Sandhoff disease - Pipeline Insight, 2024

This “Sandhoff disease - Pipeline Insight, 2024,” report provides comprehensive insights about 5+ companies and 5+ 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, 2024 report 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 SandhoffDisease).

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.Currently, the drug is in Phase III stage of Clinical trial evaluation for the treatment of SandhoffDisease.

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. 5+ 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 III include,Sanofi .

Phases

This report covers around 5+ 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
• Sanofi
• Polaryx Therapeutics
• Azafaros

Key Products

• IB1001
• Venglustat
• PLX 300
• PLX-200
• AZ 3102