Mucopolysaccharidosis III - Pipeline Insight, 2024

This “Mucopolysaccharidosis III - Pipeline Insight, 2024,” report provides comprehensive insights about 15+ companies and 15+ pipeline drugs in Mucopolysaccharidosis III 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

Mucopolysaccharidosis III Understanding

Mucopolysaccharidosis III: Overview

Mucopolysaccharidosis type III (MPS III) is a multisystem lysosomal storage disease characterized by progressive central nervous system degeneration manifest as severe intellectual disability (ID), developmental regression, and other neurologic manifestations including autism spectrum disorder (ASD), behavioral problems, and sleep disturbances. Systemic manifestations can include musculoskeletal problems (joint stiffness, contractures, scoliosis, and hip dysplasia), hearing loss, respiratory tract and sinopulmonary infections, and cardiac disease (valvular thickening, defects in the cardiac conduction system). Children with MPS IIIA do not produce enough enzyme activity to break down a substance in the body called heparan sulfate. The lack of enzyme activity is due to a defective gene called N-sulfoglucosamine sulfohydrolase (SGSH). To diagnose MPS III, mucopolysaccharides are usually first measured in urine, followed by measurement of enzyme activity in blood or a small skin sample. Increased heparan sulfate in urine, and a decrease in the activity of any one of the four enzymes (shown in the table above) is usually consistent with a diagnosis of MPS III and will identify the MPS III type (A, B, C or D). Treatment of Sanfilippo syndrome is symptomatic and supportive.

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

Mucopolysaccharidosis III Emerging Drugs Chapters

This segment of the Mucopolysaccharidosis III 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.

Mucopolysaccharidosis III Emerging Drugs

LYS-SAF302: Lysogene Lysogene’s most advanced gene therapy product candidate is LYS-SAF302, currently the subject of AAVance, a global Phase II/III clinical trial for the treatment of Mucopolysaccharidosis type IIIA (MPS IIIA), or Sanfilippo syndrome type A. LYS-SAF302 delivers a functional copy of the human SGSH gene directly to brain cells using the adeno-associated virus carrier, AAVrh.10, which has a particular tropism for cells of the CNS. In February 2020, LYS-SAF302 received Fast Track designation by the US FDA. The program has received Orphan Drug Designations in the European Union (2014) and the US (2015) for MPS IIIA, and also has Rare Pediatric Disease Designation in theUS.

Tralesinidase alfa: Allievex Corporation Allievex Corporation developing tralesinidase alfa (AX 250), an enzyme replacement therapy, to treat Sanfilippo syndrome type B. Tralesinidase alfa is a fusion of alpha-N-acetylglucosaminidase (NAGLU) with a peptide derived from insulin-like growth factor 2 (IGF-2) was being developed by BioMarin pharmaceutical. Allievex has obtained an exclusive worldwide license for tralesinidase alfa from BioMarin. Tralesinidase alfa was granted orphan drug designation by the U.S. Food and Drug Administration in 2014 and by the European Commission in 2016. The drug is in Phase I/II stage of development for the treatment of MucopolysaccharidosisIII.

Mucopolysaccharidosis III: Therapeutic Assessment

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

Major Players in Mucopolysaccharidosis III

There are approx. 15+ key companies which are developing the therapies for Mucopolysaccharidosis III. The companies which have their Mucopolysaccharidosis III drug candidates in the most advanced stage, i.e. Phase II/III include, Lysogene.

Phases

This report covers around 15+ 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

Mucopolysaccharidosis III 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.

Mucopolysaccharidosis III: Pipeline Development Activities

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

Mucopolysaccharidosis III Report Insights

• Mucopolysaccharidosis III Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

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