DelveInsight’s, “Gaucher’s Disease - Pipeline Insight, 2021,” report provides comprehensive insights about 3+ companies and 3+ pipeline drugs in Gaucher’s Disease 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 Understanding
Gaucher’s Disease: Overview
Gaucher disease is a rare, inherited metabolic disorder in which deficiency of the enzyme glucocerebrosidase results in the accumulation of harmful quantities of certain fats (lipids), specifically the glycolipid glucocerebroside, throughout the body especially within the bone marrow, spleen and liver. The symptoms and physical findings associated with Gaucher disease vary greatly from patient to patient. Gaucher disease is categorized as a lysosomal storage disorder (LSD). Lysosomes are the major digestive units in cells. Enzymes within lysosomes break down or "digest" nutrients, including certain complex carbohydrates and fats.

"Gaucher’s Disease - Pipeline Insight, 2021" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Gaucher’s Disease pipeline landscape is provided which includes the disease overview and Gaucher’s Disease treatment guidelines. The assessment part of the report embraces, in depth Gaucher’s Disease 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 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 R&D. The therapies under development are focused on novel approaches to treat/improve Gaucher’s Disease.

Gaucher’s Disease Emerging Drugs Chapters
This segment of the Gaucher’s Disease 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 Emerging Drugs
● Arimoclomol : Orphazyme
Arimoclomol is an investigative medicinal product. Arimoclomol has been shown to increase the production of cell protective HSPs, but only in physiologically stressed cells such as those impacted by disease. This increase in the production of naturally occurring HSPs inside the cells, reduces protein misfolding and aggregation and improves lysosomal function (the cells’ recycling system). The drug is in phase 2 of clinical trials for the treatment of Gaucher’s Disease.

● AVR-RD-02: AVROBIO
AVROBIO’s investigational gene therapy for Gaucher disease is being studied in a Phase 1/2 clinical trial to evaluate the safety and efficacy in individuals with Gaucher disease type 1.
Further product details are provided in the report……..

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

● Major Players in Gaucher’s Disease
There are approx. 3+ key companies which are developing the therapies for Gaucher’s Disease. The companies which have their Gaucher’s Disease drug candidates in the most advanced stage, i.e. phase II include, Orphazyme.

● Phases
DelveInsight’s report covers around 3+ 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 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
• Subcutaneous
• Intravenous
• Intramuscular
● Molecule Type

Products have been categorized under various Molecule types such as
• Bispecific Antibody
• Peptides
• Small molecule
• Gene therapy
● Product Type
Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Gaucher’s Disease: 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 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 drugs.

Gaucher’s Disease Report Insights
• Gaucher’s Disease Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Gaucher’s Disease 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 drugs?
• How many Gaucher’s Disease 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?
• What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Gaucher’s Disease 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 and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players
• Orphazyme
• AVROBIO
• Prevail Therapeutics


Key Products
• Arimoclomol
• AVR-RD-02
• PR 001