It's been more than a decade since Emily Whitehead was declared cancer-free after the first successful experimental CAR T-cell therapy, a moment that marked a huge advance in precision medicine. Since then, the scene has focused on CAR-T therapies, six of which have now been approved. One of the newest movements in this field is CRISPR therapeutics, which further advances precision medicine's ability to provide effective long-term treatments for rare and complex diseases.
According to GlobalData's Center for Pharmaceutical Intelligence, cell therapies remain the leading drug type in terms of number of clinical trials. However, there has been a marked increase in gene therapies in the past few years.
GlobalData is the parent company of Clinical trials arena.
Only 39 gene therapies had been marketed by the end of the first half of 2023. Despite this, the market is expected to grow significantly over the next decade, with GlobalData forecasting that gene therapies will generate approximately $54 billion in sales by 2029. The cell therapy market is also expected to rise, with forecasts at more than $52 billion by 2029, up from about $3 billion in 2022, according to GlobalData.
Precision medicine trials have always focused on oncology due to the mechanism of action (MoA) of most candidates, which is to target and kill T cells. This field still favors oncology, however, other indications, especially rare diseases, are now being studied to see how they can benefit from precision medicine.
Access the most comprehensive company profiles on the market, powered by GlobalData. Save hours of searching. Gain a competitive advantage.
Company profile – free sample
Your download email will arrive shortly
We are confident in the unique quality of our company profiles. However, we want you to make the decision that is most beneficial to your business, so we are offering a free sample that you can download by submitting the form below
By GlobalData
The knowledge scientists have gained about precision medicine is already being used in autoimmune diseases, says Dr. Dalip Sethi, commercial leader for cell therapy technologies in North America. “Autoimmune diseases need a lot of attention, and there are a lot of companies working on them, which is exciting to see,” Sethi says. “The regulatory mechanisms of T cells – exploring them and suppressing the autoimmune system, is very exciting to see because that is a huge need in the market.”
Technology can improve accrual rates in trials
One of the biggest pain points in any clinical trial is termination of the trial. According to GlobalData's Center for Pharmaceutical Intelligence, most precision medicine trials have been terminated due to low accrual rates.
While Peter Keeling, CEO of Diaceutics, says one of the main reasons for the decline in accrual is that a large number of precision medicine trials target rare diseases. As a result, some drugs that may show strong signals do not reach the market. “There's a discussion going on in biotech companies about how to move forward, because they know they have a signal in the drug,” Keeling says.
AI is becoming a big player in reducing dropout rates by more accurately connecting patients to the best trials, says Bikash Chatterjee, CEO of Pharmatech Associates. “Clinical operations also rely on AI to identify eligible candidates for clinical studies to facilitate patient engagement and reduce dropout rates,” says Chatterjee.
Better use of biomarkers will advance this field
One of the biggest advances in precision medicine is the use of biomarkers. Biomarker diagnostic tools have evolved significantly in recent years, with oncology remaining a major area of biomarker-based therapy. Thus, biomarkers will be used to target subpopulations across cancer types, for example, identifying specific mutations in cancer cells.
The development of new diagnostics and the discovery of new biomarkers will advance the field of precision medicine as clinicians will be in a better position to identify targeted therapies for more indications.
Further research into biomarkers and more advanced diagnostic tools will be needed to drive the development of next-generation precision medicine. Sethi believes this could be a way to use AI in precision medicine to identify both clinical trial populations and patient populations after approval.
One development is the invention of digital biomarkers, moving away from the model of traditional blood biomarkers. NeuraLight, a startup developing an AI-based platform to measure eye movements to diagnose neurological diseases.
The computer-based oculometry test tracks eye movements using a standard webcam and analyzes them using machine learning techniques, says chief commercial officer Vivian Dioskin. It is hoped that the technology will be used to test the success of diagnosis, prediction and treatment.
“We've seen oncology really blossom into precision medicine, and neuroscience is poised for the same kind of innovation and improvement in patient outcomes,” says Dioskin. “We hope that technology similar to ours can increase the clinical trials being conducted in neuroscience and usher in an era of more precise and more effective disease-modifying therapies.”
Biomarkers will be used more effectively due to standardization in the industry, notes Matthew Licklin, vice president of scientific affairs and product development at TrakCel. “It's standardized across the industry,” Licklin explains. “This standardization is really helpful because it's designed around legislation as well. Whether you're offering precision medicine in North America, Europe or Asia, it's the same.”
The use of biomarkers will push precision medicine even further, allowing the use of combination precision therapies, says Josh Ludwig, director of ScaleReady. “I see the field moving toward more combination therapies and even combination technology,” Ludwig says. “Analyzing someone’s blood with some of these new tools and identifying biomarkers and all the different targets for that specific patient and giving combination therapies. And in these applications, we can apply artificial intelligence and machine learning.”
AI will be used at all stages, from research and development to market
With AI being used in all areas of life, it is likely to have a significant impact on precision medicine at all stages of drug development. According to GlobalData's drug database, there are more than 40 cell and gene therapies developed using AI.
Chatterjee says AI will boost research into cell and gene therapies. “AI has the potential to impact both in countless ways,” says Chatterjee. “There is huge potential for gene therapy because the ability of AI to predict protein structures has been shown to enhance DNA scissor technology like CRISPR by making more cuts in a patient's DNA more precisely.”
“In fact, identifying receptor targets and drug targets would be fascinating using AI,” Licklin adds. “This will likely remove a lot of necessary preclinical work and screening by being able to identify lead drug candidates sooner.”
Technology will help advance a bright future
As technology continues to evolve and with advances in artificial intelligence, there is hope that more progress will be made in this field over the next ten years, but specialists have many different theories about what the big moment of the next decade will be.
Going back and reflecting on past learning will be the way forward, says Ludwig, noting that the world needs a common way to develop cell therapies to ensure they are available to people all over the world and not just in specialist centres.
“We really have to standardize the way we make these cells,” explains Ludwig. “And we need to do it in a way that focuses on the simplest, most simple version of culturing these cells. We want cell therapies to be available in less developed areas of the world, and to do that, we have to make them really easy to manufacture.”
However, Chatterjee believes that a myriad of technologies will enhance this field. “It is not unreasonable to expect AI to become part of the overall toolkit for drug development including precision medicine,” Chatterjee explains. “However, in the near future, AI is unlikely to be the only tool in the industry’s toolkit fueling the drug development pipeline.”
Sethi believes that all medicines could have a precision medicine component in the future, but accepts that it will take a lot of work. “When I go to the doctor, I want a drug that targets my biology,” Sethi says. “Everyone’s code is different, and precision medicine looks at that code but it needs a lot of data.”
However, Licklin agrees that data is the key to moving forward with precision medicine. “For precision medicine to succeed, we need larger and larger data sets,” Licklin concludes. “We need the ability to collect patient information in an anonymized way so that we can see trends that will help us understand the right medication and the right dose for the right patient.”
Cell and gene therapy coverage in the clinical trials arena is supported by Cytiva.
Editorial content is independently produced and affiliated with Highest standards Of journalistic integrity. Topic sponsors are not involved in creating editorial content.
