The human body is a remarkable structure composed of countless minuscule cells—the basic building blocks of life. Like bricks used to build a house, the intricate organization of these cells forms the complex structure of the body. However, like any structure, the body is not invulnerable and can suffer from structural damage and dysfunction like injuries, diseases, and wear and tear. Cell and gene therapies (CGTs) provide groundbreaking approaches to repairing or replacing dysfunctional cells and enabling the smooth functioning of the human body.
Though still in their early days, these therapies could change the way we tackle previously untreatable diseases. There is no better illustration of their potential than in the recent case of a 19-month-old baby girl in the UK diagnosed with a rare genetic disorder (metachromatic leukodystrophy [MLD]), who showed no signs of it after being treated with a gene therapy called Libmeldy developed by Orchard Therapeutics. The treatment involved extracting stem cells from the child, replacing the faulty gene with a healthy one capable of producing a crucial enzyme, and administering the modified cells back into the body.
As of August 2023, more than 30 CGTs have been approved by the US FDA, with several other therapies awaiting approvals as of the same date. The number of clinical trials being conducted for CGTs totaled 1,600+, across the North American, European, and Asia-Pacific regions.
Cell and gene therapy (CGT) is a branch of genomic therapeutics that applies to the healthcare industry with specific focus on the biotechnology and life sciences subsegments.
Examples of CGT comprise therapeutic developers that offer their platforms primarily to pharmaceutical companies to facilitate use cases such as research, product development, and commercialization of CGT candidates across areas such as regenerative medicine and therapeutics based on vectors such as ribonucleic acid (RNA), adeno-associated viruses (AAVs), and T-Cells. Furthermore, these platforms are used in pre-clinical testing of drug candidates.
We have identified key CGT use cases below:
Disruptors and incumbents in this space develop cell and gene therapeutics or materials to assist with the development of either type of therapeutic.
The industry players are segregated based on the type of therapy offered, with cell therapies being further broken down based on the type of therapy being developed: 1) chimeric antigen receptor T-cell (CAR-T) therapy, 2) stem cell therapy, and 3) other approaches of cell therapies.
As of September 2023, startups in the gene therapy segment had raised ~USD 7 billion in funding; the highest funds raised by a single segment, indicating the investment-heavy nature of developing gene therapies. Across cell therapy, startups developing CAR-T had raised nearly USD 3 billion as of September 2023, accounting for over half of funds raised by all cell therapy players.
Major global pharmaceutical giants such as Bayer, Pfizer, Novartis, and Johnson & Johnson are among the incumbents active in this space, with partnerships and product collaborations being the most common growth strategy employed by these players, along with some big-ticket acquisitions to accelerate the product portfolio.
Major global pharmaceutical giants such as Bayer, Pfizer, Novartis, and Johnson & Johnson are among the incumbents active in cell and gene therapy, with the most common growth strategy being partnerships and product collaborations with startups and other incumbents in the field. Many of the incumbents’ own in-house activities are focused on gene therapy, with most incumbents having multiple therapies in their pipelines for various diseases.
Roche’s acquisition of Genentech more than a decade ago for nearly USD 47 billion remains the largest acquisition in the space to date. The largest deal in more recent times was Novartis’ acquisition of AveXis for nearly USD 9 billion in 2018 to absorb the latter’s gene therapy portfolio for spinal muscular atrophy (SMA) and Astellas Pharma’s acquisition of Iveric Bio in 2023 for nearly USD 6 billion in a bid to expand the former’s eye disease pipeline.