Cell & Gene Therapy Background

Cell and gene therapy clinical research is a rapidly growing field that holds great promise for the treatment of a wide range of diseases. These therapies involve the use of living cells or genes to treat or prevent diseases and have the potential to revolutionize the way we approach healthcare.

One key aspect of cell and gene therapy clinical research is the development and testing of new therapies. This involves 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 to the public.

There are several 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 involve the use of 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 a wide range of diseases, including heart disease, diabetes, and neurodegenerative disorders.
• Gene editing therapies involve the modification of an individual's genetic material to correct or prevent the expression of certain genes. These therapies have the potential to cure genetic disorders and are being researched as a potential treatment for conditions such as sickle cell anemia and cystic fibrosis.
• Immune cell therapies involve the use of immune cells, such as T cells or natural killer cells, to attack cancer cells or other diseased cells in the body. These therapies have shown great promise in the treatment of cancer and are being actively researched in a variety of cancer types.

These therapies have the potential to significantly improve the lives of patients and offer new hope for those suffering from debilitating diseases. As of 2022, there are 1,022 ongoing cell and gene clinical trials and there is still much human 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. Challenges primarily include manufacturing, pressure to achieve early phase clinical success, and reimbursement issues.

Manufacturing Approaches & Improved Technology

One area of change in cell and gene therapy clinical research is the improvement of manufacturing approaches and technology. Researchers are determinedly working to arrive at scalable single-use technology solutions for the logistic and equipment issues surrounding the production process. There are unique supply chain and logistics challenges that have 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. As clinical research in the area is becoming more crowded, competition is increasing which can lead to difficulties in obtaining funding and create 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 compassionate use programs and 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 – this requires 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. While oncology remains the most active therapeutic area, drug developers are now also researching promising therapies for hard-to-treat solid tumors and, outside of oncology, for autoimmune and cardiovascular diseases.

As studies progress into 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 also need to comply with the requirement of 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.

Finally, 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 led to difficulties in obtaining reimbursement, and there is a need for new payment models to ensure that 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. However, there are still challenges that must be addressed to ensure the success of these therapies. 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 more patients and make a real impact on healthcare.

Sources:

Cell & Gene Therapy Investment Outlook In 2022 & Beyond

 FDA Approved CAR T-cell Therapies