Reduced Investment in Cell and Gene Therapies... Yet There Is Still Hope

[Asia Economy Reporter Chunhee Lee] Investment in cell and gene therapies (CGT), a new modality attracting attention in the bio industry, sharply declined last year. However, as new clinical candidates and approved drugs continue to emerge, expectations for the market itself remain high.

According to the '2023 Global Cell and Gene Therapy Market Trends' report published on the 7th by the Korea Bio Association's Bioeconomy Research Center, summarizing the JP Morgan Healthcare Conference presentation by Timothy Hunt, CEO of the Regenerative Medicine Alliance, the total investment in CGT companies last year amounted to $12.6 billion (approximately 16 trillion KRW). This represents a 44% decrease compared to $22.7 billion the previous year. While investment in CGT had shown a steady upward trend with $9.8 billion in 2019 and $19.9 billion in 2020, last year's overall investment market froze due to factors such as high interest rates.

Nevertheless, six new CGT drugs were launched in the US and Europe last year, and existing CGTs also achieved approvals for new regions or indications, demonstrating continued progress. The chimeric antigen receptor (CAR)-T therapy 'Karvykti' by Legend Biotech and Janssen received approval in the US and the European Union (EU). Among gene therapies, approvals were granted for ▲BioMarin Pharmaceuticals' 'Roctavian,' ▲PTC Therapeutics' 'Upstaza,' ▲UniQure and CSL Behring's 'Hemgenix,' ▲Ferring Pharmaceuticals' 'Adstiladrin,' and among cell therapies, Atara Biotherapeutics' 'Ebvallo' secured first approvals in the US and EU. Additionally, Novartis' 'Kymriah,' Bluebird Bio's 'Zynteglo,' and 'Skysona' expanded their business areas.

According to the report, as of last month, there are a total of 2,200 CGT clinical trials worldwide. By region, North America accounts for 43%, Asia-Pacific (APAC) 38%, and Europe 18%. Notably, 254 new CGT clinical trials were added last year, with 48% of these additions occurring in the Asia-Pacific region, showing the most remarkable growth. By phase, 202 trials have entered the final Phase 3 stage. Regarding target diseases, 60% of clinical trials focus on oncology, making cancer the most targeted field. Within cancer types, the ratio between solid tumors and hematologic malignancies is roughly equal at 50% each.

The distribution of developing companies showed a similar regional pattern. Among 1,457 companies, an 11% increase from the previous year, 686 (47.1%) are located in North America, followed by 492 in Asia-Pacific, 244 in Europe, and 35 in other regions. This situation reflects the recent intensified CGT development efforts in the US, whereas in Europe, many approved drugs have withdrawn from the market and clinical development is sluggish.

The report anticipates that decisions on the approval of 14 CGT products will be made in the US this year, with at least five expected to receive approval, making it the most active region globally for clinical trials and regulatory approvals. The US Food and Drug Administration (FDA), the regulatory authority, expanded its existing Office of Tissues and Advanced Therapies (OTAT) last September into the Office of Therapeutics Products (OTP), overseeing all advanced biologics, to enhance review capabilities and expertise related to CGT. The current review staff of 300 will be increased by 100 over five years.

Conversely, Europe is evaluated as facing challenges in CGT development and market entry. Among 24 advanced therapy medicinal products (ATMPs) that gained market access through approval, seven have withdrawn due to issues such as insurance reimbursement. Furthermore, only three new candidate substances entered clinical trials last year. The report attributes this to ongoing issues such as CGTs still being subject to genetically modified organism (GMO) regulations in Europe.

There is also speculation that this year will see the emergence of new therapies that have not existed before. The world's first gene therapy using gene editing technology (CRISPR) is expected to be approved, and in terms of indications, cell therapies for solid tumors and gene therapies for Duchenne muscular dystrophy are anticipated to receive approval.

This content was produced with the assistance of AI translation services.