Tay-Sachs Disease - Pipeline Insight, 2024

This “Tay-Sachs Disease - Pipeline Insight, 2024,” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Tay-Sachs 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

Tay-Sachs Disease Understanding

Tay-Sachs Disease: Overview

Tay-Sachs disease is a rare, neurodegenerative disorder in which deficiency of an enzyme (hexosaminidase A) results in excessive accumulation of certain fats (lipids) known as gangliosides in the brain and nerve cells. This abnormal accumulation of gangliosides leads to progressive dysfunction of the central nervous system. Tay-Sachs disease is categorized as a lysosomal storage disease. Lysosomes are the major digestive units in cells. Enzymes within lysosomes break down or “digest” nutrients, including certain complex carbohydrates and fats (like glycosphingolipids). When one of these lysosomal enzymes (such as hexosaminidase A) is missing or ineffective, glycosphingolipids start to build up in the lysosome. If there is too much accumulation of these materials in the lysosome, the cells in the nervous system degenerate and die, triggering an inflammatory response that amplifies damage in surrounding tissue.

Tay-Sachs Disease - Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Tay-Sachs Disease pipeline landscape is provided which includes the disease overview and Tay-Sachs Disease treatment guidelines. The assessment part of the report embraces, in depth Tay-Sachs 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, Tay-Sachs 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 Tay-Sachs Disease R&D. The therapies under development are focused on novel approaches to treat/improve Tay-Sachs Disease.

Tay-Sachs Disease Emerging Drugs Chapters

This segment of the Tay-Sachs 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.

Tay-Sachs Disease Emerging Drugs

Venglustat: Sanofi GenzymeVenglustat is a novel, oral investigational therapy that has the potential to slow the progression of certain diseases by inhibiting abnormal GSL accumulation. Venglustat is currently under clinical investigation and its safety and efficacy have not been evaluated by any regulatory authority. The FDA has granted Fast Track designation for the development of venglustat as an investigational therapy for the treatment of Gaucher disease type 3, Fabry disease and GM2 gangliosidosis. Sanofi has been granted Orphan Drug Designation for venglustat in Europe and the U.S. for treatment of patients with Fabry disease, ADPKD, Gaucher disease and GM2 gangliosidosis.

AXO-AAV-GM2: Sio Gene TherapiesAXO-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.

Tay-Sachs Disease: Therapeutic Assessment

This segment of the report provides insights about the different Tay-Sachs Disease drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Tay-Sachs Disease

There are approx. 5+ key companies which are developing the therapies for Tay-Sachs Disease. The companies which have their Tay-Sachs Disease drug candidates in the most advanced stage, i.e. phase III include, Sanofi Genzyme.

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

Tay-Sachs 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.

Tay-Sachs Disease: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Tay-Sachs 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 Tay-Sachs Disease drugs.

Tay-Sachs Disease Report Insights

• Tay-Sachs Disease Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

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

Key Products

• Venglustat
• AXO-AAV-GM2
• IB1001
• TSHA-101
• AX 451
• PLX 200

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