Tay-Sachs disease - Pipeline Insight, 2024

This “Tay-Sachs disease- Pipeline Insight, 2024” report provides comprehensive insights about 4+ 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 devastating genetic disorder that primarily affects the nervous system, resulting from a deficiency of the enzyme beta-hexosaminidase A due to mutations in the HEXA gene on chromosome 15. This enzyme is crucial for breaking down a lipid called GM2 ganglioside in lysosomes. When beta-hexosaminidase A is deficient or absent, GM2 accumulates, particularly in neurons, leading to progressive neurological damage. The disease is autosomal recessive, meaning a child must inherit two defective genes, one from each parent, to develop the condition. Carriers, who have only one defective gene, are typically asymptomatic. Tay-Sachs disease impairs the function of a lysosomal enzyme and involves the buildup of GM2 ganglioside, this condition is sometimes referred to as a lysosomal storage disorder or a GM2-gangliosidosis.

Tay-Sachs disease manifests in three primary forms based on the age of onset: infantile, juvenile, and late-onset. The infantile form is the most severe, with symptoms appearing around six months of age. Affected infants typically exhibit loss of motor skills, increased startle response, seizures, vision and hearing loss, and a distinctive "cherry-red" spot on the retina. These children experience rapid neurological decline and usually do not survive beyond early childhood. Juvenile Tay-Sachs presents later, in early childhood or adolescence, with symptoms such as coordination problems, speech difficulties, and cognitive decline. This form progresses more slowly than the infantile type, but it is still severe and often fatal by early adulthood.

Late-onset Tay-Sachs, also known as LOTS, can appear in adolescence or adulthood and has a much more variable presentation. Symptoms might include muscle weakness, ataxia (loss of full control of bodily movements), psychiatric disorders, and difficulties with speech and swallowing. This form of the disease progresses slowly, and while it can significantly impact quality of life, it is not typically life-threatening, allowing many individuals to have a near-normal life expectancy.

Diagnosis of Tay-Sachs disease involves a combination of clinical examination, enzyme assay, and genetic testing. Clinicians look for characteristic symptoms and the "cherry-red" spot in the eye. Enzyme assays measure the activity of beta-hexosaminidase A in blood or tissue samples, while genetic testing can identify specific mutations in the HEXA gene. Prenatal testing and carrier screening are especially important in high-risk populations, such as Ashkenazi Jews, where carrier rates are higher.

Currently, there is no cure for Tay-Sachs disease, and treatment is focused on managing symptoms and providing supportive care to improve the quality of life. This includes medications to control seizures and psychiatric symptoms, physical therapy to maintain mobility, and nutritional support. Research is ongoing in areas such as enzyme replacement therapy, gene therapy, and substrate reduction therapy, offering hope for more effective treatments in the future. Early diagnosis and comprehensive care are essential for managing the disease and supporting affected families.

"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 Genzyme

Venglustat 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 US for treatment of patients with GM2 gangliosidosis. Currently, the drug is in Phase III stage of its development for the treatment of Tay-Sachs disease.

IB1001: IntraBio

IB1001 is a novel, orally administered therapy developed by IntraBio, a US biopharmaceutical company focused on addressing rare and common neurological diseases. IB1001 is a modified amino acid (N-acetyl-L-leucine) that uses a unique transport mechanism to deliver the drug to target tissues, including across the blood-brain barrier, and results in the normalization of lysosomal and mitochondrial function. This normalization leads to a reduction in neuroinflammation and the normalization of neuronal membrane potential and intracellular ion regulation via calcium channels. Currently, the drug is in Phase II stage of its development for the treatment of Tay-Sachs 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. 4+ 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
• Intravenous
• Subcutaneous
• Parenteral
• Topical
• Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• 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
• IntraBio

Key Products

• Venglustat
• IB1001

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