Gaucher's Disease Type III - Pipeline Insight, 2024

This “Gaucher’s disease type III - Pipeline Insight, 2024,” report provides comprehensive insights about 7+ companies and 7+ pipeline drugs in Gaucher’s disease type III pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Geography Covered

• Global coverage

Gaucher’s disease type III Understanding

Gaucher’s disease type III: Overview

Gaucher disease type 3 is the subacute neurological form of Gaucher disease characterized by progressive encephalopathy and associated with the systemic manifestations (organomegaly, bone involvement, cytopenia) of GD type 1. The annual incidence of GD is about 1/60,000 and the prevalence is approximately 1/100,000. GD type 3 accounts for 5% of all patients with GD. GD type 3 is a lysosomal storage disease caused by a mutation in the GBA gene (1q21) that codes for the lysosomal enzyme, glucocerebrosidase. The deficiency in glucocerebrosidase leads to the accumulation of glucosylceramidase (or beta-glucocerebrosidase) deposits in the cells of the reticuloendothelial system of the liver, spleen and the bone marrow (Gaucher cells). The treatment for patients with GD type 3 exhibiting clinically significant non neurological manifestations is enzyme substitution therapy (imiglucerase with marketing authorization (MA) since 1997). It appears to slow progression of the neurological symptoms and is effective against the systemic manifestations.

Gaucher’s disease type III - Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Gaucher’s disease type III pipeline landscape is provided which includes the disease overview and Gaucher’s disease type III treatment guidelines. The assessment part of the report embraces, in depth Gaucher’s disease type III commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Gaucher’s disease type III collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Gaucher’s disease type III R&D. The therapies under development are focused on novel approaches to treat/improve Gaucher’s disease type III.

Gaucher’s disease type III Emerging Drugs Chapters

This segment of the Gaucher’s disease type III report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Gaucher’s disease type III Emerging Drugs

Venglustat: Sanofi Genzyme Venglustat (ibiglustat) is an investigational therapy being developed by Sanofi Genzyme for the treatment of conditions caused by lysosomal dysfunction such as Fabry, Gaucher, and Parkinson’s diseases. Venglustat is an oral inhibitor of an enzyme called glucosylceramide synthase (GCS). Enzymes modify specific molecules called substrates. GCS turns its substrate, ceramide, into glucosylceramide (GL-1) during lipid metabolism, a series of biochemical reactions that degrade and generate lipids. GL-1 acts as a substrate to other enzymes and is turned into globosides, a subclass of lipids where Gb3 belongs. When venglustat inhibits GCS, it prevents the synthesis of GL-1, thereby reducing the substrate of the following reactions that lead to the formation of Gb3 and its accumulation in the absence of a-galactosidase A. That’s why venglustat is called a substrate reduction therapy. Currently, it is in Phase III stage of clinical trial evaluation to treat Gaucher's disease typeIII.

CAN103: CANbridge Pharmaceuticals CAN103 is a recombinant human enzyme replacement therapy (ERT). It is being developed by CANbridge Pharmaceuticals as part of its rare disease partnership with WuXi Biologics (2269.HK) for the long-term treatment of adults and children with Gaucher disease, Types I and III.Currently, the drug is in Phase I/II stage of Clinical trial evaluation for the treatment of Gaucher Disease, Type III.

Gaucher’s disease type III: Therapeutic Assessment

This segment of the report provides insights about the different Gaucher’s disease type III drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Gaucher’s disease type III

There are approx. 7+ key companies which are developing the therapies for Gaucher’s disease type III. The companies which have their Gaucher’s disease type III drug candidates in the most advanced stage, i.e. phase III include, Sanofi Genzyme.

Phases

This report covers around 7+ products under different phases of clinical development like

• 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

Route of Administration

Gaucher’s disease type III pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Oral
• Parenteral
• intravenous
• Subcutaneous
• Topical.

Molecule Type

Products have been categorized under various Molecule types such as

• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Gene therapy

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Gaucher’s disease type III: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Gaucher’s disease type III therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Gaucher’s disease type III drugs.

Gaucher’s disease type III Report Insights

• Gaucher’s disease type III Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Gaucher’s disease type III Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Gaucher’s disease type III drugs?
• How many Gaucher’s disease type III drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Gaucher’s disease type III?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Gaucher’s disease type III therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Gaucher’s disease type III and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Sanofi Genzyme
• CANbridge Pharmaceuticals
• AVROBIO
• Orphazyme
• KemPharm
• Graphite Bio

Key Products

• Venglustat
• CAN103
• AVR RD 06
• Eliglustat
• Arimoclomol
• GPH 301

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