Revolutionizing early stage drug development with rapid genes
Advancements in synthetic biology are transforming drug development, with rapid gene synthesis emerging as a game changer. Tools like rapid genes, which offer full clonal genes produced and verified through next-generation sequencing (NGS) in as little as five business days, eliminate tedious cloning for the end user and are helping to propel early-stage drug discovery into a new era.
These innovations significantly reduce time spent on gene assembly and verification, addressing one of the largest bottlenecks in pharmaceutical and biotechnological research: the slow and laborious process of producing constructs. The ripple effects of these improvements are felt across key areas of drug discovery, including therapeutic target identification, biomarker discovery and validation, and drug design.
Accelerating drug discovery, development pipeline
The primary benefit of rapid gene synthesis is the reduction in turnaround time for obtaining high-quality, sequence-verified genes. Traditional gene synthesis products can take as much as 10 days to 15 days to ship, and the delay in gene assembly and verification can hinder progress in preclinical development. Rapid gene synthesis, on the other hand, can reduce this timeline to as few as five business days. This faster turnaround time allows researchers to focus more on their core research activities, shortening the overall duration of the drug discovery and early development process.
Gene synthesis with NGS-verification ensures that genetic material is accurate and ready for immediate use, minimizing the risk of experimental errors due to incorrect sequences. High-throughput screening experiments, which typically test multiple gene variations, benefit greatly from this accuracy. By eliminating the need for time-consuming colony screening, researchers can iterate their designs faster, accelerating the pace of innovation.
Shortening the design-build-test cycle
Synthetic biology is playing an increasingly pivotal role in drug discovery and development by enabling the design and engineering of biological systems to create new therapeutics. One of the most critical steps in synthetic biology workflows is the “build” phase of the design-build-test cycle, where generic constructs are synthesized and assembled for testing. Rapid genes could help overhaul this stage by significantly reducing the time it takes to obtain accurate, high-quality genetic material. In the pharmaceutical, biopharma, and biotech industries, where time is of the essence, speeding up the build stage could prove crucial for accelerating research and development.
Traditionally, synthesizing genes and assembling them into functional constructs would typically take longer than a week, even several weeks for highly complex constructs. This delay lengthens experimental cycles and slows scientists’ progress. However, rapid gene synthesis can cut this time down by half or more, greatly accelerating research and the design-build-test cycle by allowing researchers to move quickly from the design phase to the testing phase.
As soon as a gene is synthesized, verified, and delivered to the end user, it can be incorporated into functional studies. This allows researchers to immediately assess its performance and determine its characteristics in experiments, such as those involved in therapeutic target identification, biomarker discovery, and drug design.
Faster therapeutic target identification
Identifying the right therapeutic target is one of the first and most critical steps in drug discovery. Synthetic biology tools, including rapid gene synthesis, are enabling faster hypothesis testing and validation. The ability to quickly synthesize various gene sequences can allow researchers to evaluate a broader range of targets simultaneously.
For example, in cancer research, identifying the specific genetic mutations that drive tumor growth is essential for developing precision therapies. Rapid gene synthesis enables faster modeling of these mutations and more efficient validation of potential targets in a preclinical laboratory setting.
Enhancing biomarker discovery, validation
Biomarkers are integral to early drug development, guiding the discovery of new therapies by providing measurable indicators of disease presence, disease progression, or response to treatment. The fast turnaround time of rapid gene synthesis allows researchers to generate and test multiple genetic constructs, thus expediting the identification of biomarkers. This rapid generation of genetic material, paired with NGS verification, could enhance the value of these biomarkers and help increase researchers’ confidence in their experimental outcomes.
For example, researchers investigating biomarkers for neurodegenerative diseases often need to test gene variations associated with specific mutations. With rapid gene synthesis, these sequences can be generated and verified in days rather than weeks, enabling more efficient validation of biomarkers that could later inform clinical trial design.
Optimizing drug design, protein engineering
Rapid gene synthesis can also play a crucial role in helping to optimize drug design, particularly by allowing researchers to efficiently test and refine multiple versions of potential therapeutic candidates. Drug design is often an iterative process where researchers need to create and evaluate various gene constructs to identify the most effective molecular targets.
With rapid genes, researchers can swiftly generate and obtain different gene sequences, enabling them to explore multiple variations of a therapeutic target or drug’s active site simultaneously. This rapid iteration helps reduce the time spent in each design-build-test cycle, which is crucial for drug design optimization.
This can be particularly evident when optimizing the design of biologic drugs, whether that is through the modification of existing proteins or the creation of new proteins that can function as effective therapeutic agents. Rapid gene synthesis also plays a role in this protein engineering process, enabling researchers to rapidly test multiple variants of a protein. Different gene constructs can be designed to encode for protein variants with specific modifications, such as altered binding sites, improved biochemical stability, or enhanced catalytic (enzymatic) activity.
With rapid genes, these constructs can be synthesized and delivered within a week, allowing for fast testing of their function in experimental model systems. This quick iteration cycle should help accelerate and optimize the discovery and development of therapeutic proteins, such as monoclonal and next-generation antibodies, enzymes, and receptor ligands, all of which play critical roles in modern drug development.
Expediting directed evolution, high-throughput screening
A common technique used in protein engineering is directed evolution, a process where researchers create a library of protein variants and screen them for desired traits. Rapid gene synthesis accelerates this process by enabling the fast generation of large libraries of gene variants, which can then be expressed and tested for their functional properties. The faster the gene variants can be synthesized, the more quickly researchers can identify promising candidates that meet specific criteria, such as improved binding to a drug target, enhanced agonist activity, or better resistance to enzymatic degradation.
High-throughput screening of these protein variants can be made even more efficient when paired with rapid gene synthesis. By generating genes in plate formats, such as 96- or 384-well plates, rapid gene synthesis allows for simultaneous testing of dozens or even hundreds of protein variants using automation. This capability is particularly useful in early-stage drug discovery, where finding the best protein variant can mean the difference between an effective treatment and one with suboptimal therapeutic properties.
Streamlining collaboration and innovation
Another factor that can make or break the success of a therapeutic discovery and development project is team collaboration. In biotechnology and pharmaceutical companies, drug discovery often involves multiple teams working on different aspects of the project, from target validation to preclinical testing.
Rapid gene synthesis could reduce the friction caused by long wait times for critical materials, enabling different teams to work in parallel rather than sequentially. This streamlining of the process can accelerate overall project timelines, leading to faster decision-making and ultimately, reducing the time to market for new therapies. The availability of NGS-verified, high-quality genes may also lower the barriers to innovation.
With gene sequences delivered faster and with greater accuracy, researchers are more likely to pursue exploratory research that they might have otherwise put off due to time constraints. Similarly, a transparent pricing system with no hidden fees allows them to budget accurately, assess, and decide on the feasibility and scope of their experimental projects up front. This freedom to experiment can lead to the discovery of novel therapeutic approaches or potential precision therapies.
Choosing the right vector
An essential aspect of synthetic biology workflows is choosing the right vector, as it directly impacts the efficiency and success of gene expression. Vectors are used to deliver genetic material into host cells, and their selection depends on specific experimental goals and the biological system being used. The choice of vector relies on having access to a wide variety of bacterial and mammalian expression vectors.
Different research projects demand unique vector systems, depending on whether researchers are working with bacterial strains or mammalian cell lines. The availability of diverse vector options enables scientists to quickly choose the best platform for expressing their genes of interest, significantly speeding up research. For instance, bacterial vectors like pET-based systems can allow researchers to efficiently express proteins in bacterial cells, while mammalian vectors are essential for producing functional proteins in human-like systems.
In addition to pre-configured vectors, the ability to purchase genes in an end user’s own custom vectors is a critical capability for researchers working in complex, niche areas of drug discovery. Many biotech laboratories develop proprietary vectors that are optimized for their specific research models. Integrating the synthesized genes into these vectors usually requires significant in-house effort, often involving tedious subcloning and troubleshooting.
However, once a custom vector is onboarded with a vendor, the vendor takes over the cloning, integrating it into their standardized gene synthesis workflow. Researchers can then order fully cloned genes in their vector at any time. This offers tremendous time savings, as teams no longer need to re-clone genes for each experiment. Eliminating the need for in-house cloning allows teams to go directly into functional studies.
The undeniable utility of obtaining genes in proprietary vectors has been recognized as it is now possible to customize these as standard, using new custom vector onboarding tools that facilitate the easy and confidential submission of custom vector sequences online, in a simple process without the need for elaborate communication and direction. Custom vector onboarding helps ensure that researchers can continue using their established systems without disruption, while still benefiting from advances in gene synthesis technologies.
Having access to a wide array of vectors, as well as the ability to onboard custom vectors, allows researchers to tailor the gene delivery system to their specific needs, ensuring more accurate, efficient, and scalable results in both early-stage research and drug development.
Removing endotoxins from vector preparations
For many applications in drug discovery, particularly those involving therapeutic proteins or gene therapies intended for human cells, the presence of endotoxins in DNA preparations can be highly problematic. Endotoxins, which are lipopolysaccharides found in the outer membrane of Gram-negative bacteria, can trigger strong immune responses in mammalian cells. If not properly removed, even small amounts of endotoxins can compromise experimental results by causing unwanted immune activation or cell death through apoptosis.
Endotoxin-free DNA preps are therefore crucial for researchers working on projects where DNA needs to be introduced into sensitive mammalian cells. Removing endotoxins during plasmid purification ensures that experiments can proceed without the risk of introducing inflammatory responses, leading to more reliable outcomes. Moreover, for those developing gene therapies or biologics for clinical applications, endotoxin-free vectors are a non-negotiable requirement, as they help ensure the safety and efficacy of the final product. This capability further underscores the importance of high-quality synthetic biology tools in advancing cutting-edge medical research.
The introduction of fast gene synthesis and its ability to accelerate drug discovery marks an important moment in biopharmaceutical research. By drastically reducing the time needed for gene synthesis and verification, these tools enable faster progress in key areas such as therapeutic target identification and biomarker discovery.
Further, their potential role in facilitating teams to drive innovation quickly opens the door to more personalized and precision therapies that require a rapid pace of development. The impact of rapid gene synthesis on pharma will only become more pronounced, promising faster drug development and more effective treatments for patients worldwide.
