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Following the recent FDA green light for the gene therapy Casgevy, “it is unlikely” that approval of a similar product will take place “any time soon,” according to a report by GlobalData.
According to GlobalData, only 24 CRISPR-based drugs are in Phase 2 trials, with 88% of such drugs in earlier-stage or preclinical development.
CRISPR and partner Vertex Pharmaceuticals (VRTX) made history earlier this month when Casgevy became the first FDA-approved treatment utilizing CRISPR gene-editing technology. The product was approved for the treatment of sickle cell anemia and is awaiting approval for transfusion-dependent thalassemia.
“The Casgevy approvals represent a significant milestone for revolutionary genome editing systems,” Jasper Morley, drugs intelligence analyst at GlobalData, stated. “However, given the relative immaturity of the CRISPR drugs pipeline, which features very few late-stage products alongside a low likelihood of approvals, it is unlikely to see another drug approval in the near future.”
In its report, published earlier this week, GlobalData listed the CRISPR Therapeutics (NASDAQ:CRSP) drug candidate CTX-110 as the likely next CRISPR-based gene therapy to be globally launched, possibly towards the end of 2025.
That estimate seems based on the fact that CTX-110 was further down the pipeline than most other CRISPR-related prospects, as it had reached Phase 2 development for B-cell malignancies. However, even GlobalData gave the product a relatively thin chance of eventually reaching the market, saying it had less than 50/50 odds of making a Phase 3 trial and only a 31% chance of approval.
Meanwhile, in early December, CRSP announced that it was shifting its focus from CTX-110 and another prospect, CTX-130, to other potential therapies, namely CTX-112 and CTX-131.
“We are very encouraged by the progress and early clinical data from our next-generation candidates. While we saw benefits from consolidation dosing with CTX110, we believe CTX112 could result in even better outcomes for patients,” said PK Morrow, CRSP’s chief medical officer said in a Dec. 4 press release.
Editor’s note: The framing of this story has been changed from its original version to more accurately describe the likely potential timeline for future CRISPR approvals. The story has been updated to provide significant additional context regarding CTX-110, including the pipeline update provided by the company earlier this month.