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1032073

Sandhoff disease - Pipeline Insight, 2021

Published: Pre-Order | DelveInsight Business Research LLP | 60 Pages | Delivery time: 2-10 business days

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Sandhoff disease - Pipeline Insight, 2021
Published: Pre-Order
DelveInsight Business Research LLP
Content info: 60 Pages
Delivery time: 2-10 business days
  • ALL
  • Description
  • Table of Contents
Description

DelveInsight's, "Sandhoff disease - Pipeline Insight, 2021," report provides comprehensive insights about 4+ companies and 4+ pipeline drugs in Sandhoff 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

Sandhoff disease Understanding

Sandhoff disease: Overview

Sandhoff disease is a rare lysosomal storage disease. It causes the destruction of nerve cells (neurodegeneration). This leads to problems with thinking and moving. Sandhoff disease is caused by harmful changes in the HEXB gene. Harmful changes in this gene cause decreased amounts of two enzymes in the recycling centers (lysosomes) of the cell. Without these enzymes, certain fats (lipids) build up in large amounts in the nerve cells. This damages the brain and spinal cord (central nervous system). Sandhoff disease is very similar to Tay Sachs disease. Sandhoff disease is caused by harmful mutations in a gene called HEXB. These gene mutations cause decreased amounts of two important enzymes: beta-hexosaminidase A and beta-hexosaminidase B. Sandhoff disease is a rare disorder that is estimated to affect 1 in 1,000,000 individuals.

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

Sandhoff disease Emerging Drugs Chapters

This segment of the Sandhoff 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.

Sandhoff disease Emerging Drugs

IB1001: IntraBio

IB1001, N-acetyl-L-leucine, is an orally administered modified amino acid. In vivo studies have identified N-acetyl-L-leucine to be the active isomer of N-acetyl-DL-leucine and can restore neuronal function and protect against/delay disease progression in multiple neurological circuits of the brain. The mechanism of N-acetyl-L-leucine is known to be multi-modal, including altered glucose and antioxidant metabolism, reduced lysosomal storage, and the reduction of neuroinflammation in the cerebellum, leading to the attenuation of cell death. The company is conducting a multinational, multicenter, open-label, rater-blinded prospective Phase II study which will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease).

AXO-AAV-GM2: Sio Gene Therapies

AXO-AAV-GM2 is a gene therapy that delivers a functional beta-hexosaminidase enzyme through bilateral administration directly to the thalamus and spinal canal of patients using a surgically targeted approach. AXO-AAV-GM2 has the potential to be the first treatment approved for this devastating disease. The U.S. Food & Drug Administration has granted Orphan Drug Designation and Rare Pediatric Disease Designation for AXO-AAV-GM2 for the treatment of Tay-Sachs/Sandhoff disease.

Venglustat: Sanofi

Venglustat is a novel, oral investigational therapy that has the potential to slow the progression of certain diseases by inhibiting abnormal glycosphingolipid (GSL) accumulation. GSLs are cellular building blocks whose abnormal accumulation is implicated in several rare diseases, responsible for both cell dysfunction and disease progression.

Further product details are provided in the report……..

Sandhoff disease: Therapeutic Assessment

This segment of the report provides insights about the different Sandhoff disease drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Sandhoff disease

There are approx. 4+ key companies which are developing the therapies for Sandhoff disease. The companies which have their Sandhoff disease drug candidates in the most advanced stage, i.e. phase II include, IntraBio.

Phases

DelveInsight's 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

Sandhoff 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
  • 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.

Sandhoff disease: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Sandhoff 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 Sandhoff disease drugs.

Sandhoff disease Report Insights

  • Sandhoff disease Pipeline Analysis
  • Therapeutic Assessment
  • Unmet Needs
  • Impact of Drugs

Sandhoff 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 Sandhoff disease drugs?
  • How many Sandhoff disease drugs are developed by each company?
  • How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Sandhoff disease?
  • What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Sandhoff 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 Sandhoff disease and their status?
  • What are the key designations that have been granted to the emerging drugs?

Key Players

  • IntraBio
  • Sio Gene Therapies
  • Sanofi
  • Polaryx Therapeutics
  • Taysha Gene Therapies, Inc.

Key Products

  • IB1001
  • AXO-AAV-GM2: Sio Gene Therapies
  • Venglustat
  • PLX 300
  • TSHA-101
Table of Contents
Product Code: DIPI1399

Table of Contents

Introduction

Executive Summary

Sandhoff disease: Overview

  • Causes
  • Mechanism of Action
  • Signs and Symptoms
  • Diagnosis
  • Disease Management

Pipeline Therapeutics

  • Comparative Analysis

Therapeutic Assessment

  • Assessment by Product Type
  • Assessment by Stage and Product Type
  • Assessment by Route of Administration
  • Assessment by Stage and Route of Administration
  • Assessment by Molecule Type
  • Assessment by Stage and Molecule Type

Sandhoff disease - DelveInsight's Analytical Perspective

Late Stage Products (Phase III)

  • Comparative Analysis

Venglustat: Sanofi

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Mid Stage Products (Phase II)

  • Comparative Analysis

IB1001: IntraBio

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Early Stage Products (Phase I/II)

  • Comparative Analysis

TSHA-101: Taysha Gene Therapies

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Preclinical and Discovery Stage Products

  • Comparative Analysis

PLX 300: Polaryx Therapeutics

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Inactive Products

  • Comparative Analysis

Sandhoff disease Key Companies

Sandhoff disease Key Products

Sandhoff disease- Unmet Needs

Sandhoff disease- Market Drivers and Barriers

Sandhoff disease- Future Perspectives and Conclusion

Sandhoff disease Analyst Views

Sandhoff disease Key Companies

Appendix

List of Tables

  • Table 1 Total Products for Sandhoff disease
  • Table 2 Late Stage Products
  • Table 3 Mid Stage Products
  • Table 4 Early Stage Products
  • Table 5 Pre-clinical & Discovery Stage Products
  • Table 6 Assessment by Product Type
  • Table 7 Assessment by Stage and Product Type
  • Table 8 Assessment by Route of Administration
  • Table 9 Assessment by Stage and Route of Administration
  • Table 10 Assessment by Molecule Type
  • Table 11 Assessment by Stage and Molecule Type
  • Table 12 Inactive Products

List of Figures

  • Figure 1 Total Products for Sandhoff disease
  • Figure 2 Late Stage Products
  • Figure 3 Mid Stage Products
  • Figure 4 Early Stage Products
  • Figure 5 Preclinical and Discovery Stage Products
  • Figure 6 Assessment by Product Type
  • Figure 7 Assessment by Stage and Product Type
  • Figure 8 Assessment by Route of Administration
  • Figure 9 Assessment by Stage and Route of Administration
  • Figure 10 Assessment by Molecule Type
  • Figure 11 Assessment by Stage and Molecule Type
  • Figure 12 Inactive Products