Novel Drugs are Being Investigated for Gaucher’s Disease Treatment: A Clinical Trial Analysis Review by DelveInsight

Los Angeles, USA, Aug. 05, 2021 (GLOBE NEWSWIRE) — Novel Drugs are Being Investigated for Gaucher’s Disease Treatment: A Clinical Trial Analysis Review by DelveInsight 

Enzyme replacement therapy is an effective treatment for Gaucher Disease, which involves introducing a modified form of the enzyme into the patient by intravenous infusion. The rising requirement for a substantial adequate therapy for the disease is a major driving factor for the extensive research and development.

DelveInsight’s “Gaucher’s Disease Pipeline Insight” report provides comprehensive insights about 15+ companies and 15+ pipeline drugs in the Gaucher’s Disease pipeline landscapes. It comprises Gaucher’s Disease pipeline drug profiles, including clinical and non-clinical stage products. It also includes the Gaucher’s Disease therapeutics assessment by product type, stage, route of administration, and molecule type and further highlights the inactive Gaucher’s Disease pipeline products.     

Some of the key takeaways from the Gaucher’s Disease Pipeline Report  

• Major companies such as Gain therapeutics, AvroBio, Prevail Therapeutics, M6P Therapeutics, Orphazyme, Sanofi, Freeline, Graphite Bio, Cycle Pharma, SmartPharm therapeutics, Coave Therapeutics, Lysogene, Evotech, Generation Bio, Dipharma, CANbridge Life Sciences Ltd. and others are developing potential drug candidates to improve the Gaucher’s Disease treatment scenario. 
• In July 2020, Lysogene announced a research collaboration with the Weizmann Institute of Science to develop a novel AAV gene therapy approach for neuronopathic Gaucher’s disease, Parkinson’s disease, and other diseases associated with mutations of the GBA1 gene.
• Evotec and Centogene have expanded their drug discovery partnership related to the protein target glucocerebrosidase (GBA), focusing on Gaucher’s Disease. The collaboration brings together Evotec’s leading induced pluripotent stem cell (iPSC) platform and broad drug discovery and development capabilities with Centogene global proprietary rare disease platform, including iPSC lines, to address the needs of this orphan drug indication. 
• AVROBIO’s investigational gene therapy for Gaucher’s disease is being studied in a Phase I/II clinical trial to evaluate the safety and efficacy in individuals with Gaucher’s disease type 1.
• CANbridge Life Sciences Ltd has developed CAN103, a recombinant human enzyme replacement therapy (ERT). The company has obtained an exclusive global license from WuXi Biologics.
• FLT201 is a combination of AAVS3 capsid and a liver-specific promoter to drive the expression of GCasevar85. The treatment leverages Freeline’s proprietary high-potency adeno-associated virus capsid (AAVS3). Freeline is expecting to initiate the first-in-human dose-finding studies of FLT201 by late 2021.

Get an overview of pipeline landscape @ Gaucher’s Disease Clinical Trials Analysis 

Gaucher’s Disease is a rare, inherited metabolic disorder in which deficiency of the enzyme glucocerebrosidase leads to the accumulation of harmful quantities of certain fats, specifically the glycolipid glucocerebroside, throughout the body, especially within the bone marrow, spleen, and liver. 

Gaucher’s Disease Emerging Drugs

Venglustat is a small-molecule glucosylceramide synthase (GCS) inhibitor designed to reduce glucosylceramide (GL-1) production and thus is expected to decrease the formation of glucosylceramide-based glycosphingolipids substantially. Because of its effect on glycosphingolipid formation, GCS inhibition has therapeutic potential across many disorders affecting glycosphingolipid metabolism. Therefore, venglustat is developing for substrate reduction therapy in multiple diseases, including Gaucher disease type 3, Parkinson’s disease associated with GBA mutations, Fabry disease, GM2 gangliosidosis, and autosomal dominant polycystic kidney disease. The drug is currently being evaluated in a Phase II/III clinical trial to treat Gaucher’s Disease patients.

Arimoclomol is a small molecule that raises the heat-shock protein 70 (HSP70) levels; a chaperone helps to fold glucocerebrosidase (GBA), and the enzyme, which is faulty in Gaucher Disease patients. By raising the amount of HSP70, arimoclomol aims to increase levels of functional GBA and ease disease symptoms. Due to its small size, arimoclomol is also considered to be able to cross the blood-brain barrier, and for that reason, may be used to treat the neurological symptoms experienced by some Gaucher patients. The dose-finding ORARIGAU-01 Phase II trial investigates the response of 39 people with type 1 or 3 Gaucher to different doses of arimoclomol. 

AVR-RD-02 is an investigational lentiviral gene therapy designed to provide a durable therapeutic benefit for people living with Gaucher disease. The therapy starts with the patient’s hematopoietic stem cells, which are genetically modified to express functional glucocerebrosidase (GCase). Functional GCase reduces glucosylceramide and glucosylsphingosine, the accumulated substances that cause the symptoms of Gaucher disease. AVROBIO is currently evaluating AVR-RD-02 in GuardOne, a Phase I/II clinical trial. AVROBIO receives Orphan Drug Designation from the European Medicines Agency for AVR-RD-02.

For further information, refer to the detailed report @ Gaucher’s Disease Pipeline Therapeutics 

Scope of Gaucher’s Disease Pipeline Drug Insight    

• Coverage: Global 
• Major Players: 15+ Key Players
• Prominent Players: Gain therapeutics, AvroBio, Prevail Therapeutics, M6P Therapeutics, Orphazyme, Sanofi, Freeline, Graphite Bio, Cycle Pharma, SmartPharm therapeutics, Coave Therapeutics, Lysogene, Evotech, Generation Bio, Dipharma, CANbridge Life Sciences Ltd., and many others.   
• Key Drugs Profiles: 15+ Products
• Phases:  

·       Gaucher’s Disease Therapies Late-stage (Phase III)  
·       Gaucher’s Disease Therapies Mid-stage (Phase II)
·       Gaucher’s Disease Therapies Early-stage (Phase I) 
·       Gaucher’s Disease Preclinical stage and Discovery candidates     
·       Discontinued and Inactive candidates  

·       Glucosylceramide synthase inhibitors 
·       Enzyme replacements 
·      Heat shock transcription factor stimulants 
·      Glucosylceramide Replacements
·      Glucosylceramide Stimulants 

·       Gene therapy 
·      Stem cell therapy 
·     Small molecule    

·     Intravenous 
·      Inhalation 
·      Oral
·      Subcutaneous 

·       Monotherapy
·       Combination
·       Mono/Combination 

Key Questions regarding Current Gaucher’s Disease Treatment Landscape and Emerging Therapies Answered in the Pipeline Report  

• What are the current options for Gaucher’s Disease treatment?
• How many companies are developing therapies for the treatment of Gaucher’s Disease? 
• How many are Gaucher’s Disease emerging therapies in the early-stage, mid-stage, and late development stages to treat Gaucher’s Disease? 
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, and significant licensing activities that will impact the Gaucher’s Disease market? 
• Which are the dormant and discontinued products and the reasons for the same?
• What is the unmet need for current therapies for the treatment of Gaucher’s Disease?  
• What are the current novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitation of existing Gaucher’s Disease therapies? 
• What are the critical designations that have been granted for the emerging therapies for Gaucher’s Disease? 
• How many patents are granted and pending for the emerging therapies to treat Gaucher’s Disease?   

Table of Contents

Get a customized pipeline report @ Gaucher’s Disease Drugs Pipeline Report  

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About DelveInsight
DelveInsight is a leading Business Consultant, and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. It also provides Healthcare Consulting services comprising credible market analysis that will help accelerate the business growth and overcome challenges with a practical approach.