As we step into 2024, the pharma and biotech industry experiences ongoing evolution. In this blog, Dr. Caroline Beltran (PhD) forecasts three of the significant developments, technologies, and insights poised to influence the sector throughout the upcoming year.
1. The continuous evolution of CRISPR
The discovery of CRISPR technology has revolutionized the landscape of molecular biology allowing scientists to edit genes with unprecedented precision and efficiency. As researchers continue to refine and expand the CRISPR toolbox, the potential applications for shaping the future of medicine are expanding at an incredible pace. Poised to revolutionize how we treat diseases including correcting genetic abnormalities, treating hereditary diseases, cancers, CAR-T cell engineering, autoimmune conditions and infectious diseases, this research field is extremely exciting and one to watch. Notably, in the realm of cellular gene therapy, there have been significant developments with the UK recently granting market approval for the world’s first CRISPR/Cas9 gene editing therapy. Additionally, substantial breakthroughs have occurred in identifying new CRISPR systems, with the unveiling of the Fast Locality-Sensitive Hashing-based clustering (FLSHclust) algorithm. This innovative tool identified 188 new rare CRISPR systems in bacterial genomes, encompassing thousands of individual systems. Despite its immense potential, gene editing technologies face challenges including off-target effects and ethical concerns. Ongoing research aims to refine the technology and address these issues, ensuring its safe and effective use in the future.
2. The 3D revolution
The imperative for patient relevance in drug testing has propelled the exploration of novel models beyond traditional animal systems, necessitating preclinical models that can better capture the intricacies of human physiology and enhance the translatability of experimental findings to clinical outcomes. Notably, the passing of the FDA Modernization Act 2.0 paved the way for a significant shift toward alternative technologies and the dawn of the organoid era.
The recreation of physiologically relevant cellular models that can capture complex in vivo architecture has become reality with the development of cutting-edge technologies in 3D bioprinting, tissue engineering and organoid/organ-on-chip techniques. This new era in bioengineering holds immense promise to create tissue/organ models mimicking the human body with high reproducibility and repeatability that expand not only in vitro studies, but open avenue for regenerative medicine. In particular, the incorporation of perfusable microvascular networks into 3D systems have increased long term viability of dense cell structures, as well as allowing a vascular-mediated drug infused model for drug screening. Advances in stretchable and viscoelastic biomaterials that can modulate physiological processes have significant implications, specifically for cardiac and neural tissue models, and further improve their functionality. There remains several hurdles and challenges to overcome in this exciting field, but the tremendous potential of these tools is sure to be centre stage in 2024.
3. The new pillar of biotechnology: Artificial Intelligence
We’d be hard-pressed to talk about trends in 2024 without a mention of the robot in the room – Artificial Intelligence. Virtually every pharmaceutical company is now using AI to find new biomarkers, new targets and design novel compounds. There has been a surge to put more data to work. The explosion of big data, from multi-omics strategies to modelling diseases in 3D all stand to culminate at the AI mountain – better data drives faster therapeutic development. Across the board, AI is being embraced as a means to enhance operations and research capabilities not previously possible, making it easier to navigate and incorporate swaths of data to identify new patterns and novel avenues.
Of note, the FDA recently granted the first Orphan Drug Designation for a drug discovered and designed using AI – a small molecule inhibitor treatment for idiopathic pulmonary fibrosis (IPF). In clinical trials, Pfizer has been using AI and machine learning algorithms to identify the most suitable patient population for their clinical trials and monitor the patient’s health during the trial. Medical devices are even benefiting from AI with Johnson & Johnson Center for Device Innovation designing contact lenses which are personally optimized by AI for the individual wearer. Deep Genomics and Foundation, two companies using AI to incorporate genomic data, biomarkers, and other physiological data to tailor treatment plans to individual patients by predicting how they will respond to different treatment options, highlight the use of AI for precision medicine. The application for AI in the life sciences is virtually limitless, with each use case being more exciting than the next.
But as AI and machine learning take centre stage and become more common place, new challenges to instil confidence in validating these systems have emerged, as well as concerns for ethics, measures to avoid bias, discrimination, error, or misuse, data protection and cybersecurity. Guidelines around using AI in the life sciences industry are still elementary. FDA has begun to get involved and plans to gather public commentary on the use of AI in biopharma manufacturing, and Congress is in the very early stages of AI-focused legislation.
2024 holds a great deal of promise in the biotech and biopharma industries. At Synexa, we are excited to play our role in improving human health, applying our wide range of capabilities and services to support all our clients on their respective journeys. The only constant in this industry is change and we are proud to continue innovating and working with our clients to best support research that is not only thought-provoking, dynamic and interesting, but also impactful.
About Synexa and Scientific Strategies
Synexa is a specialized biomarker and bioanalytical services firm, established over 20 years ago. We have been supporting our clients at all stages of clinical development, from discovery to phase 3 studies. Our services cover analysis across the biological continuum, from nucleic acid, soluble biomarker, cell and tissue. Our team of dedicated scientists, working across labs in South Africa, Europe, the UK and US, relish solving intricate and challenging biological problems.
A key extension of this; is Synexa’s Scientific Strategies Team. There are many challenges and risks progressing a compound into clinical development. Synexa’s Scientific Strategies team can help de-risk your development by providing support around key biomarker and bioanalytical considerations. The dedicated team can offer standalone consulting support, providing objective, actionable solutions to your biomarker and bioanalytical challenges.
