Crispr Therapeutics Falls As Studies Suggest Possible Cancerous Side Effect

Crispr Therapeutics AG CRSP plunged more than 14 percent Monday after Nature Medicine published two studies warning that CRISPR-Cas9 might actually trigger cancer rather than treat targeted diseases.

The research was separately reported by the Karolinska Institute and Novartis AG (ADR) NVS, and Crispr CEO Sam Kulkarni later told STAT News the results were “plausible.”

Why It’s Important

STAT's Sharon Begley called the finding a “potential game-changer” for biotech firms developing CRISPR-reliant therapies, particularly those involving stem cells, cystic fibrosis, combined immunodeficiency and glycogen storage disease.

It also may hinder Crispr as the firm explores new indications.

“It’s something we need to pay attention to, especially as CRISPR expands to more diseases,” Kulkarni said. “We need to do the work and make sure edited cells returned to patients don’t become cancerous.”

The problem — activation of dysfunctional p53 biochemicals meant to repair DNA — might only affect one method of genome-editing, according to STAT.

This means transthyretin amyloidosis candidates by Intellia Therapeutics Inc NTLA and Regeneron Pharmaceuticals Inc REGN should be safe, as well as Editas Medicine Inc EDIT’s blindness therapy and programs for thalassemia and sickle cell disease.

What’s Next

Additional studies may follow, but in the meantime, Novartis advised therapy developers to ensure genome-edited cells have functional p53 before and after editing, and Karolinska recommended similar monitoring.

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