I have young kids. That means I’m often immersed in the land of make-believe, where anything is possible, and animals have magical powers.
Lately, I’ve noticed that my kids’ world has more in common with the reality of life science R&D than ever before. Anything is becoming possible in terms of drug discovery and development — consider, for example, that the FDA now expects to approve up to 20 new cell and gene therapies a year. As a result, novel products designed to cure cancer, eliminate type 1 diabetes, regulate cholesterol production closer to the source, or otherwise “do the impossible” are constantly emerging.
As for animals with magical powers, researchers at the University of Maryland transplanted the heart of a CRISPR genetically modified pig into a human for the first time earlier this year. Life science R&D breakthroughs like these have brought us to the cusp of a drug discovery revolution that will change how we improve and extend the life of critically ill patients.
What will it take for life science companies to keep up with this discovery rate? To help us answer that question, my colleagues and I recently surveyed nearly 500 developers and manufacturers for our latest Horizons: Life Sciences report. We asked them about the challenges they’re facing today and what they’re doing to maintain their life science R&D momentum.
Our survey results reveal four significant shifts underway in life science R&D:
1. Drug developers are embracing a multimodal future as their product pipelines grow more complex
Most of our survey respondents have the full gamut of modalities in development, and they’re looking for manufacturing approaches that push beyond traditional models to better support this degree of pipeline complexity.For example, 90% of our survey respondents are currently manufacturing or developing multiple therapy modalities in a single building or are planning to in the future; about the same proportion are open to leasing pre-constructed cleanroom space to further improve their speed and scalability. Meanwhile, more than half of respondents rely on CDMOs as a core element of their manufacturing strategy, and we’re seeing a rise in hybrid owner/CDMO operations.Each of these shifts is a response to the headwinds facing today’s industry. Manufacturers are under pressure to beat competitors to market; simultaneously, they’re contending with a pinched supply chain, a battle for talent, and limited GMP space. From this context, a new way of manufacturing is emerging — one designed to maximize scalability, flexibility, and resilience.
2. Interest in RNA-based therapies is surging
The pandemic pushed RNA-based vaccines into the spotlight. Still, the broader versatility of RNA technologies is attracting attention today, as well as their potential to improve speed to market and lower costs. Drawn by these advantages, 55% of our survey respondents have (or plan to have) coding and non-coding RNA-based therapies in their pipeline.This submarket is facing substantial challenges, though. For one thing, manufacturers must choose the right synthesis approach that will maximize purity/yield while maintaining cost control. With regulatory thresholds evolving rapidly and little standardization to guide R&D, such decisions are freighted with risk and require an experienced, strategic approach to succeed.
3. Cell and gene therapy manufacturing is coming of age
There may be no better words to describe this field than simply “rapid change.”This is especially true for gene therapy manufacturers. While 58% of our survey respondents are manufacturing gene therapies at a scale of less than 250 L, a third expect to exceed 750 L within the next three years. This is an exciting prospect for a relatively new submarket; the challenge lies in how gene therapy manufacturers will reach those volumes. In response to this challenge, many survey respondents are evaluating, using, or planning to use scalable, next-generation technologies such as sterile filtration (95%) and stable cell producer lines (93%)—an exciting prospect for a submarket on the verge of immense growth.
Those with cell therapies in their pipeline—particularly autologous cell therapies, which constrain manufacturers with their limited scalability and complex cold chains—are also facing pressure to improve patient access. This could explain why 77% of survey respondents working with cell therapies have a platform process in place or plan to develop one in the next five years. Decentralized manufacturing is also an attractive possibility for autologous cell therapy manufacturers; 95% say they are considering a hospital or clinic partnership. Solutions like these may be the key to rapid growth and commercialization for this thriving market.
4. Digitalization is king
Underpinning these shifts is an overall push toward deeper digital integration and data-driven processing. Our survey respondents are part of this step change. When asked about the five-tiered Digital Plant Maturity Model (with 1 being a pre-digital plant and 5 being a fully adaptive facility of the future), two-thirds positioned themselves at Level 3 or 4, and nearly a quarter plan to reach Level 5 within a few years.To get there, manufacturers need to navigate constrained capital budgets, internal reluctance, and competition for employees skilled in designing and maintaining autonomous, self-optimizing systems. The potential payoff is great, though: those at the forefront of this digital revolution will see improved speed, quality, and scalability.
Toward a future of life-saving medicine
The life sciences market is thrumming with excitement at the moment. We continue to find new applications for well-established biologics (last year, the FDA approved its 100th monoclonal antibody product); meanwhile, researchers are pursuing all-new concepts like in vivo genetic editing. As a result, the goal of eliminating incurable diseases is no longer the stuff of make-believe. Instead, this could be our real future with good planning, strategic partnerships, and an imagination that reaches beyond today’s limitations.
Noel Maestre is CRB‘s biotech subject matter expert, emphasizing Advanced Therapy Medicinal Products (ATMPs). He has an extensive background in mechanical and process utilities engineering with experience in the life science industry. He specializes in the design, construction and startup of biotechnology, pharmaceutical and advanced technology facilities. Maestre is a graduate of Pennsylvania State University with a Bachelor of Science in mechanical engineering.
Filed Under: Cell & gene therapy