As gene therapies ramp up in spinal muscular atrophy and hemophilia, it may soon be the turn of Gaucher disease patients to realize the latent potential of this type of treatment.
That’s according to analysts at GlobalData, which spoke to key opinion leaders (KOLs) during the recent International Gaucher Disease Awareness Month to gauge their thoughts of gene therapy in this space.
GlobalData said in an accompanying report that these KOLs believe pipeline gene therapies “will significantly impact the Gaucher disease landscape,” given that it has the curative potential to provide patients with a healthy GBA1 copy, disrupting the Gaucher drug market.
However, much “groundwork remains to be covered,” the analysts cautioned.
Gaucher disease is a rare inherited metabolic disorder caused by defects in the GBA1 gene and comes with three distinct types of the disease with varying severity.
The rare condition is typically characterized by the accumulation of fatty substances in the body, which can lead to a grossly enlarged spleen and liver. Patients are treated with enzyme replacement therapy (ERT), such as drugs from Sanofi, but patients still have a shortened life span and experience debilitating symptoms.
GlobalData points to Prevail Therapeutics (a subsidiary of Eli Lilly) and Freeline Therapeutics (recently bought out by Syncona) and their early-to-mid-stage therapies PR-001 and FLT-201, respectively. These pipeline therapies aim to facilitate glucocerebroside breakdown, offering what the biotechs promise to be a targeted, long-lasting approach.
Avrobio had last year also seen success in a small trial of its gene therapy AVR-RD-02. The five patients, four with type 1 and one pediatric case with type 3 of the disease, saw stabilization of multiple clinically relevant measures for the lysosomal disorder.
“KOLs have emphasized the promise of gene therapy, identifying these treatments as game-changers for the future of Gaucher disease management,” said Sulayman Patel, MSci, analyst at GlobalData, in a release.
“While in preliminary stages, these offer a paradigm shift from existing treatments, which offer no resolution for neurological manifestations. Indeed, our experts have highlighted the need for more safety data. However, this will be explored as we await further developments.”

https://www.fiercepharma.com/marketing/experimental-gene-therapies-cusp-disrupt-gaucher-disease-drug-market-globaldata