Personalized medicine and biotech
The shift toward tailored treatments
2023 highlights pivotal development in personalized medicine. The market, valued at $13 billion in 2022, is anticipated to reach $62.5 billion by 2032, indicating a transformative shift towards tailored treatments. The rising prevalence of genetic disorders, increased acceptance of CAR-T therapy, and technological advancements like CRISPR-based tools are further boosting market demand. Next-generation cell and gene therapies have the potential to address the root cause of genetic and acquired diseases and are at the forefront of personalized medicine.
Approval and development of gene and cell therapies
The American Society of Gene & Cell Therapy reports the global regulatory approval by therapy type;
27 gene therapies
26 RNA therapies
65 non-genetically modified cell therapies
There are 3,866 therapies in development, with gene therapies accounting for 53%, RNA therapies for 25%, and non-genetically modified cell therapies for 22%.
As of late November 2023, clinicaltrials.gov reports 20,073 studies that indicate cell therapy as the intervention and treatment. The remarkable number of gene therapies in development and the multiple studies being run illustrate the investment in precision medicine. CAR T dominates the pipeline, accounting for 47% of the genetically modified cell therapies in development, and other technologies such as TCR-NK, CAR-M, and TAC-T account for 36%. TCR-T follows at 11% and CAR-NK at 6%.
Manufacturing, financing, and regulatory approvals are challenging, given the relatively early progress in cell and gene therapies. A case in point is the FDA’s recent announcement that it is investigating the occurrence of T-cell malignancies in products targeting lymphoma. While the benefits are believed to outweigh the risks, we are still early on in our journey. Continued scientific and development innovation, supply chain reliability (vector, plasmids, etc.), and growing regulatory familiarity are paving the way for future therapies.
Addressing rare diseases with precision medicine
A particularly encouraging trend is the focus on oncological and non-oncological rare diseases within RNA therapies. Diseases such as pancreatic, liver, and ovarian cancer are being addressed with a personalized approach, considering their specific genetic or molecular origins. Similarly, non-oncological rare indications like Duchenne muscular dystrophy, amyotrophic lateral sclerosis, and Huntington’s disease are gaining attention, showcasing a commitment to personalized solutions for unmet medical needs.
Cell therapy financing trends to continue in 2024
Cell therapy financing has seen mixed trends across financing, alliances, and acquisitions. Financing deals are up 24% from Q3 2022 to Q3 2023. Acquisitions stayed steady at roughly nine acquisitions per quarter, while alliance deals have seen more fluctuation quarter to quarter. Personalized medicine requires sustained investment to support the development of innovative therapies. We expect the mix of M&A and financing activity to continue in 2024.
Slalom contributors: Munaz Muntasir, Colby Voorhees
