- Biotech developers of cell, gene and regenerative therapies raised $14 billion in funding over the first six months of 2021, an enormous sum that equals roughly three-quarters of last year's record total of $19.9 billion.
- Venture capital firms have been particularly active, according to numbers compiled by the industry association Alliance for Regenerative Medicine in a new report, pouring more than $5 billion into funding rounds for private biotech companies in the field.
- More of those companies are testing public markets, with a record 20 cell and gene therapy developers pricing initial stock offerings so far this year, including a number of the largest across the biotech industry. There were fourteen cell and gene therapy IPOs across all of 2020, by comparison.
The amount of money flowing into cell and gene therapy development suggests investor confidence in the field's future remains high, even as clinical, regulatory and commercial setbacks have hindered a number of companies, such as Bluebird bio, Biogen and Adverum Biotechnologies.
Bluebird, for example, has had to halt testing of several of its gene therapies in response to three clinical trial participants developing cancer. Two of those cases were later ruled unrelated to treatment or were misdiagnosed, while investigation into the third is ongoing. But the events have revived long-standing safety concerns that could affect other companies besides Bluebird.
And, in a further sign of the field's growing pains, Bluebird recently said it would withdraw from the European market after encountering difficulties securing reimbursement for its two gene therapies approved there.
Measured by stock market performance, setbacks like those appear to have weighed on gene therapy company shares, which collectively underperformed the broader Nasdaq Biotechnology index, according to ARM. Cell therapy companies are a different story, however, and continue to outperform the overall biotech sector.
Even as individual companies encounter difficulties developing or selling their therapies, ARM's report makes clear the pace of the field's growth. Over the first six months of 2021 alone, 13 cell and gene therapy developers priced IPOs that raised more than $100 million, while 20 private companies closed venture financings above that sum.
Globally, ARM claims there are now nearly 1,200 companies working on cell-, gene-, or tissue-based treatments, about 200 more than there were at the same point last year. Those companies are sponsoring a pipeline that's swelled to include more than 1,300 clinical trials, with upwards of 100 having advanced into Phase 3.
Clinical trial progress is beginning to bear fruit, as well. So far in 2021, the Food and Drug Administration has approved two new CAR-T cell therapies — Bristol Myers Squibb and Bluebird's multiple myeloma treatment, Abecma, and the former company's infusion for lymphoma, Breyanzi — as well as a new type of skin graft from Mallinckrodt.
Meanwhile, in gene editing, partners CRISPR Therapeutics and Vertex, as well as Intellia Therapeutics, have marked major milestones with positive early data for treatments targeting beta thalassemia, sickle cell disease and a rare inherited condition.