Manufacturing Nuances for Transformative Medicine

Co-authored by Joe Neroni, Sr. Director and Sarah Stoll, Sr. Director

In the rapidly evolving biopharma industry, selecting the right manufacturing method is crucial for life-saving success and avoiding costly delays.

Companies face numerous innovative options for creating leading-edge therapeutics, each presenting unique challenges and opportunities.

The production of radioligands (RLTs), mRNA therapies, cell therapies, and gene therapies demands complex procedures, specialized equipment, and top-notch expertise.

Attracting experienced talent and continuously training a dedicated workforce are critical across these modalities.

Drug developers must consider facility location, shipping, and supply chain logistics to ensure timely drug delivery to patients. Additionally, scaling up or out can significantly impact the time required to deliver these intricate treatments.

Understanding the nuanced differences and industry trends in producing these modalities helps companies strategically plan their approach, ultimately accelerating the delivery of life-saving treatments to patients in need.

Radioligands: Promising Therapeutics, Complicated Logistics

In oncology research, radioligand therapies (RLTs) provide an innovative solution to the problem of how to attack cancer cells without damaging healthy cells.

Although it is a lifesaving therapy, RLTs are difficult to produce and distribute.

The last several years have seen an explosion of research and clinical trials testing new radiopharmaceuticals.

Due to their novelty, one of the most obvious challenges plaguing RLTs is the shortage of skilled workers with deep knowledge or experience on how to produce them.

Radioligand therapy companies can bridge the talent gap by partnering with academic institutions and research centers, offering internships, and fostering collaborations to attract and train professionals with expertise in nuclear medicine, radiopharmaceuticals, and related fields.

They can also invest in ongoing education and development programs for existing employees to enhance their skills and knowledge in this specialized field.

Radiotherapy companies can address the challenge of time in shipping to patients by optimizing logistics and strategically locating their manufacturing facilities.

Having facilities in metropolitan locations can expedite shipping due to better access to transportation networks. This proximity reduces transit times, ensuring timely delivery of radiotherapy equipment and materials.

Additionally, a metropolitan location can provide access to a skilled workforce and resources, further streamlining production and distribution processes.

Efficient logistics and strategic facility placement contribute to overcoming time-related challenges in the delivery of radiotherapy services, especially for the 'last mile': getting the drugs to patients.

Companies planning to invest in RLT drugs should consider these factors and strategize their logistics early to avoid 'last mile' holdups.

mRNA Drugs: Timing is Everything

Like radioligand therapies, mRNA therapeutics are both highly promising and logistically complex.

In the past few years, mRNA therapies and vaccines have gained notoriety for their potential to deliver genetic information and transiently deliver proteins to treat genetic diseases, tumors, and viral infections. However, sales and operational planning (S&OP) and demand management are intricate processes impacted by post-pandemic vaccine uptake and demographic impacts on the demand for booster vaccinations.

The need for multiple treatments may pose challenges in maintaining a durable therapeutic response.

Researchers are exploring ways to enhance the longevity of therapeutic effects through modified mRNA sequences or repeated dosing.

Manufacturers of mRNA therapies can stay adaptable for patient demand by establishing flexible production processes that quickly respond to changes in dosage requirements.

Manufacturers can also emphasize collaboration between researchers and patient advocacy organizations to accelerate the development of mRNA therapies that promise efficacy and longevity, reducing the need for frequent boosters.

Ultimately, the landscape of mRNA therapeutics is marked by both promise and complexity.

The potential to address various medical challenges, from genetic diseases to viral infections, is evident. Yet, the intricacies of S&OP and demand management, especially with the need for multiple treatments, add a layer of complexity.

Emphasizing collaboration with researchers and patient advocacy organizations further paves the way for the development of mRNA therapies that not only ensure efficacy but also reduce the necessity for frequent boosters, ultimately enhancing their impact on patient health.

Cell Therapies: Bespoke Treatments Need Custom Solutions

Cell therapies offer patients custom-made treatments for otherwise incurable diseases and conditions, but developing these therapies can be complicated under even the best circumstances, as supply chain, cost, and foundational science obstacles continue to impact the industry.

Nonetheless, the promise these therapeutics hold continues to spur research and investment, driven by forward-thinking approaches and new technologies designed to optimize these applications for cost and time.

To produce cell therapies, companies may opt to use autologous (stem cells from the patient receiving the treatment) or allogeneic (stem cells from a healthy donor).

Because both methods of producing cell therapies are equally important to saving patient lives, companies face additional manufacturing nuances when determining the mode of donor cell retrieval.

Autologous and allogeneic cell therapies encounter common fundamental challenges.

While the personalized nature of cell therapies is crucial for treating complex diseases and severely ill patients, it poses obstacles to manufacturing scale-up.

Companies can employ various strategies to maximize patient cells in cell therapies and overcome manufacturing scale-up challenges:

1. Improved patient identification: Streamline the process of identifying suitable patients by developing more accurate diagnostic and patient selection criteria, allowing for a larger pool of potential candidates.

2. Automation and standardization: Invest in automation and standardized protocols to enhance the efficiency of cell processing and reduce the risk of errors during manufacturing, which can help scale up production.

3. Scalable manufacturing platforms: Develop adaptable and scalable manufacturing platforms that can handle varying patient cell quantities without sacrificing quality or personalized aspects of the therapy.

4. Process optimization: Continuously refine and optimize manufacturing processes to minimize waste, reduce costs, and improve the yield of viable cell therapies.

5. Supply chain optimization: Ensure efficient and secure supply chains for sourcing patient cells and delivering the final therapies to treatment centers, which is crucial for scaling up.

By implementing these strategies, companies can strike a balance between personalization and scalability, making it possible to maximize the use of patient cells in cell therapies.

Gene Therapies: From Bench Research to Commercial Manufacturing

Creating gene therapies is an intricate process that does not easily translate from bench research to commercial distribution.

Unfortunately, the fundamental scale-up processes that manufacturers employ for other drugs don’t apply to gene therapies, i.e., the yield does not necessarily increase in proportion to the amount of raw material used.

The difficulties in producing gene therapies on a commercial scale affect demand, footprint, cost, and throughput.

One solution is to partner with an academic or R&D team early in the development process to create a feasible manufacturing procedure and implement CMC-specific equipment.

Working with expert researchers helps companies create a methodology that smoothly transitions from bench research to commercial production. Meanwhile, technology is rapidly catching up with the science behind gene therapies, creating opportunities for scaling up rather than scaling out.

Final Thoughts

Ultimately, successfully navigating the intricate landscape of manufacturing transformative medicines demands a multifaceted approach.

The complexities of logistics driven by the need for precision in speed must be met with innovative manufacturing solutions that can swiftly adapt to evolving patient needs.

Furthermore, the active involvement of patients and shaping their treatment journey while raising awareness of the transformative potential of these medicines is crucial.

Biopharma companies must continually reevaluate their manufacturing processes as technology evolves to keep up with industry best practices and remain competitive in an aggressive arena.

It is equally important to keep pace with the evolving regulatory and quality standards to ensure risk-based approaches to manufacturing adhere to evolving cGMPs.

Working with an experienced partner to help understand the nuances in the industry and the unique complications inherent to these popular, innovative modalities is the first step in determining an efficient and cost-effective approach to revolutionize treatment.

Click here to download our recently published Precision Manufacturing Ebook for more patient-focused manufacturing insights.

About Precision for Medicine

As the preferred solution provider for global developers, Precision has successfully industrialized 10+ commercial cell, gene, and novel therapies, 60+ facility builds and capital expansions, and $6B+ in technical operations capital investments. Our manufacturing team has the proven track record and expertise to provide industry-leading technical operations and manufacturing solutions to shorten time-to-market for patients in need.

Visit our website to learn more about Precision for Medicine’s integrated capabilities across labs, clinical trials, manufacturing, and data intelligence to deliver critical insights for life-science innovators.