Cell and gene therapies (C>s) comprise a rapidly expanding segment of the life science industry. Due to this growth, companies can encounter significant challenges when scaling their therapeutics from research through clinical trials to commercialization.
The C> industry is working to standardize manufacturing processes and practices, and here at Aldevron we have joined forces with multiple industry consortia to help address these challenges.
Many C> manufacturers face a looming crisis of capacity and expertise. According to a recent statistic from BioPharm International, demand for C> manufacturing capacity is currently estimated to be five times the available space. This limitation is only expected to grow in the coming years as certain in-demand cell and gene therapy workflows, specifically viral vector manufacturing, are predicted to see an estimated 20% year-over-year growth through 2025 as reported by CRB. Rapid growth of the C> pipeline in the last five years means pressure on capacity is to be expected, but lack of standardized manufacturing practices will only continue to hinder the industry’s ability to keep up with demand and plan for expected additional growth.
As with many emerging biopharmaceutical technologies, companies often work independently to build manufacturing facilities and develop processes. In some cases, this leads to adoption of inefficient short-term solutions, such as fixed-bed reactors or custom reagents, two problems common to multiple therapeutics.
Additional challenges, such as sourcing adequate raw materials at the appropriate quality level, means that C> companies can also struggle to transition from R&D to pivotal clinical trials. This, in turn, limits the speed at which they can ultimately bring therapeutics to the market. With the C> program pipeline now more well established and manufacturing capacity already constrained, the industry has an urgent need to standardize.
Working together across the industry
Today, the C> industry is coming together to standardize manufacturing practices and solve other common industry challenges. At Aldevron, we are leveraging our 23-plus years of experience partnering with leading researchers and organizations to help lead the way with other industry colleagues and institutions.
We have listened to our clients and partnered with several major C>-focused industry consortia, with the aim of solving common problems and driving changes in the industry. Our subject matter experts routinely share expertise in manufacturing, validation and other relevant areas at meetings, conferences, and working groups – both online and in person.
Aldevron is an active member of the Alliance for Regenerative Medicine (ARM), the International Society for Cell and Gene Therapy (ISCT), American Society of Gene and Cell Therapy (ASGCT) and International Gene Synthesis Consortium (IGSC).
Since 2018, Aldevron has also been a member of BioPhorum’s Cell & Gene Therapy Phorum. Within this Phorum, we have recently contributed to two industry publications, Gene and Cell Therapy Critical Starting Material: A Discussion to Help Establish Release Specifications for Plasmids and the Bacterial Master Cell Banks Used to Produce Them in December 2020 and Cell & Gene Therapy Considerations when Assigning Responsibilities During a Product Transfer Between a Sponsor and a Contract Development Manufacturing Organization in April 2021, with two additional publication papers scheduled for release in Q4 2021.
Learning through partnership
Our involvement in C> industry consortia has allowed us to openly discuss current challenges and opportunities for improvement with our industry colleagues. By better understanding standardization challenges and other common industry problems, we can take the lead in developing new products and services to better serve our clients and support them while addressing these challenges.
A key initiative at Aldevron has been to develop standardized biologic reagents for the C> industry. These include our CRISPR/Cas9 nucleases, which are designed to be ready to use as a standardized product without the need for further customization. Our adeno-associated virus (AAV) and lenti plasmid systems, meanwhile, are also standardized products, and only require customized manufacturing of the client’s gene of interest.
Both our plasmid and Cas9 products are available at research and clinical quality levels. Each product line further supports standardization by maintaining consistent manufacturing processes and release specifications between quality levels, meaning our clients can use them throughout a project lifecycle from research to clinical stage and, ultimately, to therapeutic commercialization.
As active participants in C> consortia such as BioPhorum and others, Aldevron seeks to support the next generation of researchers and biopharmaceutical companies. Our standardized products are intended to help early-stage researchers avoid the need to develop a custom protein or plasmid construct for some, or all, of their biologic reagent requirements. Instead, they can use a standardized set of tools to more rapidly bring their products to market.
Sharing our knowledge
One of the largest standardization challenges today is being faced by companies whose C> products are already undergoing clinical trials. These companies have already developed their protein or DNA reagents and their manufacturing workflow and cannot easily change, for example, one or more of their plasmid constructs.
It is for this reason Aldevron has proactively joined industry consortia – to work in partnership with colleagues to develop updated recommendations that can best support therapeutic research and development. We advise our clients to look at standardization as early as possible within their programs and we encourage investment early in scalable and standardized technologies so they are well positioned to move from research into clinical trials and ultimately commercialization in the future.
Aldevron: Empowering the Industry
Aldevron has actively supported the C> industry since our founding over 20 years ago. We truly see the value of industry consortia as they promote a collective approach to overcoming challenges and shaping the future. Standardization provides a clear path to bring relevant therapeutics rapidly to the market, and ultimately, supplying patients faster access to life-changing healthcare.
Aldevron’s expertise and experience together support our current and prospective partners, empowering solution-based innovations that benefit us all. Contact us today to learn more!