Breakthroughs in Pharmaceutical Technology: Advancements Shaping the Pharmaceutical Sector in 2024

It's not a secret: a healthy pharmaceutical sector equates to positive outcomes for patients and investors. There have been significant technological breakthroughs in the pharmaceutical industry in the past few years, especially in vaccine development and cancer treatment. Furthermore, the advances in GLP-1 drugs have revolutionized obesity management, gene editing, and gene therapy for rare disease treatment.

This rapid growth in the pharmaceutical sector can be attributed to three major external factors: the coronavirus (COVID-19) pandemic, increased investments, and the development of new modalities. While these three factors may seem independent, they are complexly interlinked. The COVID-19 pandemic was a significant, meaningful, and unexpected test for the pharmaceutical industry and humanity. Researchers, scientists, and pharmaceutical companies formed unexpected and beneficial collaborations to develop vaccines in the face of an unknown virus. And in the end, leaders, people, and investors understood the importance of the pharmaceutical sector in society, leading to increased investments. These investments and funds fueled the development of new modalities, therapeutics, and treatment methods that will change the sector's future.

Following in the wake of these developments, 2024 marks a pivotal year for the pharmaceutical sector. The pharmaceutical industry is at an inflexion point, poised for growth and innovation. However, this progress will largely depend on emerging technologies and whether pharmaceutical companies can embrace them with open arms. We've identified several pharma technologies that are expected to change our approach to medicine in the future.

Nanomedicine in advanced drug delivery

Drug delivery systems have evolved from syringes, nasal sprays, and eyedroppers to nanomaterials. Nanomaterials can be of various shapes but have a size between 1 and 100 nm. The past decade has seen an increase in research focused on applying nanomaterials in medicine, leading to the establishment of a new field: nanomedicine. Nanomedicine can revolutionise drug delivery in various ways:

• Nanomedicine comprises a carrier and an active pharmaceutical ingredient (API). The API can be synthesised at the nanoscale (i.e., as nanocrystals), which can then be delivered directly to the patient. This improves the solubility of the API in bodily fluids compared with the API ingested directly via pills, tablets, etc. This increased solubility implies a higher efficiency per dosage, which means formulations with smaller dosages or lower quantities of API will be more effective in treating diseases.
• Nanomaterials can also be used as carriers. For example, nanocapsules, nanospheres, polymeric nanoparticles, etc., can be used as drug delivery systems. These materials are usually developed with specific functionalities that trigger when exposed to particular bodily conditions. This is the opposite of generic drug delivery systems such as tablets and capsules, which dissolve when contacting saliva. Consequently, this allows the targeted delivery of the API. This improves the effect per dose.
• Nanomaterials can also be used to increase the half-life of the drug. The half-life of a drug is the time it takes for the human body to eliminate half of the ingested drug amount. Simply put, small-molecule drugs have short half-lives, and large-molecule drugs have long half-lives. However, using the targeted delivery mechanism of nanomaterials, the drug can be protected from the bodily mechanisms that break down and eliminate the drug. This increases the half-life of the drug.

While these are only a few examples, they excellently display how nanomaterials can transform drug delivery in the future. Nano-drug delivery systems can treat generic diseases like viral infections and diseases such as cancers and tumours.

Next-generation proteomics

Next-generation proteomics is the advanced study of the proteome, a set of proteins produced or modified by organisms. Next-generation proteomics enables comprehensive and high-throughput analysis of proteins, including their structures, interactions, and functions. It can be used to map complex networks within diseases, which will help researchers better understand disease mechanisms inside and outside the body of organisms at the molecular level. This analysis will supplement the discovery of biomarkers for disease diagnosis, prognosis, and treatment, which are crucial for understanding diseases. These biomarkers are used in drug development and can also be used to identify new therapeutic targets. Next-generation proteomics can revolutionise the pharmaceutical space in various ways:

• Next-generation proteomics can identify protein signatures associated with different types of cancers, neurodegenerative diseases, cardiovascular diseases, etc. For example, the Clinical Proteomic Tumor Analysis Consortium has reported proteomic characterisation data for various types of cancer (e.g., endometrial cancer, renal cancer, breast cancer, lung adenocarcinoma, and brain cancer), which has led to the discovery of novel therapeutic targets and biomarkers.
• Next-generation proteomics can also streamline the identification of new drug targets, reducing the time and cost associated with drug development. Proteomic technologies can also sift through large databases to identify protein functions that are the key to different pathways. This speeds up the initial stages of drug discovery.
• Proteomics can provide insights into protein functions, mechanisms, pathways, and interactions, which are vital in drug discovery and development. The mechanistic insights also allow the identification of points within these pathways that can be disrupted to achieve treatment. This mechanistic insight can also be used to understand correlations among diseases.

In addition to these examples, next-generation proteomics is also hypothesised to be used to monitor disease progression and treatment response in real-time.

Genetic editing

Gene editing is a technology that allows researchers to alter the DNA of a specific organism. It involves the editing, removing, or modifying genetic material at particular locations in the genome. The focus on gene editing has exploded since Jennifer Doudna and Emmanuelle Carpenter received the Nobel Prize in Chemistry in 2020 for developing CRISPR-Cas9 gene-editing technology. This achievement has opened the door to various therapies and tools. Gene editing is considered a significant breakthrough pharmaceutical technology for several reasons.

• Gene editing holds the potential to provide permanent solutions to genetic disorders. Gene editing can correct mutations that lead to genetic disorders at the source. This allows the treatment of diseases caused by a single genetic mutation, such as sickle cell anaemia and muscle dystrophy, which can be treated by correcting the defective gene. This is extremely promising because current conventional treatments can only manage symptoms rather than treat disorders.
• Gene editing can also promote the development of personalised medicine. By understanding the genetic makeup of every individual, gene editing can be used to develop personalised therapies tailored to the patient's genetic profile. This will likely improve the efficacy of medication and reduce the side effects associated with treatments. 
• Gene editing can also modify immune cells to recognise and attack cancer cells better, increasing the effectiveness of immunotherapies.
• Gene editing can be used to engineer plants and microorganisms that are used to produce pharmaceutical compounds. By improving their functionalities, growth rate, or other characteristics, the associated costs can be reduced, and their accessibility can be increased.

In addition to CRISPR-Cas9, various other therapies have been developed, such as the CAR-T cell therapy, Kymriah, by Novartis. Different companies like Editas Medicine have also developed other CRISPR-Cas9-based therapies.

3D bioprinting and tissue engineering

With the development of these new therapeutics, it is understandably necessary to test them before approving them for clinical trials. To this end, three-dimensional (3D) bio-printing has been the core focus. Bio-printing and tissue engineering are used to create biological tissues and structures. These technologies have a lot of potential to revolutionise the pharmaceutical space:

• 3D bio-printing has promising applications in organ transplants. The technology can address the storage of donor organs by bio-printing organs that are tailored to the recipient.
• Tissue engineering can be used to create tissues to repair damaged organs. For example, bio-printed skin grafts can facilitate joint repair for burn victims. These skin grafts are customised to match the patient's cells, which reduces the risk of rejection.
• Bio-printed tissues can also be used as models of human biology. They are much more accurate than cell cultures and animal models, which are very commonly used. The bio-printed tissues can replicate the complexity and functionality of human tissues, making them a more accurate platform for drug testing.
• Bio-printed tissues can also be developed using the patient's cells. These tissues can then be used to test the patient's response to a new therapy. This allows the development of personalised treatment plans tailored to the patient's genetic makeup and needs.

3D bio-printing and tissue engineering have already found applications in the pharmaceutical sector. Organovo and Tengion are biotechnology companies that specialise in 3D bio-printing human tissues.

Artificial Intelligence

Any technological breakthrough list is incomplete without artificial intelligence (AI) and for a good reason. As we've already covered in previous issues, AI can make waves in all phases of drug development, from drug formulation to clinical trials and marketing insights. We've published comprehensive articles highlighting the role of AI in the pharmaceutical industry, but here's a short recap:

• AI can be used to identify promising drug targets and design new drug formulations.
• AI can be used to design clinical trials, identify suitable volunteers, and analyse their data. AI can be used to simulate clinical trials to understand potential factors that may fail.
• AI can automate regulatory processes such as applications, data analysis, and computation.
• AI can be used to understand regulatory guidelines better, identify potential non-compliance issues, and develop strategies to fix them.
• Machine learning models can predict potential issues during drug development stages (e.g., clinical trials, regulatory approval, marketing, post-sale support, etc.).
• Real-world data can also be fed into AI to obtain new insights, helping researchers track the efficacy and side effects of the formulation even after editing clinical trials.

In the end, this is but a short list. AI has already revolutionised the pharmaceutical space in the past year, and we can expect many more applications in the future.

Quantum computing

Molecular mechanics is the traditional approach to modelling compounds in synthetic and medicinal chemistry. However, molecular mechanics faces several limitations, which limit its applications in the pharmaceutical sector. To this end, quantum mechanical methods, specifically quantum theory and quantum computing, have exhibited substantial advantages, not least the accuracy of their predictions. Today, several companies use quantum computing methods such as density functional theory to accelerate drug discovery.

• Quantum computing leverages the principles of quantum mechanics to perform infeasible calculations for conventional computers. Quantum computing can revolutionise the drug discovery process by accelerating simulations, optimising molecular structures, and solving complex biological problems.
• Quantum computers can simulate the quantum mechanical behaviour of molecules more accurately than conventional computers. This is particularly useful for understanding complex molecular interactions and chemical reactions. This allows the analysis of their responses in complex systems, which accelerates understanding of the side effects of drugs.
• Quantum computing can handle calculations on the complexity of large biomolecules, such as proteins and DNA, and insights into their structure and function, which is difficult to achieve with classical simulations. This increased accuracy allows better simulations of large-molecule compounds.
• Quantum computers can enhance molecular docking processes by optimising the fit between the drug and its biological targets. This can lead to the discovery of more effective medications with higher binding affinities. The computers can also optimise the energy states of molecules, helping researchers identify the most stable conformations of drugs.

Quantum computing holds enormous unrealised potential in the field of drug discovery. While several companies use quantum theory to accelerate drug development and therapeutics, this space still has much exploration potential.

In the End

According to various experts, the pharmaceutical industry has hesitated to digitise, primarily due to the complex processes and stringent regulatory requirements. Nevertheless, the COVID-19 pandemic has shown the accurate picture: in various scenarios, pharmaceutical companies fall short because they rely on manual labour, inefficient processes, and poor digitisation. Thankfully, different pharmaceutical companies and experts have identified these gaps and are developing technological solutions to overcome the shortcomings. Therefore, as we stand at the brink of new technological development, companies that embrace digital transformation and invest in new therapies will be well-positioned to capture a larger market share.