SpliSense, a biopharmaceutical company developing transformative mRNA-altering therapies for cystic fibrosis (CF) and other pulmonary diseases, today announced that the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have both granted orphan drug designation (ODD) to SPL84-23-1, an antisense oligonucleotide (ASO) targeting the 3849 mutations in the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) gene associated with a severe form of CF lung disease.

Source: SpliSense

Cystic fibrosis (CF), a single-gene disease with autosomal recessive inheritance, is caused by a mutation in the CF gene aptly named the CFTR protein. The CFTR protein forms a chloride channel critical to efficient mucus transport. Mutations in CFTR disrupt chloride secretion, sodium reabsorption, and water transport, leading to mucus hyper concentration and decreased mucociliary clearance. Dehydrated mucus secretions led to endobronchial infection with a narrow spectrum of distinctive bacteria. They exaggerated inflammatory response, which results in the development of severe bronchiectasis rather than fibrosis and, eventually, respiratory failure. The earliest manifestations of CF are gastrointestinal and nutritional disorders.

• According to the CF Foundation, about ~38,000 Americans, ~5,200 Canadians, and ~27,000 Europeans are lined with CF. The disease is less familiar in other regions of the world.

However, the current Cystic fibrosis treatment market share, market uptake, attribute analysis concerning the most potential emerging therapies (VX-371, MRT5005, Cavosonstat, QBW276, 4D-710, etc..) has been provided under the market outlook section of the study covering 8 MM countries; The United States, EU5 (Germany, Spain, France, Italy, UK) Japan and China.

In terms of pharmacologic therapies, several pharmaceutical products are being approved and under different phases of development for Cystic fibrosis treatment. The key companies in the advanced development stage are Krystal Biotech, Inc., Vertex Pharmaceuticals Incorporated, Translate Bio, Inc., Aridis Pharmaceuticals, Inc., Novartis Pharmaceuticals, etc.

Based on solid domain and business knowledge, Thelansis Knowledge Partners has published the market outlook report on Cystic fibrosis to clearly understand disease area background, epidemiology, current and future competitions, the country-specific standard of care, and the complete market forecast for 2020 to 2030.

Thelansis specializes in pharmaceutical market research and market Insight Report Company, published reports across the therapeutic area, including rare / ultra-rare and mainstream indication. Over the period, we have built a robust repository of 6,000+ Bio-pharma reports that cover Epidemiology studies and Market forecasting based on the KOL opinions.

Competitive intelligence and track of trial results throughout the phases of development executed by a team of a mix of Scientific and Business backgrounds. As an organization, the primary focus is to provide real-world data evidence and market insight to pharmaceutical companies for their decision-making

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