State of the Industry: The Financial, Clinical, and Scientific Landscape for Cell and Gene Therapies
Cell & Gene Therapy Insights 2020; 6(1), 47–54
10.18609/cgti.2020.007
2019 was a year of significant growth in the regenerative medicine sector. Thousands of patients are now benefitting from commercial regenerative medicines, and the impact of the early cell and gene therapies is dramatic. The clinical pipeline is robust, with nearly a hundred Phase 3 trials underway, several late-stage products poised for approval, and next-gen technologies such as gene editing beginning to enter the clinic. Therapeutic developers are increasing their focus on solving manufacturing challenges. The sector continued to attract billions in investment, further fueling our scientific, clinical and commercial progress.
Each year, the Alliance for Regenerative Medicine (ARM), the international advocacy organization for the cell and gene therapy and broader regenerative medicine sector, hosts a Cell and Gene State of the Industry briefing, presenting global sector data at the Biotech Showcase at the JP Morgan Healthcare Conference. The industry overview is followed by two panels focusing on the latest developments in the sector. Recordings and slides are available on the ARM website [1]. This year, for our tenth annual briefing, I spoke to over 1,000 attendees in-person and online. This group of people – scientific and industry experts, regulators, payers, investors, and patient advocates – are incredibly knowledgeable and motivated to bring these therapies to patients across the globe, and we at ARM are grateful to be spearheading efforts to get safe and effective medicines to patients.
Since I came to ARM in 2017, this sector has really come of age – and there is still so much more to come. While 2019 was very successful, we still have considerable work to be do together to ensure that the early clinical promise and commercial successes of these therapies translates to widespread patient access. With that in mind, I wanted to share some of the major trends we’ve seen in 2019, as well as the outlook for 2020.