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The company shared early-stage results for CTX310, a treatment that edits genes inside the body to lower heart disease risks. At the highest dose tested, patients saw their LDL cholesterol, the "bad" kind, drop by an average of 53 percent, while triglycerides fell by 48 percent. The treatment was well tolerated with no serious safety issues reported.
This is an important step because it shows the company's technology can work for common conditions like heart disease, not just rare blood disorders. While its first approved drug requires a complex process outside the body, this program targets the liver directly with a single infusion. If these results hold up in larger trials, it could open up a much larger market for the company's gene-editing tools.
Source: GlobeNewsWire
Analysts at Goldman Sachs raised their price target for the company to $55. This move follows the company's recent quarterly update and suggests a slightly more optimistic view of the stock's value. Even with the increase, the new target remains well below the average analyst target of $71.
Source: Goldman Sachs
The company reported a loss of 94 cents per share, which was better than the $1.19 loss analysts expected. While revenue remains small at about 10 million dollars, the focus for a biotech firm at this stage is on how its first approved drug, Casgevy, is performing and how its other research is progressing.
Management noted that Casgevy is gaining traction and recently received approval for children as young as 2 years old. The company also started early-stage trials for two new treatments that work inside the body to treat high blood pressure and a specific lung and liver disorder. This progress is important because it shows the company is moving beyond its first blood-disorder drug into larger markets.
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Source: 8-K filing
H.C. Wainwright set a target of $80 for the stock, which is higher than the average analyst target of $71. This reflects a more bullish view on the company's ability to turn its gene-editing platform into a successful commercial business. For a biotech company, these targets often depend on how quickly new drugs can reach patients and whether early lab results hold up in larger trials.
Source: H.C. Wainwright
The company has split its last eight reports between beats and misses. This choppy record shows management is still figuring out how to forecast costs and timing for a business that is just starting to sell its first products.
| Expectation | |
|---|---|
| EPS | $-1.06 |
| Revenue | $15M |
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