Niche start-ups spearheading cell and gene therapy innovation

Smaller companies targeting rare and extremely specific treatment areas are leading the charge in this emerging market.  

Cell and gene therapies are considered some of the most lucrative opportunity areas for biopharmaceutical developers due to the broad range of genetic diseases and significant unmet need for curative interventions.  

Medical science has traced thousands of clinical conditions to a genetic cause, while cancer, which is considered among the leading causes of death worldwide, also has genetic origins. 

The report, ‘Investor Series: Opportunities in the Cell and Gene Therapy Market’, found that start-ups targeting niche treatment areas are driving innovation in the sector, while big pharma players are also actively acquiring capabilities related to upcoming advanced therapy medical products (ATMPs). The first gene therapy trial was conducted in 1990 and it took almost three decades for the first of such interventions to enter the market. 

Among several companies capitalizing on the market is Swiss multinational Novartis, whose product for the treatment of spinal muscular atrophy (ZOLGENSMA) generated net revenues of approximately $1.35 billion in 2021 alone. Another is US biotech bluebird bio, whose gene therapy was given a stamp of approval this week by US Food and Drug Administration (FDA) advisers. If it goes to market, it will be the third gene therapy to be made available in the United States. 

The report notes that R&D activities slowed in the sector during the COVID-19 pandemic - a consequence of complex manufacturing protocols, extensive logistical considerations and supply chain-related concerns. However, the field still witnessed a considerable inflow of capital, with over $21 billion invested into various companies since the start of the pandemic. 

There are currently more than 1,200 product candidates in various stages of development and some experts suggest that by 2025, the US FDA may start approving around 10 to 20 cell and gene therapy products annually.