Press release
Gaucher's Disease Type III Pipeline: 7+ Developers Advancing Targeted CNS Therapies and Gene-Based Solutions | DelveInsight
Gaucher's Disease Type III, the chronic neuronopathic form, is gaining targeted attention from over 7 companies, including Sanofi Genzyme, CANbridge Pharmaceuticals, AVROBIO, and Orphazyme. The pipeline features CNS-penetrant enzyme replacement therapies, gene therapies, and small molecules targeting both systemic and neurological manifestations. With limitations in current therapies that address brain involvement, developers are pioneering delivery platforms that cross the blood-brain barrier, signaling a transformative phase for Gaucher's disease type III treatment and long-term management.DelveInsight's "Gaucher's Disease Type III Pipeline Insight, 2025" comprehensively analyzes the current clinical landscape and growth prospects in the Gaucher's disease type III market. The report covers disease insights, treatment guidelines, and a detailed pipeline assessment from preclinical to marketed stages. It includes drug mechanisms, clinical studies, regulatory progress, and key developments such as collaborations, mergers, funding, and designations.
For emerging Gaucher's disease type III drugs, the Gaucher's disease type III pipeline analysis report provides a 360° view of the therapeutic landscape by development stage, product type, route of administration, molecule type, and mechanism of action (MOA). The pipeline research covers business opportunities, challenges, future partnerships, strong competitors, and growth strategies.
Explore the full pipeline analysis for Gaucher's disease type III and uncover key opportunities @ https://www.delveinsight.com/report-store/gauchers-disease-type-iii-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr
Key Takeaways from the Gaucher's Disease Type III Pipeline Report
• DelveInsight's Gaucher's disease type III pipeline analysis depicts a strong space with 7+ active players working to develop 7+ pipeline drugs for Gaucher's disease type III treatment.
• The leading Gaucher's disease type III companies include Sanofi Genzyme, CANbridge Pharmaceuticals, AVROBIO, Orphazyme, KemPharm, Graphite Bio, and others are evaluating their lead assets to improve the Gaucher's disease type III treatment landscape.
• Key Gaucher's disease type III pipeline therapies in various stages of development include Venglustat, CAN103, AVR RD 06, Eliglustat, Arimoclomol, GPH 301, and others.
• In February 2025, Spur Therapeutics announced positive results from its Phase 1/2 GALILEO-1 trial of FLT201, an AAV gene therapy for Gaucher disease type 1. The data showed significant and lasting improvements in glucosylsphingosine (lyso-Gb1) levels, blood counts, organ volume, and bone marrow burden following a single infusion. FLT201 also demonstrated a favorable safety profile. These findings were presented at the 21st Annual WORLDSymposium.
Request a sample and discover the recent breakthroughs happening in the Gaucher's disease type III pipeline landscape @ https://www.delveinsight.com/report-store/gauchers-disease-type-iii-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr
Gaucher's Disease Type III Overview
Gaucher disease type 3 is a subacute neurological form of Gaucher disease that combines progressive brain involvement (encephalopathy) with the systemic features seen in type 1, such as organomegaly, bone abnormalities, and cytopenia. It is a rare lysosomal storage disorder caused by mutations in the GBA gene located on chromosome 1q21, which lead to a deficiency of the enzyme glucocerebrosidase.
This enzyme deficiency results in the buildup of glucosylceramide within cells of the reticuloendothelial system, primarily affecting the liver, spleen, and bone marrow. GD type 3 represents about 5% of all Gaucher disease cases, with an overall annual incidence of 1 in 60,000 and a prevalence of roughly 1 in 100,000. Although no cure exists, enzyme replacement therapy with imiglucerase-approved since 1997-is used to treat non-neurological symptoms and may help slow the progression of neurological decline.
Find out more about Gaucher's disease type III medication @ https://www.delveinsight.com/report-store/gauchers-disease-type-iii-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr
Gaucher's Disease Type III Treatment Analysis: Drug Profile
Venglustat: Sanofi Genzyme
Venglustat (ibiglustat) is an investigational oral therapy developed by Sanofi Genzyme, designed to treat diseases caused by lysosomal dysfunction, including Fabry, Gaucher, and Parkinson's diseases. It works as a glucosylceramide synthase (GCS) inhibitor, blocking the synthesis of glucosylceramide (GL-1) and preventing the accumulation of substrates like Gb3 in diseases where α-galactosidase A is deficient. Venglustat is currently in Phase III clinical trials for Gaucher's disease type III.
CAN103: CANbridge Pharmaceuticals
CAN103, a recombinant human enzyme replacement therapy (ERT), is being developed by CANbridge Pharmaceuticals in collaboration with WuXi Biologics. It targets the long-term treatment of both adult and pediatric patients with Gaucher disease, Types I and III. Currently, it is in Phase I/II clinical trials for Gaucher disease, Type III.
Learn more about the novel and emerging Gaucher's disease type III pipeline therapies @ https://www.delveinsight.com/report-store/gauchers-disease-type-iii-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr
Gaucher's Disease Type III Therapeutics Assessment
By Product Type
• Mono
• Combination
• Mono/Combination.
By Stage
• Late-stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates
By Route of Administration
• Oral
• Parenteral
• Intravenous
• Subcutaneous
• Topical
By Molecule Type
• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Gene therapy
Scope of the Gaucher's Disease Type III Pipeline Report
• Coverage: Global
• Key Gaucher's Disease Type III Companies: Sanofi Genzyme, CANbridge Pharmaceuticals, AVROBIO, Orphazyme, KemPharm, Graphite Bio, and others.
• Key Gaucher's Disease Type III Pipeline Therapies: englustat, CAN103, AVR RD 06, Eliglustat, Arimoclomol, GPH 301, and others.
Dive deep into rich insights for drugs used for Gaucher's disease type III treatment; visit @ https://www.delveinsight.com/report-store/gauchers-disease-type-iii-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr
Table of Contents
1. Introduction
2. Executive Summary
3. Gaucher's Disease Type III Pipeline: Overview
4. Analytical Perspective In-depth Commercial Assessment
5. Gaucher's Disease Type III Pipeline Therapeutics
6. Gaucher's Disease Type III Pipeline: Late-Stage Products (Phase III)
7. Gaucher's Disease Type III Pipeline: Mid-Stage Products (Phase II)
8. Gaucher's Disease Type III Pipeline: Early Stage Products (Phase I)
9. Therapeutic Assessment
10. Inactive Products
11. Company-University Collaborations (Licensing/Partnering) Analysis
12. Key Companies
13. Key Products
14. Unmet Needs
15. Market Drivers and Barriers
16. Future Perspectives and Conclusion
17. Analyst Views
18. Appendix
Contact Us:
Jatin Vimal
jvimal@delveinsight.com
+14699457679
Healthcare Consulting
https://www.delveinsight.com/consulting-services
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. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform, PharmDelve.
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Companies Mentioned are:
Sanofi, Shire, Actelion Pharma, Pfizer, ISU Pharm, ....
Sample/Inquiry at:
https://www.marketinsightsreports.com/reports/0905827183/global-gaucher-disease-market-size-status-and-forecast-2018-2025/inquiry?Source=openpr&mode=47
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Enzyme Replacement Therapy
Substrate Reduction Therapy
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Non-Neuronopathic Gaucher Disease
Neuronopathic Gaucher Disease
Geographically,…