The Logistics of Developing an Emerging Therapy

When entering the discovery and early development phases of a medication, a company needs to look beyond simply detecting a disease or ailment that could benefit from a novel therapy. There are several factors that should be considered when developing a new modality.

One of these considerations is manufacturing, according to Wade Macedone, CEO of Andelyn Biosciences. “Translating a potential treatment from the laboratory to a therapeutic requires deep knowledge of manufacturing science and technology (MS&T) as well as process development, analytical development, quality and regulatory disciplines,” says Macedone. Viral vector delivery of gene therapies, for example, have manufacturing requirements that are different from antibodies and other modalities. “Everything from the delivery vector, targeting specificity of the serotype, the size of the therapeutic payload and dosing, to the particulars of upstream cell culture and downstream purification will vary from therapy to therapy,” says Macedone. “This is why expertise across the manufacturing disciplines is so important.”

Cell and gene therapies require balance

There are two types of cell therapies, explains Michael Pinaud, senior vice president, Business Operations, Ascend Advanced Therapies; autologous cell therapies use patient cells and allogeneic cell therapies use donor cells. Autologous products have more specific logistical challenges. “Material is first collected at an apheresis center, then sent to a central manufacturing location, processed, and finally back to the patient for administration. All this movement takes time and cost and requires maintenance of the chain of identity to ensure the right product gets to the right patient,” says Pinaud. Allogeneic, in comparison, have fewer steps but are costly to produce.

“For either type of cell therapy, costs will be driven by indication, dose required, in vitro or in vivo therapy, etc.,” says Pinaud. “Development costs are also very much dependent on the number of patients required for clinical development and the dose.”

Gene therapies, which are also complex, bring in a new or modified gene, turn off a gene, or replace a gene, according to Pinaud. “Most are delivered using viral vectors, which are costly to produce due to challenges with scalability, productivity, and overall recoveries. Limited availability of some raw materials (e.g., plasmids for transient transfection) is also an issue. Fortunately, progress is being made along many fronts, with contract development and manufacturing organizations (CDMOs) taking a lot of the lead in investing in development of optimized and flexible platform manufacturing processes and stable producer cell lines.”

A new cell or gene therapy may require many years and high costs, according to Macedone, with cost varying from indication to indication. Often rare diseases also impact children, which might involve fundraising and patient foundations, Macedone points out, which may aid in costs. “There is always a balance, and every program has its idiosyncrasies. This is where the power of a well-designed modular platform comes into play,” says Macedone.

Read the article in the Emerging Therapies eBook.

About the author

Susan Haigney is lead editor for BioPharm International®

Article details

BioPharm International®
eBook: Emerging Therapies
September 2024
Pages: 22–26

Citation

When referring to this article, please cite it as Haigney, S. The Logistics of Developing an Emerging Therapy. BioPharm International Emerging Therapies eBook 2024, September.