Gene-editing stocks are regaining favor on Wall Street this year as evidence builds for their treatments of deadly diseases such as sickle cell.
Shares of
Crispr Therapeutics
(ticker: CRSP) are up 45% this year, compared with the
Nasdaq Composite
‘s 31% rise. A week ago, the Food and Drug Administration indicated it might reach an approval decision around this year’s end for the company’s sickle cell treatment.
A thumbs-up would be the first U.S. approval for a therapy based on the Nobel Prize-winning technology known as Crispr.
Vertex Pharmaceuticals
(VRTX) partnered with Crispr Therapeutics on sickle cell.
Gene-editing companies first became public six years ago. When genetic technologies were leading the fight against Covid in 2021, Wall Street couldn’t get enough of any stock leveraging Crispr—a technology that can rewrite the faulty genes responsible for diseases such as sickle cell or the related blood cell disorder beta thalassemia.
The Crispr-based company
Intellia Therapeutics
(NTLA) saw its stock soar from $12 to $177 from 2020 to 2021 as it advanced its treatment for a debilitating disorder called ATTR amyloidosis. Then biotech stocks fell out of favor.
Intellia fell 80% from its 2021 peak. Crispr Therapeutics fell from around $220 at the start of 2021 to below $40 when this year began. It has since revived, as has Intellia, which is up 29% this year.
One factor in genetic medicine’s disfavor on Wall Street might have been the long clinical trials required to assure the safety of the novel approach. But the finish line is now in sight.
This past weekend, researchers told a European medical conference the sickle cell treatment of Crispr Therapeutics and Vertex freed some 90% of patients from the artery clogging crises caused by the disease, in the12 months after the one-time treatment. A comparable portion of beta thalassemia patients were freed of the need for transfusions in six months of follow-up.
The FDA already approved a genetic therapy for beta thalassemia last year from
bluebird bio
(BLUE). The company’s Zynteglo treatment uses a different approach from Crispr to insert healthy genes in thalassemia patients—with 90% achieving freedom from transfusion. The first seven patients started on the $3 million treatment this year. Bluebird is also right behind Crispr and Vertex in pursuit of a sickle cell approval.
In the U.S., sickle cell primarily affects people of sub-Saharan African ancestry, and medical resources haven’t always been accessible for that community. So it is gratifying the genetic disorder is now attracting commercial attention. Lots of attention, as it turns out.
At last week’s European Hematology Association meeting, along with the update from Crispr Therapeutics, researchers heard good initial results from a sickle cell treatment being tested by another Crispr technology developer,
Editas Medicine
(EDIT). Other gene editors pursuing sickle cell include
Beam Therapeutics
(BEAM).
So there may be a crowd of competing treatments for sickle cell patients. Demand could be limited to the most severe patients, at first, because current treatments require an initial chemotherapy to wipe out the genetically flawed blood cells before engrafting their genetically-altered replacements. Some half of patients experience severe nausea and fever during this preconditioning, and women emerge infertile.
Genetic medicine companies are searching for milder conditioning approaches.
With 100,000 Americans living with sickle cell, Raymond James analyst Steven Seedhouse thinks that some 1,700 patients a year will be receiving genetic treatments by 2027. He thinks each of these one-time procedures will cost about $1 million.
Gene-editing fixes for other illnesses should follow. For investors, it has been a long wait.
Write to Bill Alpert at william.alpert@barrons.com
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