In the face of COVID-19, cell and gene therapy space shows 'remarkable resilience': report

In the early days of COVID-19, the Alliance for Regenerative Medicine (ARM) was unsure how the pandemic and its accompanying economic downturn would affect the cell and gene therapy space.

“It was a really specific time when the world and the markets were clearly reeling from the first appreciation for the seriousness of COVID-19,” Janet Lambert, the organization’s CEO said.

Now, the numbers are in—and they’re better than ever. In the first half of 2020, the regenerative medicine sector raised $10.7 billion, more than the total capital raised in 2019 and a 120% jump over the first half of 2019, ARM found in a new report titled, “Innovation in the Time of COVID-19.” The proceeds were shared pretty evenly between cell therapy companies ($7.5 billion) and gene and gene-modified cell therapy companies ($7.9 billion), with companies focused on tissue engineering reeling in $84 million.

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That $10.7 billion was driven by a couple of outsize deals and includes $1.4 billion raised in five IPOs, $1.6 billion in follow-on offerings and $3 billion in venture capital. Chinese CAR-T player Legend Biotech led the pack with its mammoth $487 million Wall Street debut in June, but its peers netted considerable sums too. That same month, gene therapy companies Generation Bio and Akouos raised $230 million and $244 million, respectively. In February, another gene therapy outfit, Passage Bio, raised $284 million and gene-editing biotech Beam Therapeutics bagged $207 million.

On the venture side, Sana Biotechnology scored $700 million—almost as much as the five next largest private rounds raised by Orca Bio Elevate Bio, Legend, Freeline Therapeutics and Poseida, the report found. Like Legend, Generation Bio and Akouos also completed sizable private rounds the same year they went public.

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“All this enthusiasm for this sector right now is evidenced by these really astonishing financing numbers… I think the drivers of that enthusiasm remain in place and make me optimistic for the second half of 2020,” Lambert said. “We continue to see really promising clinical results. We continue to see products making it to market. We continue to see patient, regulator and payer enthusiasm for these products.”

Part of that enthusiasm stems from “an appreciation for the biotech sector generally,” Lambert said.

“Attention is being paid to what the biopharma sector can do for us all as we try to weather and get out of the pandemic,” she said, echoing the sentiments of venture capitalists who’ve managed to raise life sciences funds in spite of the pandemic.

The other side of the equation is the nature of biotech—because the drug development cycle is long, biotech investors aren’t looking for quarter-to-quarter returns, but at milestone readouts that can come more than a year after IPO, Jordan Saxe, head of healthcare listings at Nasdaq, said in a previous interview.

“Biotech is actually fairly well positioned to weather these kinds of events because you’re not relying on day-to-day consumer spending. You’re relying on meaningful clinical catalysts at the end of the day to really generate value, and that’s still going to be there in this environment, said Jason Pitts, Ph.D., a principal at Sofinnova, in ARM’s report.

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All this “gas in the tank” isn’t just bankrolling existing cell and gene therapies, but also driving company formation, Lambert said. For the first time, ARM counts more than 1,000 companies working in the sector, with more than 1,000 clinical trials going on worldwide. More than half of those studies are in phase 2, with just over a third in phase 1 and the remainder in phase 3.

Of those studies, 11 are testing regenerative medicine approaches against COVID-19, with several academic research centers and biopharma companies working on new treatments to treat the disease in the short and long term.

“Most of them are using cell therapies to address ARDS, or acute respiratory distress syndrome, which is a consequence of COVID-19,” Lambert said. Unlike other prospects in the pipeline, such as antibodies, which could potentially be used to prevent infection as well as treat it, regenerative treatments focus on repairing damage to the lungs or other organs that patients can suffer as part of COVID-19.