As 2023 comes to a close, experts at Revvity, a life sciences and diagnostics business, are looking toward the new year. In this roundtable article, six experts share their predictions for 2024 in a variety of critical industries.
Michelle Fraser, Head of Cell & Gene Therapy
Cell and Gene Therapy: Approvals Surge
In the rapidly evolving landscape of cell and gene therapies, the momentum is palpable. According to ASGCT, the pipeline boasts more than 3,700 therapies in development, setting the stage for a transformative period. Forecasts from TuftsMedicine further underscore this anticipation, highlighting the likelihood of at least five FDA approvals by the close of 2023, with a notable emphasis on the promising realm of CAR-T/TCR therapies for blood cancers. The statistics reveal a compelling narrative – CAR-T/TCR therapies exhibit a more than twofold likelihood of approval upon entering Phase I compared to their hematological oncology counterparts. Similarly, orphan gene therapies demonstrate double the chances of approval post-Phase I trials, outpacing average drugs in analogous therapeutic areas.
A noteworthy acceleration is evident, with over 500 new cell and gene therapies entering clinical trials annually. Their expedited time to market, averaging five years as opposed to the 10+ years for traditional small molecule drugs, positions this class of therapeutics for significant prominence in the pharmaceutical landscape. As we approach 2024, the prospect of witnessing a surge in approvals becomes increasingly imminent. Concurrently, the FDA's proactive initiatives to bolster the advancement of these groundbreaking therapies promise to usher in a new era of standardization. Expectations include the streamlining of manufacturing processes, consolidation of protocols for testing Critical Quality Attributes, refined approaches to patient identification and enrolment, and the evolution of companion diagnostics. This concerted effort towards standardization is poised to shape a more efficient and robust framework for the evaluation and deployment of novel therapies, marking a pivotal step towards their widespread adoption in the coming years.
Gene Editing & CRISPR: A New Era of Precision and Complexity
In the dynamic realm of gene editing and CRISPR technologies, the pace of advancement is nothing short of remarkable. Developers in the cell and gene therapy arena are fervently working to create improved therapies characterized by greater efficacy, improved safety profiles, and streamlined manufacturing processes. At the heart of this pursuit lie innovations in payload and delivery mechanisms. Within the gene editing landscape, developers are narrowing in on novel nucleases with specificity to, and optimal activity, at the targeted site. This endeavor seeks to navigate regions that may elude the original Cas9 nuclease. Simultaneously, developers are honing deaminases with high editing efficiency, coupled with a meticulous avoidance of bystander or off-target editing. The quest for precision extends to the realm of guide RNA design algorithms, ensuring the accurate navigation of nucleases to their intended target sites.
The evolving toolkit is empowering therapeutic developers to engineer improved therapies. This includes the capability to knockout multiple genes, suppress or upregulate specific genes, or insert new copies of genes. Notably, there are also technologies poised to edit mRNA rather than the genome, inducing transient changes, and addressing genetic alterations influenced by environmental factors through epigenetic modifications. As our comprehension deepens regarding the intricate interplay between the genome, proteins, and cellular functions, coupled with the expanding arsenal of genome and transcriptome manipulation tools, the landscape of cell and gene therapies is witnessing a paradigm shift. The trajectory points towards increased sophistication, efficacy, and safety in these therapeutic interventions.
Anticipating the trajectory in 2024, the horizon is set for substantial strides in gene modulation technologies. Expect to see a surge in the adoption of more intricate gene editing approaches, signaling a year marked by significant advancements in the refinement and application of these transformative technologies.
Craig Monell, Senior Vice President, Business Operations, BioLegend (Part of Revvity)
Cellular Multiomics: Shaping Life Sciences and Diagnostics
The coming year promises to be a transformative one for the life sciences, with cellular multiomics taking a leading role in helping to advance our understanding of cellular composition and function, and potentially revolutionizing clinical diagnostics. In the rapidly evolving field of cellular multiomics, we can anticipate significant breakthroughs that will shape the future of life sciences. Cellular profiling assays are transitioning from traditional reliance on a limited set of markers within a singular molecular class to newer high-plex, high-cell number multiomics assays. These assays target a combination of RNA, proteins, DNA, and various other marker types, and they are deepening our understanding of cellular composition and function. We can expect this very active applications development area to deliver significant advances in assays including expanded marker numbers and diversity; increased numbers of cells interrogation; streamlined workflows; enhanced resolution in spatial analysis; and reductions in the cost per cell or marker analyzed. These improvements will make multiomics technologies more accessible and drive their adoption. Additionally, AI/ML-driven informatics will simplify the interpretation of complex multiomics datasets, making it easier to extract meaningful insights from vast amounts of data. While researchers in the highly competitive life sciences arena are rapidly making progress, we can also expect to see CROs and life science tool providers working diligently to standardize specific assays. This standardization is a crucial step toward incorporating these technologies into clinical trials and eventually deploying multiomic signature-based diagnostics and companion diagnostics.
Madhuri Hegde, FACMG, SVP and Chief Scientific Officer
Next Generation Sequencing for Newborn Screening
Next generation sequencing (NGS) is providing efficient, holistic insights into the human genome, which has become a viable option for population-wide screening, including within newborn screening (NBS). A recent study conducted by Revvity Omics has shown the clinical value of proactive, sequencing-based screening in apparently healthy newborns. The use of proactive genomic screening would enable healthcare professionals to uncover a wide range of risks for looming pediatric onset conditions, allowing for earlier interventions and personalized treatment plans based on individual genetic make-up. As NGS becomes more accessible to the general public, harnessing this tool could have a profound impact on the health and wellbeing of families and future generations.
Yves Dubaquie, Senior Vice President, Diagnostics
Life Sciences and Diagnostics: The Convergence Becomes Real
The era of a one-size-fits-all approach to healthcare is over as personalized medicine makes it possible to match specific patient groups with tailor-made treatments that can improve clinical outcomes, and in some cases, save lives. However, siloes and technological gaps stand in the way of personalized medicine reaching its fullest potential. As science becomes both more complex and more personalized, we will see even greater collaboration between life sciences and diagnostics. This convergence is key to helping drive new scientific breakthroughs in laboratories, bringing a new product through the clinic, and ultimately tailoring a unique diagnostic which can help identify individuals who stand to benefit the most from this new therapeutic or cure. This full “continuum” involving research to development, diagnosis to cure, will improve healthcare for everyone, everywhere.
Stephanie Franco, Senior Scientific Affairs Liaison, EUROIMMUN (Part of Revvity)
Blood-based Biomarkers and Genetic Screening for Patients with Early-stage Alzheimer’s Disease
With the excitement surrounding the FDA approval of Leqembi and pending approval of donanemab (anti-amyloid treatments for Alzheimer’s disease (AD)), we see further clinical investigations into blood-based biomarkers (BBBMs) and genetic screening as imperative to furthering improvements in AD diagnosis and treatments. It has been demonstrated that ApoE e4 carriers are at a higher risk for developing amyloid related imaging abnormalities (ARIA) when undergoing anti-amyloid treatments, such as Leqembi and donanemab. Thus, it has been recommended in the Leqembi instructions for use to perform ApoE genetic screening prior to treatment, which suggests this genetic screen could become a requirement for treatment. Furthermore, several new and existing biomarkers in plasma (e.g p-tau-217) are being investigated as having value in accurate diagnosis and treatments which are more economically friendly, easily accessible and less invasive than CSF draws and PET screens. We also see further investigations into combination therapies with targets other than amyloid to help reduce cognitive decline in patients with mild to moderate AD. Further exploration of BBBMs and ApoE genetic testing are surely to be pivotal in the next chapters of AD diagnosis and treatments. The changing landscape of Alzheimer’s disease research and clinical trial data presented at the most recent Clinical Trials for Alzheimer’s Disease (CTAD) conference was described as profound and we agree.
Ruth Brignall, Global Scientific Affairs Manager
Global Healthcare Landscape: The Eradication of Tuberculosis
Despite being a preventable and curable disease, TB continues to claim the lives of millions each year, largely impacting low and middle-income nations.
Access to diagnostics and treatment is crucial for eliminating TB. Early and accurate diagnosis enables timely treatment initiation and prevents further transmission. Affordable and effective treatment ensures patients complete therapy, reducing drug-resistant strains. This comprehensive approach is vital in the fight against TB.
Current diagnostic tests for active TB rely on microbiological confirmation of TB bacteria from sputum samples, or visual signs of TB in the lung observed on a chest X-ray. As a result, around 36% of new TB cases, who do not have pulmonary TB or cannot produce a sputum sample, go undiagnosed or unreported. The main challenge lies in the absence of non-sputum-based diagnostics. Developing accurate non-sputum-based tests is crucial to diagnose individuals suffering with active TB who cannot produce sputum.
In 2024, we expect the development of innovative TB diagnostics, like non-sputum-based diagnostics, to begin addressing unmet needs within the TB space. We also anticipate that we will see improved access to existing tests for better coverage where needed the most.