Cell and Gene Therapy (CGT) has been booming worldwide in recent years. Continuous advancements in this innovative field are revolutionizing how biopharma companies treat and potentially cure certain debilitating human diseases, such as cancer or genetic disorders, thereby enhancing healthcare results.
According to the Alliance for Regenerative Medicine (ARM)’s H1 2022 report Regenerative Medicine: The Pipeline Momentum Builds, following two successive record years of financing flowing into the sector, 2022 is on track for a significant retrenchment. The biotechnology sector’s equity performance is down, with the strongest punishment doled out to small, early-stage cell and gene therapy companies. Inflationary conditions and expectations have been particularly unkind to the sector. Initial public offerings have nearly dried up, after hitting a record in 2021. But venture capital financing for the sector, while off its 2021 peak, remains robust and is the single largest driver of sector investment. This is a clear sign of continued excitement around scientific breakthroughs and the opportunity to change the treatment paradigm across a range of rare and prevalent diseases (ARM, 2022, p. 2).
There were 2,093 trials ongoing globally at the end of June 2022 (ARM, 2022, p. 7). By region, North America leads with 808 active clinical trials (down 15% from 2021), followed by Asia Pacific with 640 trials (down 15%), Europe with 329 (down 14%), and 88 active trials in all other regions (down 7%) (ARM, 2022, p. 8).
Asia Pacific takes the lead with 61 new clinical trials, accounting for the largest share at 42% of the total 144 new clinical trials during the first half of 2022, and includes a healthy number early in the pipeline (30 new clinical trials in Phase 1); 26 in Phase 2 and 5 in Phase 3.
North America follows closely behind, with 51 new clinical trials at 35% (19 in Phase 1, 24 in Phase 2, and 8 in Phase 3). Europe is the only region with a bottom-heavy pipeline, which has slowed ‘to a trickle’, accounting for the smallest share (11%) with 16 new clinical trials started in H1 2022 (2 in Phase 1, 7 in Phase 2, and 7 in Phase 3). 16 new clinical trials were initiated in other regions as well, but in contrast to Europe, 7 of them were in Phase 1, 7 in Phase 2, and 2 in Phase 3 (ARM, 2022, p. 8).
Cell therapies make up the largest category of ongoing trials (968, 46%), followed by cell-based immuno-oncology (CBIO) (721, 34%) and gene therapies (372, 18%). Tissue-engineered therapies comprise the remaining 32 (2%) of ongoing clinical trials in the cell and gene therapy sector. All categories have declined compared to 2021 (ARM, 2022, p. 9).
Since 2021, a 5% increase in Cell, Gene, and Tissue-engineering therapeutic developers worldwide (1,369) was observed. North America leads with 651 developers (+2% vs. 2021), followed by Asia Pacific with 453 (+10% vs. 2021). European developer growth remains flat since 2021 (230).
China is the fastest-growing country, accounting for 236 developers at 52% of the Asia Pacific region (ARM, 2022, p. 11). According to recent research conducted by Nature, CAR T-cell therapy clinical trials in China have accounted for over 50% of registered CGT studies worldwide. While lagging behind Cell Therapy, China’s gene therapy (GT) market shows promise with great potential, as China’s large population base and ready patient pools could be an inherent advantage. (Liu, 2022).
The sector experienced an unprecedented uptick in investment during the pandemic in 2020 and 2021, due to the high level of public attention on healthcare innovation and the low cost of capital.
After two record-breaking years of investment in regenerative medicine and advanced medicines, the biotech sector’s 2022 performance is likely to land between $9.8B and $13.5B in total annual investment based on current patterns, returning to the pre-pandemic levels in 2018 and 2019. There were 53 deals worth $5.6B in North America (-68% YoY), 23 deals worth $675M in Asia Pacific (-69% YoY), and only 13 deals worth $398M in Europe (-88% YoY) during the first half of 2022.
“What is the culprit of this downturn? External factors including inflation and retrenchment in biotech financing, including cell and gene therapy, after record levels of investment, have combined to produce an extremely challenging investment environment during 2022. The slowdown affects nearly all parts of the interconnected financing system, from early-stage venture capital to initial public offerings (IPOs) and the performance of publicly traded companies. Small, early-stage companies — and there are many such cell and gene therapy companies — are particularly affected by inflation expectations because they are furthest from generating profits (ARM, 2022, p. 14).
Despite a 27% decrease in venture capital from H1 2021, it remains a bedrock of funding for the sector and is pacing at 40% of 2021 FY — the smallest decline of the financing categories along with private placements (which increased in number from 3 to 11 YoY). This indicates continued investor excitement about scientific breakthroughs and new treatment possibilities.” (ARM, 2022, p. 15).
According to Precedence Research, the global cell and gene therapy market size was exhibited at $18.61B in 2022 and is projected to surpass $93.78B by 2030, growing at a CAGR of 22.41% during the forecast period from 2022 to 2030. By therapy type, the gene therapy segment accounted highest revenue share of 52% in 2022. By region, North America is anticipated to account for the largest portion of worldwide revenue, and it is anticipated that it will continue to hold the lead during the forecast period. Asia Pacific is expected to hold its position as the hub of cell research and therapy. Additionally, it is anticipated to grow at the fastest CACR, as regenerative medicine-focused biotech companies are proliferating in the Asia Pacific region.
Notes: This breakdown article provides additional content from NAI Interactive Ltd.’s presentation at the GCFF Virtual Conference 2022 – Healthcare Investment (Asia Pacific) on Nov 16, 2022.
PPT Download: https://nai500.com/wp-content/uploads/2022/11/FINAL_Gilbert-PPT-GCFF-Nov-16.pdf.
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Alliance for Regenerative Medicine (ARM). (2022). Regenerative Medicine: The Pipeline Momentum Builds. (2022, October 20). https://alliancerm.org/sector-report/h1-2022-report/ .
Liu, B. (2022, Apr 11). One to Watch: China’s Cell and Gene Therapies Industry. Pharmaceutical Executive. https://www.pharmexec.com/view/one-to-watch-china-s-cell-and-gene-therapies-industry .
Precedence Research. (2022). Cell and Gene Therapy Market. https://www.precedenceresearch.com/cell-and-gene-therapy-market .
Precedence Research. (2022, Nov 30). Cell and Gene Therapy Market Size to Surpass USD 93.78 BN by 2030. Yahoo Finance. https://finance.yahoo.com/news/cell-gene-therapy-market-size-120000593.html .
Wei, J., Guo, Y., Wang, Y. et al. Clinical development of CAR T cell therapy in China: 2020 update. Cell Mol Immunol 18, 792–804 (2021). https://doi.org/10.1038/s41423-020-00555-x .