Gaucher disease, a rare inherited metabolic disorder linked to GBA1 gene defects, leads to the harmful accumulation of glucocerebroside, impacting organs like the spleen, liver, and nervous system.
According to Sulayman Patel, MSci, an Analyst at GlobalData, “Our research indicates that KOLs agree on the significant potential of gene therapy to replace the current standards of care, enzyme replacement therapy (ERT), and substrate reduction therapy (SRT). However, KOLs express both hope and caution due to the early stage of these therapies.”
Presently approved therapies, such as ERTs and SRTs, aim at addressing systemic issues, leaving neurological symptoms unattended. This particularly affects type 2 and 3 patients, who often experience seizures, cognitive impairment, and vision problems.
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To bridge this gap, companies like Prevail Therapeutics (a subsidiary of Eli Lilly) and Freeline Therapeutics are developing PR-001 and FLT-201, respectively, in Phase I/II stages. These pipeline therapies aim to break down glucocerebroside specifically, offering a targeted, enduring solution.
Patel further states, “However, they underline the need for more safety data, acknowledging the early stages of development.”
Previous successes of gene therapy in treating spinal muscular atrophy and hemophilia patients demonstrate its efficacy and safety, providing compelling evidence for its potential application in Gaucher disease.
In conclusion, Patel emphasizes, “These insights emphasize the potential impact of gene therapy on Gaucher disease. By addressing the root cause and offering long-term solutions, gene therapy poses a formidable challenge to existing treatments, which fail to alleviate neurological issues and create significant financial burdens for payers.”
Source: Medindia
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