Gaucher's disease type I - Pipeline Insight, 2024

This “Gaucher’s disease type I - Pipeline Insight, 2024,” report provides comprehensive insights about 4+ companies and 4+ pipeline drugs in Gaucher’s disease type I 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 I Understanding

Gaucher’s disease type I: Overview

Gaucher disease (GD) type 1 is the most prevalent form of the disease in western countries, making up about 95 percent of cases there. GD type 1 is a lysosomal storage disease caused by a mutation in the GBA gene (localized to 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, of the spleen and the bone marrow (Gaucher cells). Diagnostic methods involve ultrasound and magnetic resonance imaging (MRI) for the initial evaluation and subsequent monitoring of hepatosplenomegaly, radiography and bone scintigraphy to detect bone lesions and complications, osteodensitometry for the evaluation of osteopenia of the lumbar spine and femoral neck, and cardiac ultrasound for the detection of pulmonary arterial hypertension. The standard treatment for GD type 1 is enzyme substitution therapy, administered intravenously (e.g.: imiglucerase with European marketing authorization (MA) since 1997 and velaglucerase with a MA since 2010). Substrate reduction therapy (miglustat), administered orally, provides an alternative second-line treatment when enzyme substitution therapy is not suitable. Bisphophonates can also be proposed to prevent bone complications.

Gaucher’s disease type I - 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 I pipeline landscape is provided which includes the disease overview and Gaucher’s disease type I treatment guidelines. The assessment part of the report embraces, in depth Gaucher’s disease type I 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 I 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 I R&D. The therapies under development are focused on novel approaches to treat/improve Gaucher’s disease type I.

Gaucher’s disease type I Emerging Drugs Chapters

This segment of the Gaucher’s disease type I 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 I Emerging Drugs

GPH301: Graphite Bio GPH301 is a next generation gene-edited autologous HSC product candidate leveraging the CCR5 locus technology for the treatment of Gaucher disease. With GPH301, a functional copy of the gene for glucocerebrosidase (GCase) is inserted into the chromosomal location of the CCR5 gene. This locus is known as a safe harbor both due to the lack of serious deleterious effects in humans with CCR5 mutations and because the expression of genes inserted there can be precisely controlled by regulatory elements inserted together with the gene of interest. Graphite Bio intends to develop GPH301 for the treatment of both Type 1 and Type 3 Gaucher disease. In Type 1 disease, Graphite Bio will explore targeted conditioning regimens. This same approach can be used for production of therapeutic proteins for other diseases including other lysosomal storage diseases. Proof of concept in Gaucher may accelerate development of a pipeline of CCR5 safe harbor protein productioncandidates.

CAN103: CANbridge Pharmaceuticals CAN103 is a recombinant human enzyme replacement therapy (ERT). CANbridge Pharmaceuticals recently announced that the Investigational New Drug (IND) application for CAN103 has been accepted by the Chinese National Medical Products Administration (NMPA). CAN103 is an enzyme replacement therapy (ERT) being developed by CANbridge 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.

AVR-RD-02: AVROBIOAVROBIO’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. The trial is now enrolling in Canada, Australia and the U.S. The trial is intended to recruit 8 to 16 individuals between the ages of 16 and 35 with Gaucher disease type 1, including both those who are treatment-naïve and those who are stable on enzyme replacement therapy.

Gaucher’s disease type I: Therapeutic Assessment

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

Major Players in Gaucher’s disease type I

There are approx. 4+ key companies which are developing the therapies for Gaucher’s disease type I. The companies which have their Gaucher’s disease type I drug candidates in the most advanced stage, i.e. phase I/II include, AVROBIO.

Phases

This report covers around 4+ 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 I 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 I: 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 I 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 I drugs.

Gaucher’s disease type I Report Insights

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

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

Key Players

• Graphite Bio
• CANbridge Pharmaceuticals
• Freeline Therapeutics
• ILIAS Biologics
• AVROBIO, Inc.

Key Products

• GPH301
• CAN103
• FLT 201
• Exosome-based Gaucher's disease Type-I and Gaucher's disease Type-III enzyme therapeutic
• AVR-RD-02

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