Gene Therapy for Cystic Fibrosis - A Pipeline Analysis Report

Overview of the drug development pipeline for cystic fibrosis

It has been observed that cystic fibrosis is a genetic defect characterized by the buildup of thick, sticky mucus that can damage the lungs. It may also affect other organs such as liver, intestines, pancreas, and kidneys. This thick mucus blocks the airways and damages the lungs, which makes it more prone to bacterial infection. These bacteria multiply and lead to a serious chronic problem. Cystic fibrosis generally occurs due to a defect in both the copies of the gene for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. The common symptoms of cystic fibrosis are a cough producing mucus, wheezing, breathlessness, exercise intolerance, repeated lung infections, and an inflamed nasal passage or stuffy nose. Monotherapy is emerging as the most efficient approach for the treatment of cystic fibrosis.

According to this pipeline analysis report, all the drug molecules in the pipeline are being developed for cystic fibrosis. 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.

Pipeline analysis report on drug development for cystic fibrosis: 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 cystic fibrosis. 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:

• 4D Molecular Therapeutics
• CRISPR Therapeutics
• Editas Medicine

Therapeutic assessment of the drug development pipeline for cystic fibrosis by route of administration

• Intravenous
• Aerosol inhalation
• Parenteral
• Implant

The intravenous route of administration (ROA) involves the administration of the drug through the veins, which will have a more direct effect on the target cells. In the aerosol inhalation technique, the drug molecules are administrated through air particles delivered by a device that is inhaled and absorbed into the patient's body through lungs.

Therapeutic assessment of the drug development pipeline for cystic fibrosis by therapy

• Monotherapy

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