KOLs believe gene therapies will disrupt Gaucher disease drug market

Following the conclusion of International Gaucher Disease Awareness Month, key opinion leaders (KOLs) interviewed believe pipeline gene therapies will significantly impact the Gaucher disease landscape.

Gene therapy has the curative potential to provide patients with a healthy GBA1 copy, disrupting the Gaucher drug market. However, much groundwork remains to be covered, says pharma analytics firm GlobalData.

Gaucher disease, a rare inherited metabolic disorder caused by defects in the GBA1 gene, results in harmful glucocerebroside accumulation, which may affect the spleen, liver, and nervous system.