Gene Therapy for Spinal Muscular Atrophy - A Pipeline Analysis Report

Overview of the gene therapy pipeline for spinal muscular atrophy

It has been observed that spinal muscular atrophy is a genetic condition causing weakening of the muscles and resulting in the loss of movement in the affected individual. People affected with the SMA medical condition will suffer a breakdown of nerve cells in the brain and spinal cord, causing the brain to stop sending signals responsible for controlling the muscle movement to the spinal cord. SMA is often caused by the degeneration of the spinal cord motor neurons and the mutations in SMN1 gene. According to the SMA foundation, one in 50 people in the US are SMN1 carriers while one child out of 10,000 is born with SMA in the country. Furthermore, the SMA foundation estimates that the total number of SMA patients in the US is nearly 9,000 and the probability of an infant developing SMA is almost equal between the genders. Also, as per the NIH, the global prevalence of SMA carriers is as high as 1 in 48 people. Consequently, such rising incidences of the SMA health condition is expected to boost the development of gene therapy molecules for spinal muscular atrophy in the upcoming years.

According to this pipeline analysis report, most of the gene therapy molecules in the pipeline are being developed for SMA type 1. 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 gene therapy molecules for the treatment of SMA. 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 gene therapy molecules discontinued by companies.

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

• AveXis
• Genethon
• Voyager Therapeutics

Therapeutic assessment of the gene therapy pipeline for SMA by route of administration

• Intravenous
• Intrathecal

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 gene therapy pipeline for SMA by therapy

• Monotherapy

According to this pipeline analysis report, all the molecules that are currently in the gene therapy pipeline for spinal muscular atrophy 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 spinal muscular atrophy?
• What are the companies that are currently involved in the development of gene therapy molecules for spinal muscular atrophy?
• 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.