Gene Therapy Market: Big Pharma Bets Big

Last week, I came across an interesting article by Andrew Dunn titled Sanofi investing in gene therapy as R&D focus turns toward rare disease. The article caught my attention because in June, this year, Sanofi scaled back its four-year-old gene therapy alliance with Voyager Therapeutics, for the second time. If we just look at the news titles, Sanofi has shown contrasting behavior in the gene therapy space.

In the last few months, Sanofi has taken several steps in revamping its R&D efforts, which involved axing of more than 450 jobs employees in France and Germany, to setting up a new headquarter in the UK. As per the company’s new R&D chief, the company is steering away from areas for which, it has historically been known for, namely cardiovascular and diabetes. Now, the company is looking to expand its capabilities in gene therapy, an area across which, all its peers have already had a headstart.

In this article, we will look at the initiatives that different big pharma players have taken in the gene therapy space.

Over the years, various well-established big pharmaceutical players have marked their presence in the gene therapy domain, by either expanding their respective product portfolios, partnering with other companies, investing in gene therapy-related product development initiatives or building intellectual capital related to such advanced therapies.

If you look at the below-added exhibit, we can clearly see the activity of big pharma in the gene therapy space. Almost all the big pharma players have a late-stage asset in the development pipeline (of their own / their partner company). Novartis, for example, has actively invested in various companies, such as Akouos, Anaeropharma Science, Homology Medicines, Intellia Therapeutics, Poseida Therapeutics, and Vivet Therapeutics, in order to assist them to progress their initiatives related to gene therapies and gene-editing therapies.

Gene Therapy Big Pharma heat map shows which player is strong in early or late phase of development, funding, technology or partnership development based on the data gathered by Roots Analysis

In this crowded space wherein several big pharma players are involved, it is important for every player to identify the areas wherein they have a USP. The below-added exhibit shows the benchmarking of different big pharma players across different therapeutic areas they are focused on.

Gene Therapy Big Pharma heat map shows USP for each player based on the data gathered by Roots Analysis

Novartis is primarily focused on developing gene therapies intended for ocular disorders, oncological disorders, and metabolic disorders, whereas Pfizer is targeting neurological and hematological disorders. It is worth highlighting that therapeutic areas such as autoimmune disorders, cardiovascular disorders, infectious diseases, and liver diseases hold significant opportunities for these players, owing to very limited competition within the big pharma players.

Each of these big pharma players has adopted different strategies to set themselves apart in the gene therapy space. These involve, employing different gene modification approaches (gene augmentation, immunotherapy, oncolytic therapy, antibody-based therapy, gene regulation, and gene editing), developing different type of gene therapy (ex-vivo, in-vivo), and using various types of vector (adenovirus vector, adeno associated virus vector, lentivirus vector, non-viral vector, and other novel vectors).

What remains to be seen is who will win the race in this gene therapy market, which is expected to be worth close to $12 billion by 2030.