Cystic Fibrosis - Drug Pipeline Landscape, 2023

Cystic Fibrosis - Drug Pipeline Landscape, 2023

Cystic Fibrosis (CF) is a progressive, genetic disease that causes persistent lung infections and limits the ability to breathe over time. In people with CF, a defective gene causes a thick, sticky buildup of mucus in the lungs, pancreas, and other organs. In the lungs, the mucus clogs the airways and traps bacteria leading to infections, extensive lung damage, and eventually, respiratory failure. In the pancreas, the mucus prevents the release of digestive enzymes that the body needs to break down food and absorb vital nutrients.

In cystic fibrosis, a defect (mutation) in CFTR gene, changes a protein that regulates the movement of salt in and out of cells. Many different defects can occur in the gene. The type of gene mutation is associated with the severity of the condition.

Symptoms of Cystic Fibrosis include trouble with bowel movements or frequent, greasy stools, wheezing or trouble breathing, frequent lung infections, Infertility.

Healthcare providers diagnose cystic fibrosis with by using different tests including newborn screening, sweat test, genetic tests, chest X-rays, sinus X-rays, lung function tests and sputum culture.

Treatment of cystic fibrosis include medications, medications targeting genes, vest therapy, pulmonary rehabilitation, and surgical procedures.

Major pharmaceutical companies are involved in the development of potential drug candidates to improve the Cystic Fibrosis treatment such as VX-121/TEZ/D-IVA, VX-561, 4D-710, AP-PA02, BX004-A and others. Key players involved in the development of therapies to treat cystic fibrosis are Vertex Pharmaceuticals Incorporated, 4D Molecular Therapeutics, Armata Pharmaceuticals Inc, BiomX Inc, Krystal Biotech Inc and others. Two drugs are under late-stage Phase III clinical trials and 20+ drugs are in Phase II clinical trials and some other drugs are under Phase I, IND/CTA Filed, preclinical and discovery stages of development.

Report Highlights

Global Insight Service's, Cystic Fibrosis - Drug Pipeline Landscape, 2023 report provides an overview of the Cystic Fibrosis pipeline drugs. This report covers detailed insights on Cystic Fibrosis drugs under development, assessment by target, mechanism of action, route of administration and molecule type. Product pipeline by companies, stage of development and key regulatory designations, deals and milestones have been presented to provide insights and thus help industry participants in their decision making. Cystic Fibrosis pipeline report helps gain insights on drugs which are under development stage of drug development process across globally.

Methodology

The research process includes extensive secondary research on public domain and other authentic sources to add or update the pipeline products information. The secondary research sources include, but are not limited to company websites, annual reports, financial reports, company pipeline chart, investor presentations and SEC filings, journals and conferences, and clinical trials registries.

Scope

The pipeline landscape report provides analysis of pipeline products based on several stages of development ranging from Discovery to Pre-Registration. The report provides a review of pipeline therapeutics by companies based on information derived from company and industry-specific sources. The pipeline report covers assessment of therapeutics by mechanism of action (MoA), drug target, route of administration (RoA) and molecule type. Comprehensive profiles of the pipeline products with details such as company overview, development stage; molecule type, target, mechanism of action, route of administration, dosage form, regulatory designations, key deals, clincial trials, and key upcoming milestones are included.

Reasons to Buy
Helps to find and recognize significant therapeutics under development. Thorough understanding of pipeline structure and helps in developing corrective measures for pipeline projects.
Effective R&D strategies can be developed with deep knowledge of competitor information, analysis, and insights.
Plan collaborations with various industry partners that have role in some or the other stage of drug development such as contract manufacturing, co-development, contract research organization and commercialization etc.
Helps to create in-licensing and out-licensing opportunities by identifying prospective partners with attractive projects to expand business potential and scope.

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