Experts consulted by GlobalData anticipate a significant overhaul in the Gaucher disease scenario because of forthcoming gene therapies in development.
Experts believe gene therapies will disrupt Gaucher disease drug market
Go to source) The gene therapies offer the potential to cure by supplying patients with a healthy GBA1 //copy, disrupting the current Gaucher drug market. However, GlobalData, a prominent data and analytics company, notes that there's much groundwork yet to cover.
Gaucher’s Disease
Gaucher’s disease is a rare genetic disorder. It belongs to a group of diseases called glycolipid storage diseases and is due to the lack of an enzyme called glucocerebrosidase.
Gaucher’s disease is a rare genetic disorder. It belongs to a group of diseases called glycolipid storage diseases and is due to the lack of an enzyme called glucocerebrosidase.
‘Leaders highlight the potential of gene therapy, considering these treatments as transformative for Gaucher disease management. #genetherapy #metabolicdisorder #Gaucher ’
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.
Gene Therapies Set to Upend Gaucher Disease Drug Market
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.
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.
Defective Processes in Removing Cellular Waste Reason Behind Neurodegeneration in Gaucher Disease
A new study reveals that the neurodegeneration caused by Gaucher disease is due to defects in the processes that break down and remove unwanted material from the cells.
A new study reveals that the neurodegeneration caused by Gaucher disease is due to defects in the processes that break down and remove unwanted material from the cells.
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.
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Inborn Errors of Metabolism
Inborn errors of metabolism are genetic disorders that hamper the body’s metabolism resulting in severe clinical manifestations.
Inborn errors of metabolism are genetic disorders that hamper the body’s metabolism resulting in severe clinical manifestations.
Reference:
- Experts believe gene therapies will disrupt Gaucher disease drug market - (https://www.globaldata.com/media/pharma/experts-believe-gene-therapies-will-disrupt-gaucher-disease-drug-market-says-globaldata/)
Test Your Knowledge on Gene Therapy
Gene Therapy - Potential Treatment for Incurable Diseases Gene therapy is a novel procedure in which genetic material is introduced into a patient’s cells to replace a faulty gene in order to try and cure disease. It holds promise in ...
Gene Therapy - Potential Treatment for Incurable Diseases Gene therapy is a novel procedure in which genetic material is introduced into a patient’s cells to replace a faulty gene in order to try and cure disease. It holds promise in ...
