Leber's Hereditary Optic Neuropathy (LHON) - A Pipeline Analysis Report

This pipeline analysis report provides detailed insights into the clinical trials landscape of the drug development for leber’s hereditary optic neuropathy (LHON) including molecules at pre-clinical and discovery stage. The report also offers comprehensive information about the therapeutic assessment of the pipeline molecules based on various segmentations such as therapy, route of administration, and target.

Overview of the drug development for leber’s hereditary optic neuropathy (LHON)

LHON is the most common mitochondrially inherited disease, which is associated with mitochondrial base pair mutations. The base pairs of mitochondria, which get affected due to LHON are G11778A, T14484C, and G3460A. The occurrence of mutations in these pairs affect respiratory chain complex I genes, mitochondrial genes ND1, ND4, and ND6, and others. In individuals suffering from this disease, the vision gets impacted due to degeneration of retinal ganglion cells and their axons, which leads to an acute or subacute loss of central vision of both eyes. The disease mainly impacts the central vision, thus causing a problem in reading, writing, and recognizing whereas peripheral vision remains unimpacted. LHON is one of the most common optic neuropathies found in young adult males. According to the US National Library of Medicines, the prevalence of LHON in most populations is unknown. It affects 1 in 30,000-50,000 people in Northeast England and Finland. As a result, the rising incidences of LHON is expected to boost the drug development for LHON in the next few years.

According to this pipeline analysis report, most of the drug molecules in the pipeline are being developed for LHON. 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 molecules for the treatment of LHON. 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 molecules discontinued by companies.

Some of the companies covered in this pipeline analysis report are:

• Alkeus Pharmaceuticals
• Biovista
• Fortify
• Khondrion
• Spark Therapeutics

Therapeutic assessment of the drug development for LHON by route of administration

• Ophthalmic
• Intravenous
• Intravitreal
• Oral

The ophthalmic route of administration (ROA) involves the application of the drug directly into the external eyes, which will have a more direct effect on the target cells.

Therapeutic assessment of the drug development for LHON by therapy

• Monotherapy

According to this pipeline analysis report, all the molecules that are currently in the gene therapy pipeline for leber’s hereditary optic neuropathy (LHON) 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 gene therapy molecules in the various development stages for leber’s hereditary optic neuropathy (LHON)?
• What are the companies that are currently involved in the development of gene therapy molecules for leber’s hereditary optic neuropathy (LHON)?
• 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.