Mike Hennessy Jr. is the President and CEO of 

Mike Hennessy Jr.

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On Aug. 14, 2024, the World Health Organization (WHO) declared mpox (previously called monkeypox) as a public health emergency of international concern (PHEIC) as a result of the increasing rates of infection that are being witnessed in African countries (1). The International Health Regulations Emergency Committee of independent experts had advised WHO on the level of concern about the spread of the virus and the potential for the virus to penetrate other continents.

Two years ago, in July 2022, the multi-country outbreak of mpox was also declared a PHEIC; however, after less than a year, in May 2023, that PHEIC was declared over as a sustained decline in global cases was seen (1). A new virus strain of mpox, clade 1b, has emerged and has been rapidly spreading.

When the first PHEIC was declared for mpox in 2022 for a less deadly strain of the virus, the bio/pharma industry went to bat quickly and utilized experience from the COVID-19 pandemic to work efficiently. One of the main players from the 2022 outbreak, Bavarian Nordic, is proving its worth once again, committing to donate 40,000 doses of its mpox vaccine to the Africa Centers for Disease Control and Prevention (2). “Mpox is spreading at an alarming rate in Africa, calling for further action from the international community,” said Paul Chaplin, president and chief executive officer of Bavarian Nordic, in a press release (2).

The 40,000 vaccines from Bavarian Nordic will form part of a larger donation of 215,000 doses that will be provided by the European Commission’s Health Emergency Preparedness and Response Authority (3). However, many more vaccines will be needed, and WHO has triggered the Emergency-Use Listing process for mpox vaccines and started working with countries and vaccine manufacturers on potential donations (1).

“The emergence of a new clade of mpox, its rapid spread in eastern DRC [Democratic Republic of Congo], and the reporting of cases in several neighbouring countries are very worrying,” warned Dr Tedros Adhanom Ghebreyesus, director-general of WHO, in his PHEIC declaration announcement (1). “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.”

1. WHO. WHO Director-General Declares Mpox Outbreak a Public Health Emergency of International Concern. Press Release, Aug. 14, 2024.
2. Bavarian Nordic. Bavarian Nordic Receives Order from HERA and Announces Significant Donation of Vaccines in Response to the Mpox Outbreak in Africa. Press Release, Aug. 13, 2024.
3. EC. Mpox: HERA to Donate Over 215,000 Vaccines to Africa CDC Amid Urgent Outbreak. Press Release, Aug. 14, 2024.

Mike Hennessy Jr is president and CEO of MJH Life Sciences®

When referring to this article, please cite it as Hennessy, M. Responding to Mpox. 

Articles

A coordinated and international response is needed to help control the latest mpox outbreak in Africa.

Responding to Mpox

This year has indeed been a bountiful year of sport already, with the recent finale to the EURO 2024 soccer competition seeing Spain setting the record for the most titles and the United States co-hosting the T20 World Cup cricket tournament for the first time, to name but a few. However, the most impressive sporting events that have a global audience are the Olympic and Paralympic Games, which are taking place in Paris, France from July 26 until Aug. 11, 2024 and Aug. 28–Sep. 8, 2024, respectively.

Host city Paris has waited 100 years to welcome the Olympic Games back and has never before been the host for the Paralympic Games, which should make for some historic and memorable occasions over the next few months. Sanofi, which is a giant in the field of vaccines and anti-inflammatory treatments, is not usually involved with sporting or cultural events, but has made an exception for the 2024 games being held in the city where the company is headquartered (1).

Younger viewers are increasingly being attracted to watch the games—thanks to the inclusions of sports such as breakdancing and skateboarding—which is something that Sanofi, as a premium sponsor for the 2024 games, hopes to use to its advantage. “We’re using the Olympics to show that we are [cool],” said Josep Catlla, head of corporate affairs at Sanofi, when speaking with Reuters (1).

It is hoped that, through the increased awareness gained from sponsoring the games, the company will be able to attract new talent and remove its conservative image (1). In 2022, Sanofi committed to its ‘ignite the potential in everyone’ campaign for the Paris 2024 games (2). This campaign is aimed at demonstrating the similarities between science and sport, such as overcoming challenges to continue to progress forwards, and to make science inclusive and open to all (3).

We will have to wait and see whether Sanofi’s investment in the Olympics and Paralympics will pay off in the long run with the public or their investors, and whether or not it will be possible to ignite the potential of pharma for the next generation of scientists.

1. Naidu, R.; Burger, L. Drugs Group Sanofi Makes Olympic Jump in Rebrand Bid. Reuters, Jan 10, 2024.
2. Sanofi. Sanofi Commits to Ignite the Potential in Everyone. 

When referring to this article, please cite it as Hennessy, M. Igniting Pharma’s Potential. 

Will Sanofi’s plan to recruit the younger generation through its participation in the upcoming 2024 Olympic and Paralympic Games, being held in Paris, pay off?

Igniting Pharma’s Potential?

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On June 20, 2024, FDA announced that it had expanded its approval of delandistrogene moxeparvovec-rokl (Elevidys), a gene therapy for the treatment of Duchenne muscular dystrophy (DMD), to cover both ambulatory and non-ambulatory DMD patients of four years and older with a confirmed mutation in the DMD gene (1). This expanded approval will allow for a greater number of patients suffering from DMD, which is a rare and serious genetic condition that progresses over time, to be treated. In addition, the expanded approval will help to meet the urgent unmet medical need of this patient population.

The area of rare, difficult-to-treat diseases is where cell and gene therapies truly shine, offering treatment options where there have previously been none and in some cases, even the potential to eliminate the disease altogether. Given the promise of these advanced therapies, it is no wonder that the global market is expected to witness significant double-digit growth over the next few years (2).

However, as the cell and gene therapy sector continues to mature, greater attention is being paid to increasing therapeutic efficacy while also improving patient safety. This particular topic has been explored in this issue’s cover story on pages 10–12 (3), where experts reveal some of the strategic approaches being used to overcome constraints and challenges related to emerging therapies, such as reducing vector tropism to reduce liver damage or regulating the immunoreceptor complex with a dimerizing agent.

As researchers from all over the industry and regulatory bodies continue to focus on their investigations into approaches and strategies to improve not only the therapies themselves, but also methods to test these novel drugs, their efficacy and safety for patients will be optimized, and more unmet medical needs will be fulfilled.

1. FDA. FDA Expands Approval of Gene Therapy for Patients with Duchenne Muscular Dystrophy. Press Release, June 20, 2024.
2. Research and Markets. 

Cell and Gene Therapy Global Market Report 2024

. Market Report, February 2024.
3. Mirasol, F. Taking Stock of a Maturing CGT Sector. 

When referring to this article, please cite it as Hennessy, M. With Age Comes Wisdom. 

A greater number of patients with Duchenne muscular dystrophy will be able to be treated after FDA's approval of a gene therapy.

Publisher's Note: With Age Comes Wisdom

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Despite the bio/pharma industry’s stringent regulations and general tendency to adopt newer technologies more slowly than other industries, automated processes and robotics are increasingly commonplace in manufacturing facilities. Although initially used to improve efficiencies of repetitive tasks, automation and robotics have become more sophisticated and are now being employed for a much wider variety of tasks, such as quality control, optimizing processes, and analyzing vast quantities of data (1).

A particular benefit of automating processes and/or using robotics is the reduction of human intervention and, thereby, human error or contamination. This benefit is particularly important for aseptic processing, where particulate generation must be kept to a minimum to ensure the safety and sterility of the pharmaceutical product being manufactured.

The utility of automation to improve efficiencies in aseptic processing is examined in greater detail in this issue’s cover story, which is featured on pages 10–13 (2). “Automating processes and bringing robotics into aseptic processing, together with single-use systems, [are] helping to streamline processes, minimizing human intervention and reducing the risk of errors or contamination,” explains Aimee Hodge from PCI Pharma Services in the article (2).

In the cell and gene space, a deal between Astellas Pharma and YASKAWA Electric Corp. has been formed that will focus on the digitization of cell manufacturing through robotics (3). Under the terms of the agreement, the two companies will seek to develop a platform that will use a humanoid robot, Maholo, to link early-stage research to commercialization of cell therapies (3).

Additionally, once created, the companies would consider allowing startups and academia to use the platform, which could lead to the development of an advanced therapy ecosystem that nurtures innovation. Startups and academia are not always able to commercialize their cell therapy discoveries as a result of the significant cost and manufacturing challenges that are inherent with these complex drugs (3).

1. Praxie. Transforming the Industry: The Advantages of Automation in Pharmaceutical Manufacturing. 

2. Thomas, F. Streamlining Aseptic Processes through Automation. 

3. Astellas Pharma. Astellas Announces a Collaboration with YASKAWA to Create an Innovative Cell Therapy Ecosystem through the Integration of Pharmaceutical and Robotics Technologies. Press Release, May 21, 2024.

When referring to this article, please cite it as Hennessy, M. Automating Processes. 

Improved efficiencies and reduced costs are clear advantages of automating drug manufacturing processes.


Publisher's Note: Automating Processes

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Research efforts are continuing to expand in the area of biological therapies. These large-molecule drugs are proving to be significant revenue drivers for bio/pharma companies and are providing therapeutic options for disease areas that have previously suffered from unmet medical needs (1).

Additionally, biologics are useful in providing more targeted disease treatment when compared with small-molecule drugs and lessening the risk of side effects for patients. The targeted nature of these large-molecule therapies is explored in this issue’s cover story, featured on pages 10–13, along with the potential that technological advances hold in pushing future boundaries and some of the challenges facing both the development and manufacture of biologic drugs (2).

Advanced therapy medicinal products (ATMPs), which are promising and often curative biologic therapies, face their own specific development and manufacturing challenges. Scalability of manufacturing for these emerging therapies is a particular bottleneck and something that San Francisco-based Cellares, a self-titled integrated development and manufacturing organization (IDMO), has sought to change. In an interview with Pharmaceutical Technology® Group, Fabian Gerlinghaus, CEO and co-founder of Cellares, detailed how the company’s Cell Shuttle platform and integrated consumable cartridge automated platform can improve scalability of ATMP manufacturing (3).

The IDMO has also garnered interest from Bristol Myers Squibb (BMS), which joined the Technology Adoption Partnership (TAP) program in August 2023 (4). Participation in the TAP program has meant that BMS can use Cellares’ automated manufacturing platform for the manufacture of its pipeline products.

Most recently, in April 2024, BMS and Cellares built upon their relationship by entering into a deal worth $380 million, where Cellares will transfer select chimeric antigen receptor T-cell therapies onto its manufacturing platform, with the aim of meeting growing demand and improving turnaround time (5).

1. Bender, M. Pharmaceutical & Life Sciences Trends 2024. 

 (accessed April 24, 2024).
2. Thomas, F. Biologics On Target for the Future. 

, 48 (5), 10–13.
3. Spivey, C. Cellares Innovates with Automated Cell and Gene Therapy Platform. 

, March 7, 2024.
4. Cellares. Cellares Announces Bristol Myers Squibb has Joined Technology Adoption Partnership Program to Evaluate Automated Manufacturing of CAR-T Cell Therapy on the Cell Shuttle Platform. Press Release, Aug. 28, 2024.
5. Cellares. Bristol Myers Squibb and Cellares Announce a $350M Worldwide Capacity Reservation and Supply Agreement for the Manufacture of CAR T Cell Therapies to Bring the Promise of Cell Therapy to More Patients, Faster. Press Release, April 22, 2024.

When referring to this article, please cite it as Hennessy, M. Tapping into the Potential of Biologics. 

Technological advances are helping advance biologics development and manufacturing and reduce bottlenecks.

Tapping into the Potential of Biologics

Experts believe it is likely cancer cells are more common in the body than previously thought, but typically caught early and removed in the body’s cancer-immune cycle. When the system is in good working order (and protecting us), dead cancer cells release neoantigens, which are acquired by dendritic cells and moved to a nearby draining lymph node for disposal. Dendritic cells then present this antigen to T cells, which become effector T cells, and after a few more steps, “move through the blood toward the tumor tissue. These T cells then infiltrate into the tumor and recognize and bind the MHC-I–antigen complex presented by cancer cells through the T cell receptor. Through these processes, cancer cells are finally killed. …When cancer cells die and additional neoantigens are released, the immune reaction continues the cycle again from the first step. [However], in cancer patients, at least one of these steps is defective and the cancer-immune cycle does not work properly” (1).

The obvious utility in identifying tumor neoantigens is consolidating in weight and importance, leading to coordinated leapfrog advances in pharmaceutical sciences. But each patient has their own personal biology, creating their own personal tumor microenvironment (e.g., there are more than 24,000 distinct human leukocyte antigens) (2). A paper describing a powerful new identification platform was briefly discussed in an interview with 

® (3), where study author, Stephen Schoenberger, told 

 editor “we let the patient’s immune system tell us which mutations can be confirmed as neoantigens and then have asked whether there are bioinformatically quantifiable features of these mutations apparent in genomic sequence data. Our goal in doing so is a computational prioritization algorithm of sufficient predictive power as to obviate the need for 

 functional validation step, as this would be difficult to scale for the number of patients that our findings suggest could benefit from personalized immunotherapy” (3).

Kim, S.; Cho, S. The Evasion Mechanisms of Cancer Immunity and Drug Intervention in the Tumor Microenvironment. 

. 2022 May 24:13:868695. DOI: 10.3389/fphar.2022.868695. eCollection 2022.

Fu, L.; Huang, C.; Huang. Z. Neoantigens: Promising Targets for Cancer Therapy. 

. 2023 8 article 9. Nature.com, https://www.nature.com/articles/s41392-022-01270-x

Spivey, C. Research into Cold Cancers Heating Up. 

When referring to this article, please cite it as Hennessy, M. May I Present the Neoantigen. 

Experts believe it is likely cancer cells are more common in the body than previously thought, but typically caught early and removed in the body’s cancer-immune cycle. When the system is in good working order (and protecting us), dead cancer cells release neoantigens. 