Overview of the drug development for hurler syndrome
Hurler Syndrome is an autosomal recessive disorder, which is caused by the mutation in the iduronidase (IDUA) gene. The two common types of mutation observed in the IDUA gene are W402X and Q70X, which consist of mutant alleles. Hurler syndrome is also referred to as mucopolysaccharidosis type I (MPS I), which happens when the body lacks the alpha-L-iduronidase enzyme, which is needed to break down long chains of sugar molecules. These chains of molecules are called glycosaminoglycans (GAGs). The bodies of people with this syndrome fail to produce an enzyme called Iysosomal alpha-L-iduronidase. As a result, in the absence of this enzyme, GAGs get accumulated significantly, which will damage the organs, including the heart. According to the NIH, approximately 1 in 100,000 newborns has severe Hurler syndrome. Such rising incidences of this syndrome are encouraging market players including Angiochem and ArmaGen are focusing on developing molecules for the treatment of neurological complications of lysosomal disorders, such as Hurler Syndrome and neurodegenerative diseases such as Alzheimer’s disease and Parkinson’s disease, eventually, promoting the drug development for Hurler syndrome.
According to this pipeline analysis report, most of the drug development molecules in the pipeline are being developed for Hurler syndrome. Our market research analysts have also identified that most of these molecules are in the pre-clinical development stage and a considerable number of molecules have been discontinued from development.
Companies covered
This pipeline analysis report provides a detailed analysis of the companies that are involved in the drug development for the treatment of Hurler syndrome. In addition to providing information on the various stages of molecules developed by companies for different indications, this pipeline analysis report also provides information about the drug development molecules discontinued by companies.
Some of the companies covered in this pipeline analysis report are –
- CRISPR THERAPEUTICS
- Eloxx Pharmaceuticals
- INVENTIVA PHARMA
- JCR Pharmaceuticals
- Magenta Therapeutics
Therapeutic assessment of the drug development for hurler syndrome by route of administration
- Intravenous
- Oral
- Parenteral
- Intraperitoneal
- Subcutaneous
The intravenous route of administration (ROA) involves the application of the drug directly into the veins, which will have a more direct effect on the target cells.
Therapeutic assessment of the drug development for hurler syndrome by therapy
- Monotherapy
According to this pipeline analysis report, the majority of the molecules that are currently in the drug development for hurler syndrome are being developed as monotherapy drugs and most of these molecules are in the pre-clinical stage of development.
Key questions answered in the report include
- What are the drug development in the various development stages for hurler syndrome?
- What are the companies that are currently involved in the drug development for hurler syndrome?
- Insight into discontinued/inactive molecules with appropriate reasoning?
- What are the major regulatory authorities approving drugs in various regions?
- Detailed profiling of each active molecule
Technavio also offers customization on reports based on specific client requirement.