Gene Therapy Ups and Downs

This month’s gene therapy news includes celebrations of promising new developments running beside gloomy takes on the future of the industry.

Early in February, a STAT headline quipped “The CRISPR companies are not OK”: 

Interviews with over 75 investors, academics, executives, analysts, and employees reveal a swaggering industry suddenly and deeply humbled. Put simply, they’ve realized — billions of dollars and more than a few ill-conceived companies later — that there are surprisingly few places today where you can both cure a disease with gene editing and make money.

The article goes on to explore in depth the patterns that have led to the current predicament: persistent scientific stumbling blocks, shifts in the market’s appetite for long-term investments, and steady scaling back from lofty ambitions of “curing all genetic disease” to doing what’s “doable.”

Endpoints raised the question “Will CRISPR matter?,” similarly pointing to the field’s financial troubles as well as the seeming retreat from earlier ambitions to target rare diseases. 

And this was all before bluebird bio sold to private equity for a fraction of its previous value and Pfizer pulled its last remaining gene therapy off the market, signaling challenges for gene therapy companies more broadly. 

The sky-high costs of gene therapies also continue to generate coverage. ProPublica explored how companies justify record prices on drugs developed with taxpayer funds and private donations. El País looked at an Italian nonprofit seeking regulatory approval for a gene therapy in Europe, while CNBC questioned what would happen to a Biden-administration plan to help state Medicaid plans afford gene therapies for sickle cell disease.

Taken together, the articles highlight the disconnects between the promise of revolutionary treatments, the real difference gene therapies have made in the lives of some patients, and the lack of a clear path to profitability in investors’ eyes.

Meanwhile, news reports touted a first in utero gene therapy success, a “cure” for an inherited form of blindness, and promising results in a clinical trial for Duchenne muscular dystrophy.

Are these ups and downs just bumps in a longer road toward gene therapy success, or another round on the roller coaster of hype and disillusionment?