 was held in New York City on April 16–18, 2024.

Click here for more coverage from INTERPHEX 2024

Global product manager at Cytiva, Eric Corti, discusses the challenges with designing a new single-use mixing system that ensures leak-free fluid handling operations at INTERPHEX 2024.

PharmTech Interview with Dennis Powers from G-CON at INTERPHEX 2024

As biologics continue to experience growth in the biopharma industry, access to appropriate cleanroom facilities that can provide a suitable and sterile environment is increasingly in need. In an interview at 

 spoke with Dennis Powers, senior vice-president, Product and Strategy, G-CON Manufacturing to discuss podular cleanroom solutions.

Primarily, G-CON had developed a standard POD cleanroom platform for the cell and gene therapy field, where a smaller footprint can aid in improving efficiencies. “The standard pods are well suited for cell therapy applications, small footprint aseptic, filling applications, [and] process development labs,” Powers said. Over the years, the company has developed a number of prefabricated designs that can be applied to different applications and customized depending on the manufacturers’ needs, he added.

With regards to the market for prefabricated podular/modular cleanroom solutions, Powers noted that there is definitely a rise in the number of companies in the field now. “We’re certainly seeing the industry starting to recognize the value in prefabricated modular construction,” he stated.

Additionally, modularization is not only changing facilities but is also being implemented into processes as well, Powers remarked. “So, as processes become more modular and more configurable [with a] smaller footprint, they fit very well into a prefabricated modular cleanroom infrastructure,” he said.

However, given the fact that the biopharma industry is more conservative than others, adoption of new technologies and approaches can be slower. “So, you have to not only have a great product, but you also have to educate the industry on that product, the value that product brings, and convince them why it's better than what they've been using traditionally,” Powers emphasized.

While at the show, the company launched the floorlessPOD, which is a freestanding structure that can be installed directly to the host facility floor, Powers explained. “[The floorlessPOD] allows us to eliminate the need for ramps platform steps to get up into the cleanroom, [and] it allows for direct integration into sub-slab utilities floor drains,” he said.

At INTERPHEX 2024, Pharmaceutical Technology® chatted with Dennis Powers from G-CON Manufacturing about podular cleanroom options and the benefits they offer for manufacturers.