Mainstreaming Genetic Medicine: A New Era for Chronic Disease Management
This is part of our ongoing content series from the Syneos Health Trends report.
Exploring genetic medicine as a healthcare revolution where chronic diseases are no longer just managed but cured.
Genetic medicine is making a bold leap from niche treatments for rare diseases and cancer to the mainstream. With advances in cell and gene therapies (CGT), the potential for curative solutions to chronic, autoimmune and neurodegenerative diseases is becoming increasingly realistic. Yet, commercial adoption faces hurdles related to cost, access and operational logistics.
The Transition from Rare to Common Conditions
CGT has shown immense promise in treating rare conditions, but now researchers are broadening their scope to more prevalent chronic diseases. “Everything developed over the past 15 to 20 years in oncology is now being transposed for other indications,” noted Manfred Lauth, MD, Vice President of Medical & Scientific Strategy at Syneos Health. This shift includes autoimmune diseases such as lupus and rheumatoid arthritis, where early clinical results indicate the possibility of long-term remission.
Similarly, breakthroughs in neurodegenerative conditions are underway, such as gene therapies designed to cross the blood-brain barrier, a historic challenge in treating diseases like Parkinson’s and Alzheimer’s. According to Abhi Gupta, Head of Cell and Gene Therapy at Syneos Health, “the recent FDA approval of a gene therapy for Duchenne muscular dystrophy exemplifies how regulators are recognizing the urgent need for innovative solutions.”
Operational and Commercial Challenges of CGT Adoption
Despite clinical success, the adoption of genetic therapies into real-world settings remains uneven. Regarding treatment and trial access for sickle cell disease patients, Gupta notes, “While there are treatment centers in major cities like New York and Boston, there’s a significant gap in the South, where many sickle cell disease patients reside.” This disparity highlights the operational hurdles that must be overcome for widespread accessibility.
Moreover, the price tag of these therapies—often exceeding $1 million per treatment—raises questions about affordability and reimbursement. However, Gupta suggests that innovative pricing models, such as outcomes-based agreements, could help align costs with long-term savings for healthcare systems.
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Collaboration and Innovation: Keys to Scaling
Bringing CGT to a broader audience will require a collaborative effort across academia, industry and regulatory bodies. “Many big pharma companies are realizing they can’t do it alone, so they’re turning to partnerships with biotech and academic institutions,” noted James Barwick-Silk, Senior Director of Cell and Gene Therapy Strategy at Syneos Health. In addition, global initiatives are emerging to standardize regulations and streamline approvals, which could further accelerate the rollout of these therapies worldwide.
An often-overlooked area of innovation is logistics. As Gupta highlights, ensuring the safe transportation of these high-value, temperature-sensitive treatments is critical to success. “Losing a single dose isn’t just a financial loss—it could mean losing a patient or an entire trial”.
When effective, CAR-T may spare patients exposure to toxicity and long-term side effects, compared with non-targeted treatments, but chemotherapy may be required to condition the patient’s immune system before the therapy, and may also be necessary down the road down the road if the cancer returns. These contingencies can complicate the consent process, as families struggle to understand the complex tradeoffs.
A Dawn of Hope for Chronic Disease Patients
While mainstreaming genetic medicine presents complex challenges, the potential benefits are game-changing. The ability to provide long-term, potentially curative treatments for chronic conditions could significantly reduce healthcare costs and improve quality of life for millions. “We are moving away from symptomatic or disease-modifying treatments toward curative therapies.”
As 2025 unfolds, genetic medicine stands on the brink of transformation. By addressing operational barriers, embracing collaborative innovation, and developing sustainable pricing models, we may soon witness a healthcare revolution where chronic diseases are no longer managed but cured. Genetic medicine, once a futuristic concept, is rapidly becoming today’s reality.
Learn more about how Syneos Health is reimagining what’s possible in healthcare. Download our 2025 Health Trends report today.
Contributors
Manfred Lauth, MD | Vice President, Medical & Scientific Strategy
Abhi Gupta | Senior Vice President & Head of Cell and Gene Therapy
James Barwick-Silk, Senior Director of Strategy, Cell and Gene Therapy
