.png)
Treating autoimmune and inflammatory diseases has long relied on broad-spectrum drugs that suppress the immune systems. While these medications can be effective, they often act as blunt tools, leading to serious side effects such as increased risk of infections, long-term organ damage, and reduced quality of life. In this post, we explore how new therapies, from biologics and small molecule inhibitors to emerging CAR-T cell treatments, could be the future of treatment. We also examine the gaps still holding precision medicine back.
Existing approaches
There are currently a variety of different treatments available for mitigating and treating autoimmune and inflammatory diseases. These include:
Conventional immunosuppressants
Corticosteroids and disease-modifying antirheumatic drugs (DMARDs) are commonly used in combination to treat autoimmune and inflammatory diseases. Corticosteroids are often used as an additional, temporary therapy to rapidly bring down inflammation while waiting for the effect of slower acting DMARDs to kick in and limit the progression of joint damage in conditions such as RA. While these two types of immunosuppressants are some of the most commonly used treatment types, they often result in broad immune suppression and adverse side effects for patients.
Non-steroidal anti-inflammatory drugs (NSAIDs)
NSAIDs can also be used for short-term symptom management to help treat the pain and inflammation caused by autoimmune and inflammatory diseases. These treatments include ibuprofen, naproxen, and meloxicam. However, they do not address the cause or biological mechanisms behind the conditions and are only effective in terms of offering short-term pain relief.
Biologics
Biologic treatments consist of complex molecules produced from or containing components of living organisms. As discussed, they enable a more targeted approach than conventional immunosuppressive treatments, acting on specific components of the body’s immune system and limiting side effects. Administered via injection or intravenous infusion, these types of medications (including TNFs) have shown significant efficacy in slowing or stopping inflammation that can damage joints and organs in conditions such as arthritis and other inflammatory diseases.
However, despite offering improved patient outcomes and reduced side effects, these treatments aren’t without their risks. While more targeted, they still come with an increased risk of infection and the chance of adverse effects such as the development of other autoimmune conditions.
Emerging treatments
There are also several emerging therapy and precision medicine approaches which hold significant promise for the effective treatment of autoimmune and inflammatory disease. These are made all the more important by the growing numbers of people affected around the world.
Small molecule inhibitors
Targeted synthetic DMARDs (tsDMARDs), also known as small molecule inhibitors, are showing promise as a more targeted approach to treating autoimmune rheumatic disease. These treatments have enabled higher rates of remission, a reduction in disease progression, and significant improvements to patients’ quality of life.
One category of tsDMARDs, Janus kinase (JAK) inhibitors, inhibit one or more JAK enzymes and help slow down the overactivity of the immune system that can lead to inflammation and pain. As of 2025, these kinds of inhibitors are the only tsDMARDs currently available for treating inflammatory arthritis.
However, while these kinds of small molecule treatments offer improved patient outcomes, they also come with an increased risk of infections including pneumonia, urinary tract infections, and cellulitis. Patients who are given these therapies require regular blood test monitoring, close follow-up, and access to prompt rheumatology advice.
Cell therapies
CAR-T cell therapies, initially developed for cancer treatment, are now showing promise for treating B cell (white blood cell)-associated autoimmune diseases. This type of therapy involves genetically engineering a patient's T cells to express chimeric antigen receptors (CARs) which then target specific antigens associated with disease-causing cells.
Unlike some other treatment types, by directly addressing the underlying drivers of autoimmunity rather than simply managing symptoms, CAR-T therapy offers the potential for long term remission from autoimmune and inflammatory diseases. Engineered CAR-T cells also offer a highly targeted approach, enabling preservation of overall immune function and reducing the risk of infections and other side effects associated with broad immunosuppression treatments.
Early stage clinical trials for anti-CD19 and anti-BCMA CAR-T cells have shown promising results in using engineered cells to treat B cell-associated autoimmune diseases such as lupus and myasthenia gravis.
However, despite these advancements and the huge potential of these treatments, there are a number of significant safety concerns including life threatening reactions, excessive immunosuppression and the risk of secondary malignancy. Additionally, the high cost of these treatments is a prohibitive factor, with a single infusion costing between $375,000-$425,000 in the United States as of 2023.
Current gaps and blockers
Despite the potential of precision medicine and genetic engineering approaches, there are significant challenges which need to be addressed. The interoperability and safe sharing of data is vital to the future success and integration of precision approaches to autoimmune and inflammatory disease and overcoming the heterogeneity of these conditions.
Insufficient patient stratification
One of the major challenges in precision medicine for autoimmune and inflammatory diseases is identifying and developing patient stratification frameworks for categorizing these highly heterogeneous conditions. Subcategorizing patients into well-characterized groups is key to matching targeted and more effective treatment to the right individuals and identifying those who can most benefit from genetically-tailored treatments. Currently, a lack of sufficient data to validate such subgroups and cohorts is proving a challenge to developing robust stratification frameworks in these disease areas.
However, recent studies have demonstrated the potential of molecular stratification, such as grouping patients based on their transcriptomic profiles, to help identify standardized disease clusters defined by specific molecular patterns. While recent research has made progress in developing such frameworks, these approaches remain underutilized due to limited integration into clinical practice and insufficient validation in large cohorts. Collaborative, international efforts will be required to standardize stratification methods in these disease areas, as well as validate biomarkers across diverse patient populations.
Regulatory and market access challenges
With the emergence of pioneering new treatment types, such as engineered regulatory T cells, in vivo CAR-T cells and the use of CRISPR, the development of regulatory frameworks which take into account new safety, efficacy, and manufacturing standards will be crucial to delivering precision medicine treatments to market. The complexities of developing such frameworks in the rapidly changing precision medicine landscape are further compounded in the context of autoimmune and inflammatory disease, due to the diversity and intricacies of these conditions.
As new precision technologies continue to emerge, regulators and research teams will need to engage in preemptive planning to ensure nimble and timely responses to the development of these pioneering treatments. Additionally, ethical considerations such as equitable access to advanced therapies remain unresolved. Without clear policies and streamlined pathways for approval, many promising treatments face delays in getting to market. Prohibitive development costs may also result in therapies only being available in limited geographical regions and then only to patients who are able to pay for them out-of-pocket, limiting their impact on patient care.
Developing robust regulatory frameworks and policies which evolve in-step with precision medicine initiatives and technologies is therefore vital to successfully and safely delivering precision therapies to market, as well as helping to ensure equity of access.
Limited integration of emerging technologies
As precision medicine technologies continue to develop, substantial investment in technical infrastructure and specialized expertise (such as training for healthcare providers, the provision of more genetic counselors, and opening more software development and data analytics roles) will be required to ensure effective integration into healthcare systems and the delivery of personalized treatment plans. A unified, collaborative approach to tech and software integration, such as the integration of genetic data with electronic health records (EHRs) and specialist software systems, will be necessary to ensure equity of access and direct benefit to patients in the clinic.
Beyond the direct-to-patient clinical setting, synthetic biology and AI-driven computational approaches hold huge promise for identifying and developing potential new therapies for autoimmune and inflammatory conditions. Such technologies have the potential to enable far faster, more accurate identification of disease mechanisms and biomarkers than human analysis. Ultimately such technologies have the potential to deliver highly accurate diagnoses and inform the development of life-changing treatments. Yet without consistent infrastructure, stratification and management frameworks that enable researchers to collect, integrate and validate highly heterogeneous data, technological efforts to better understand these diseases will remain limited.
Integration to drive innovation
Precision medicine in autoimmune and inflammatory diseases is poised to revolutionize treatment, diagnosis and understanding of disease mechanisms. By offering tailored interventions that address the complexities of these conditions, patients can be offered personalized treatment plans which reduce the risks of broad immunosuppression, with the potential to significantly improve outcomes and quality of life. However, the current landscape remains fragmented, with gaps in diagnostic accuracy, therapeutic targeting, and a lack of integration of emerging technologies.
Key takeaway
The future of autoimmune and inflammatory disease treatment lies in precision, not just in science, but in the systems we build around it. From designing better tools to match the right treatment to the right patient, to ensuring those treatments are safe, accessible, and affordable, the path forward requires more than innovation alone.
To fully realize the potential of precision medicine, a holistic and integrated approach is essential – one that bridges scientific innovation, clinical application, and patient-centered care through universal data standards and cross industry collaboration. If we can build the right infrastructure and frameworks now, precision medicine won’t just be a future hope, it can become the new standard of care for complex immune conditions around the world.
Read our full whitepaper, "The future of precision medicine in autoimmune and inflammatory disease: A strategic framework," to find out more.
