Gene Therapy for Sickle-Cell Diseases - A Pipeline Analysis Report

Overview of the gene therapy for sickle-cell diseases

Sickle-cell diseases are a group of blood disorders caused by an abnormality in the hemoglobin in red blood cells. Sickle-cell diseases are mostly inherited from parents. The patients inherit one copy of the mutated hemoglobin gene from each parent in the chromosome 11. The people with one abnormal copy of the hemoglobin gene are called carriers, and they do not show the traits of the disease or develop the anemia. Sickle-cell diseases can lead to severe complications such as stroke, chronic pain, abortion, pulmonary hypertension, and osteomyelitis. The diseases are race- and geography-specific indications. The disease is more common in the Sub-Saharan regions, India, and the Middle-Eastern countries compared with other parts of the world. According to the National Library of Medicine, a part of the National Institutes of Health, sickle-cell diseases are more prevalent in African-Americans than in the Hispanic Americans, with a ratio of 3:1. The ratio of males to females with sickle-cell diseases is 1:1. As a result, with the rising incidences of sickle-cell diseases, the gene therapy for sickle-cell diseases is expected to increase considerably during the next few years.

According to this pipeline analysis report, most of the gene therapy molecules in the pipeline are being developed for sickle-cell diseases. 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 sickle-cell diseases. 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:

• Angiocrine Bioscience
• Calimmune
• Editas Medicine
• Genethon
• MaxCyte

Therapeutic assessment of the gene therapy for sickle-cell diseases by route of administration

• Intravenous

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

Therapeutic assessment of the gene therapy for sickle-cell diseases by therapy

• Monotherapy

According to this pipeline analysis report, all the molecules that are currently in the gene therapy pipeline for sickle-cell diseases 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 sickle-cell diseases?
• What are the companies that are currently involved in the development of gene therapy molecules for sickle-cell diseases?
• 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.