Alport Syndrome - A Pipeline Analysis Report

Overview of the drug development pipeline for Alport Syndrome

It has been observed that Alport syndrome is a genetic condition caused by gene mutations to a protein called collagen. This condition damages the tiny blood vessels in kidneys, which further leads to various diseases in kidneys and even kidney failure. It can also lead to hearing and vision loss. Various symptoms related to the Alport syndrome include hearing loss, leiomyomatosis, eye abnormalities, hematuria and proteinuria, and high blood pressure. According to the National Center for Biotechnology Information (NCBI), 1 out of 50,000 newborns is affected by the Alport syndrome. Moreover, often males are more vulnerable to Alport syndrome than females. Consequently, Technavio’s market research analysts have estimated that with rising incidences of Alport Syndrome, the need for drug development for Alport syndrome has increased considerably in the recent years.

According to this pipeline analysis report, most of the gene therapy molecules in the pipeline are being developed for treatment for Alport 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 development of drug development molecules for the treatment of Alport 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:

• DiaMedica Therapeutics
• Ember Therapeutics
• Forsee Pharmaceuticals
• Reata Pharmaceuticals
• Regulus Therapeutics
• Variant Pharmaceuticals

Therapeutic assessment of the drug development pipeline for Alport Syndrome by route of administration

• Oral
• Subcutaneous

The oral route of administration (ROA) involves the application of the drug directly through the mouth cavity, which will have a more direct effect on the target cells.

Therapeutic assessment of the drug development pipeline for Alport Syndrome by therapy

• Monotherapy

According to this pipeline analysis report, all the molecules that are currently in the drug development for Alport 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 molecules in the various development stages for Alport syndrome?
• What are the companies that are currently involved in the development of drug development for Alport 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.