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CRISPR Therapeutics Holds the First Approved CRISPR Therapy, but Its Stock Is Stuck

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CRISPR Therapeutics Wins Approval for Gene Therapy

CASGEVY carries a list price of $2.2 million per one-time treatment, and in the first quarter of 2026 CRISPR Therapeutics booked $1.46 million in total revenue against a net loss of $122.9 million. That gap explains much of why the company’s shares have gone nowhere, even though it holds the first approved CRISPR gene-editing therapy in the world.

The approval came in 2023, only 10 years after the company’s founding, which is quick for biotechnology and a record for so novel a technology. CRISPR Therapeutics decided from the start to use CRISPR-Cas9 exclusively, unlike some competitors, including companies tied to Jennifer Doudna, a co-discoverer of CRISPR, that also pursued systems such as CRISPR-Cas12a. A partnership with Vertex Pharmaceuticals, a specialist in rare diseases, helped. Emmanuelle Charpentier, one of the two women awarded the 2020 Nobel Prize in Chemistry for the technology, founded the company.

What the therapy does

CASGEVY, technical name exa-cel, treats sickle cell disease and transfusion-dependent beta thalassemia. Sickle cell disease stems from a mutation that forms abnormal hemoglobin, leaving red cells shaped like sickles that get stuck in blood vessels. Doctors collect a patient’s stem cells, edit them in a lab to produce fetal hemoglobin, and infuse them back. Treatment resulted in 94.2% of beta-thalassemia patients reaching independence from transfusion, and 97.4% of sickle cell disease patients. More than 500 patients worldwide have initiated treatment since launch.

Why the stock is stuck

A June 2026 analysis from NAI500 found shares still trapped in a trading range set in 2022. Each patient’s treatment takes months, from screening to infusion at specialized centers, and revenue is recognized only once the cycle is complete. Payors impose strict prior authorization requirements that slow access.

Vertex holds the larger stake under a 60/40 profit-and-loss split and leads commercialization. CRISPR has no other product on the market. Consensus estimates put full-year 2026 revenue at roughly $44 million and 2027 at about $151.7 million, against a market capitalization of $5.5 billion.

In March 2026 the company raised $600 million through convertible senior notes due 2031, convertible into more than 7.8 million common shares on top of a 96.5 million share base. It ended the first quarter with approximately $2.44 billion in cash, cash equivalents and marketable securities. The analyst consensus 12-month price target is $80.62, roughly 40% above recent trading.

The pipeline

The most watched experimental asset is CTX112, an off-the-shelf CD19 CAR-T therapy made from healthy donor cells. It has earned FDA Regenerative Medicine Advanced Therapy designation but remains in Phase 1/2 trials. Earlier programs target cardiovascular disease, diabetes, muscular dystrophies and cystic fibrosis. Of the Vertex diabetes collaboration, the company collected $130M in upfront and milestone payments in 2023, though Vertex backed out of the deal in January 2024.

Investors, in the NAI500 analysis, are waiting for a catalyst: faster CASGEVY patient volumes, positive late-stage CTX112 data, or a sign that profitability is near.

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