Cell & Gene Therapy Background
Cell and gene therapy clinical research is a field that holds promise for the treatment of many diseases. These therapies involve using living cells or genes to treat or prevent diseases and have the potential to revolutionize how we approach healthcare.
One aspect of cell and gene therapy clinical research is developing and testing new therapies. First, identifying the specific cells or genes that may be effective for a particular disease or condition, and then conducting clinical trials to determine whether the therapy is safe and effective. These clinical trials are essential for evaluating the effectiveness and safety of a therapy before it can be made commercially available.
There are different types of cell and gene therapies that are currently being researched, including stem cell therapies, gene editing therapies, and immune cell therapies:
- Stem cell therapies use stem cells, which are cells that can develop into a variety of different cell types. These therapies have the potential to repair or regenerate damaged tissue and are being researched as a potential treatment for diseases, including heart disease, diabetes, and neurodegenerative disorders.
- Gene editing therapies involve modifying an individual's genetic material to correct or prevent the expression of certain genes. These therapies may cure genetic disorders and are being researched as a potential treatment sickle cell anemia, cystic fibrosis, and more.
- Immune cell therapies use immune cells, such as T cells, to attack diseased cells in the body. These therapies have shown promise in the treatment of cancer and are being researched in a variety of cancers.
These therapies have the potential to significantly improve the lives of patients and offer hope for those suffering from debilitating diseases. In 2022, there were 1,022 ongoing cell and gene clinical trials and there is still much research to be done.
Changes in R&D Landscape
Cell and gene therapy clinical research has seen significant advancements in recent years, but there are still challenges facing startups in this field, including manufacturing, pressure to achieve early phase clinical success, and reimbursement issues.
Manufacturing Approaches & Improved Technology
One area of change in this landscape is the improvement of manufacturing approaches and technology. Researchers are working to arrive at scalable single-use technology solutions for the logistic and equipment issues surrounding the production process, which has been heightened by the pandemic supply chain crisis. Improving technology has also led to an increase in the number of emerging companies and volume of clinical trials. With more clinical research organizations, competition increases, leading to difficulties in obtaining funding and creating pressure on clinical sites and investigators.
Solutions to these challenges include reducing the burden on clinical sites through enhanced training leveraging user-friendly, on-demand technology. To address drug product supply chain and logistics challenges associated with ex vivo trials, it is essential to develop and support the end-to-end chain of custody and patient journey from an early stage in protocol development to de-risk the treatment process related to autologous or allogeneic therapies.
Alternative Pathways to Advance Novel Treatments
Another area of change is the use of alternative regulatory pathways to advance novel treatments for patients with unmet needs, particularly in rare diseases. These pathways, such as the US Food and Drug Administration’s various accelerated approval programs, allow for faster development of treatments for these patients.
The 21st Century Cures Act and the Regenerative Medicine Advanced Therapy (RMAT) Designation have also been implemented to speed up the review process for cell and gene therapies. However, these changes have led to an increase in regulatory submissions and reviews, and the need for new approaches to real world evidence. Clinical research teams can address these challenges by focusing on patient safety, proactively managing data readiness, and establishing strong lines of communication with patients, care partners, and families. This includes:
- New approaches to feasibility and study start up, especially for rare disease that rely on a more direct line of communication to patients, care partners and families.
- Collaborating with regulatory start up teams with specific expertise in dealing with nuances of CGT research trials, such as Institutional Biosafety Committees (IBC) reviews.
- Clinical project teams operating with a higher level of integration across functional areas to ensure data readiness and cleanliness, requiring new tools and technology for information sharing and communication with sponsors, sites, CROs and third parties involved.
Expansion of CAR T and Beyond
In the last five years, five CAR T-cell therapies received approval by the US Food and Drug Administration for hard-to-treat blood cancers and bone marrow cancers. Drug developers are now also researching therapies for hard-to-treat solid tumors and, outside of oncology, for autoimmune and cardiovascular diseases.
With studies in later phases of clinical research and new therapeutic areas, there is an increased need to handle larger volumes of data with specific regional or site-level requirements. Researchers need to comply with the long-term safety follow up of treated patients, which presents unique challenges as protocol requirements must remain observational. Clinical research teams are addressing these challenges by establishing strong data management processes and working closely with regulatory agencies.
Changes in the reimbursement landscape for cell and gene therapies have led to challenges for startups progressing new drug candidates. The high cost of these therapies has made reimbursement difficult, and there is a need for new payment models to ensure patients have access to these treatments. Sponsors and clinical research teams can work to address these challenges by collaborating with payers and policymakers early on to develop payment models that ensure access to these therapies for patients.
Overall, cell and gene therapy clinical research is a rapidly advancing field with significant potential for improving patient outcomes, but there are challenges to be addressed still. By addressing these challenges in close collaboration with experienced clinical research teams, sponsors are in a stronger position to get ahead of these growing challenges and help bring these therapies to patients and make an impact on healthcare.