iSpeak Blog

The Need for Speed: Reducing Risk, Cost, and Delays for Novel Gene Therapies

Amrit Chaudhuri
The Need for Speed: Reducing Risk, Cost, and Delays for Novel Gene Therapies

Novel cell and gene therapies are already revolutionizing medicine. These new therapies can modify a patient's DNA and transform their cells into powerful tools for treating a wide range of diseases, from cystic fibrosis and heart disease to diabetes, hemophilia, and cancer. The introduction of groundbreaking technologies such as CRISPR gene editing has already produced significant improvements in the treatment of patients, and as this technology continues to evolve, gene therapies hold the potential to provide even more miraculous results.

While novel gene therapies have the power to revolutionize medicine, progress is limited by the speed at which scientists can identify targets, develop therapeutic assets, and implement new manufacturing procedures. One major factor that acts as a barrier to progress is the time and cost associated with setting up a lab. Labs are the backbone of R&D, but the labs of today are not designed with the flexibility to accommodate the demands of innovative therapeutics. By focusing on the backbone of R&D – labs – scientists can dramatically accelerate their quest for cures for the more than 7,000 known genetic diseases.

Hurry Up and Wait

All research begins in a lab, but few companies or researchers have focused on how to make labs more productive for research. Gene therapy researchers aiming to bring their innovation to market must divert significant human, financial, and logistical resources to building and operating lab spaces. Planning, designing, and constructing a new R&D lab can take anywhere from two to five years or longer, significantly delaying the process of developing new therapeutics. Companies that build their own research facilities can spend more than $400 million, equivalent to $40 million per year for a decade, before obtaining their first usable data. A big part of the issue is that companies must anticipate the unknown, attempting to predictively develop scaled and massively expensive resource sets for a future they cannot reliably predict. Biopharmaceutical companies and research institutions need a new paradigm for designing, building, and operating facilities that can power their innovations and bring them to market swiftly and efficiently.

The Outsourcing Revolution

Existing scientific research facilities present a significant challenge due to their inflexible structure and specialized nature, limiting the ability of biopharmaceutical companies and research institutions to pivot easily. This often leaves those organizations with only two options: either retrofit an existing space or build a new one. Both choices can be prohibitively expensive, even for well-funded organizations, and may not yield a positive return on investment in the long run. The time-consuming process of building a new facility is not well-suited for gene therapies, which requires speed and agility. Even if an organization predicts a surge in demand for specific therapeutics and builds a dedicated facility, the demand may not materialize, may be limited in duration, or the infrastructure may be ill-suited for the required workflows, leading to a huge waste of time and capital.

Recognizing this need for speed and adaptability, contract research organizations (CROs) and contract development & manufacturing organizations (CMO/CDMOs) have marketed themselves as a solution for biopharmaceutical companies seeking to accelerate the rate at which they complete drug discovery, development, and clinical and commercial production. Hiring an outsourcing partner enables those organizations to delegate parts of their work to contracted partners while continuing to work on their own projects and focus their internal research teams on innovation. This approach is supposed to help biopharmaceutical companies avoid bottlenecks and complete their research more efficiently.

There are certain drawbacks to this approach, however. In theory, CROs and CDMOs stand ready to perform drug development activities in prime facilities, on behalf of biopharmaceutical companies and research institutions. In practice, however, these facilities are usually only state-of-the-art in one way: they are overspecialized for a single branch of research and are unable to adapt to different modalities without expensive overhauls and program delays. Additionally, partnering with outsourcing organizations may lead to a loss of intellectual property, coordination challenges between the primary organization and the outsourcing partner, and the prioritization of speed over quality.

Going Modular

A more flexible, modular approach is required for biopharmaceutical companies and research organizations to adapt to the evolving lab needs that inevitably manifest over the course of the R&D journey. Rather than rigid, specialized lab facilities, the labs of the future must be changed to meet their users’ needs. This means easily replaceable or changeable amenities, revolutionary infrastructure that allows for quick reconfiguration, and extensive operational support that allows researchers to focus on their core competency: scientific research.

The best solution for biopharmaceutical companies and research institutions, then, is not to build their own specialized, single-purpose labs, nor to collaborate with outsourcing partners, but to gain access to lab space that can be configured to suit their unique needs and process requirements. Thankfully for researchers, established industry experts are stepping up to provide precisely this kind of modular space. By adopting this ‘best of both worlds’ approach, biopharmaceutical companies of all sizes and stages of growth can efficiently access and use lab facilities that meet their needs in significantly less time than traditional approaches.

Customizable on demand, these spaces offer biopharmaceutical companies a way to seamlessly ramp up new research projects in a matter of weeks. Under this model, lab space is also highly scalable, meaning companies can pay for the exact amount of space they need right now, with the option to increase or decrease their usage as needed. This allows companies to preserve cash — an especially important benefit now that the “IPO train” of the last economic boom has left the station. In the current uncertain environment, biopharmaceutical companies are under pressure to balance their financial resources effectively. Reducing upfront capital investments in infrastructure and controlling facility operating costs become imperative to maximize funds for R&D activities, providing a longer runway for these organizations to bring their therapeutic candidates to the clinical stage.

When we talk about medical or life science research, something that often gets lost in the conversation is the researchers themselves. Novel gene therapy researchers are in a race to develop the next groundbreaking therapies, with prestige, funding, and potentially, cures to devastating illnesses, at stake. This “scientific gold rush” puts the need to explore new options beyond traditional laboratory choices in even sharper relief. Scientists need the best facilities possible, which means customizable, adaptable facilities where researchers can pivot to new projects and innovative ideas with agility. These modular facilities enable biopharmaceutical companies to focus on what they do best — developing new therapies — rather than going down the real estate rabbit hole. Considering the numerous benefits of this approach, it is highly likely modular lab spaces will play a crucial role in meeting the ever-growing need for speed in gene therapy research.

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