Draft:Cystic Fibrosis: An Overview

Enterprise Therapeutics doses first subjects in Phase I trial for novel cystic fibrosis therapy ETD001

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Monday, June 14, 2021

Biotechnology, Pharmaceutical, Health, Clinical trials, Branches of biology, Medical specialties, Anatomy, Channelopathies, Cystic fibrosis, Epithelial sodium channel, Mucus, Draft:Cystic Fibrosis: An Overview, Bronchiectasis

Enterprise Therapeutics Ltd (Enterprise), a biopharmaceutical company dedicated to the discovery and development of novel therapies to improve the lives of patients suffering from respiratory disease, today announced it has successfully dosed the first subjects in a Phase 1 trial for its novel inhaled cystic fibrosis (CF) therapy, ETD001.

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Enterprise Therapeutics Ltd (Enterprise), a biopharmaceutical company dedicated to the discovery and development of novel therapies to improve the lives of patients suffering from respiratory disease, today announced it has successfully dosed the first subjects in a Phase 1 trial for its novel inhaled cystic fibrosis (CF) therapy, ETD001.
ETD001 is an ENaC ion channel inhibitor with best-in-class potential aimed at treating all people with CF.
Dr John Ford, CEO, Enterprise Therapeutics, said: We are excited to be entering the clinic with ETD001.
ETD001 could significantly improve quality of life for people living with cystic fibrosis and other respiratory diseases linked to mucus obstruction.

SpliSense Presents Preclinical Data for SPL23-ASO, mRNA-Altering Drug Candidate for the Treatment of Cystic Fibrosis, at the European Cystic Fibrosis Conference

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Friday, June 11, 2021

Cystic fibrosis, Medical specialties, Branches of biology, Cystic fibrosis transmembrane conductance regulator, Clinical medicine, Draft:Cystic Fibrosis: An Overview, Congenital absence of the vas deferens

symposium at the 44th European Cystic Fibrosis Digital Conference , June 9-12, 2021.

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symposium at the 44th European Cystic Fibrosis Digital Conference , June 9-12, 2021.
SpliSense utilizes short, precisely targeted proprietary RNA stretches called ASOs to correct various mutations in the CFTR mRNA.
"We are pleased to add another promising preclinical candidate to our pipeline," said Gili Hart, PhD, CEO, SpliSense.
Cystic fibrosis (CF) is a genetic disease that leads to respiratory infections and disabilities and affects over 90,000 people worldwide.

Metagenomi Receives Award From Cystic Fibrosis Foundation to Explore Novel Gene Editing Systems for Therapeutic Applications for Cystic Fibrosis

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Thursday, May 27, 2021

Biotechnology, Other Health, Health, Philanthropy, General Health, Other Philanthropy, Other Science, Research, Genetics, Foundation, Science, Branches of biology, Cystic fibrosis, Medical specialties, Biology, Cystic Fibrosis Foundation, Draft:Cystic Fibrosis: An Overview, Eric Sorscher

Metagenomi , a gene editing company, today announced that it has received an award from the Cystic Fibrosis Foundation to identify and evaluate novel gene editing systems for the development of therapeutics to treat cystic fibrosis.

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Metagenomi , a gene editing company, today announced that it has received an award from the Cystic Fibrosis Foundation to identify and evaluate novel gene editing systems for the development of therapeutics to treat cystic fibrosis.
Metagenomis gene editing capabilities are transformational and could provide opportunities for gene editing-based therapeutics that are not possible with other systems.
We are committed to leveraging our advanced gene editing toolbox for therapeutic applications across disease areas, including cystic fibrosis, and we look forward to collaborating with the Cystic Fibrosis Foundation to broaden therapeutic options for patients.
In cystic fibrosis patients, mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene cause the CFTR protein to become dysfunctional.

Global Cystic Fibrosis Disease Insights and Market Forecasts Report 2020-2026: Sales and Patient Forecasts by Key Therapies - Trikafta, Pulmozyme, Kalydeco, Symdeko, MRT5005, Orkambi & ELX-02 - ResearchAndMarkets.com

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Thursday, May 6, 2021

Health, Other Health, Medical specialties, Cystic fibrosis, Channelopathies, Organ systems, Cystic Fibrosis Trust, Draft:Cystic Fibrosis: An Overview

b'The "Cystic Fibrosis - Disease Insights and Market Forecasts to 2026" report has been added to ResearchAndMarkets.com\'s offering.\n"Cystic Fibrosis (CF): Disease Insights and Market Forecasts to 2026" report delivers an in-depth understanding of the disease, historical & forecasted epidemiology of Cystic Fibrosis (CF) disease in the US, EU5 (Germany, France, Italy, Spain, UK), and Japan and the historical & forecasted market size, for Cystic Fibrosis (CF), drug wise in $ sales and patient numbers at global level and in the US, EU5 (Germany, France, Italy, Spain, UK), Australia and Canada till 2026.\nThis section of the report gives the overview of Cystic Fibrosis (CF) disease indication in detail.

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b'The "Cystic Fibrosis - Disease Insights and Market Forecasts to 2026" report has been added to ResearchAndMarkets.com\'s offering.\n"Cystic Fibrosis (CF): Disease Insights and Market Forecasts to 2026" report delivers an in-depth understanding of the disease, historical & forecasted epidemiology of Cystic Fibrosis (CF) disease in the US, EU5 (Germany, France, Italy, Spain, UK), and Japan and the historical & forecasted market size, for Cystic Fibrosis (CF), drug wise in $ sales and patient numbers at global level and in the US, EU5 (Germany, France, Italy, Spain, UK), Australia and Canada till 2026.\nThis section of the report gives the overview of Cystic Fibrosis (CF) disease indication in detail.
The section highlights the basic definition, causes and symptoms of disease and details the types of Cystic Fibrosis (CF) disease.
Further details on the profile of Cystic Fibrosis (CF) patients, Symptoms experienced by the patients, and disease progression in patients are outlined in the report.
The report further highlights the multiple methods through which the patient can be diagnosed for Cystic Fibrosis (CF) disease.\nThis section of the report provides the overview of Cystic Fibrosis (CF) diagnosed prevalent and treated patients in the US, EU5 and Japan market year wise starting from 2018 till 2026.

Life Edit Therapeutics Announces Award from Cystic Fibrosis Foundation

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Tuesday, March 23, 2021

Life Edit Therapeutics Inc, a next generation gene-editing company, today announced that it has received an award from the Cystic Fibrosis Foundation to identify potential gene editing approaches to treat certain patients with cystic fibrosis (CF).

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Life Edit Therapeutics Inc, a next generation gene-editing company, today announced that it has received an award from the Cystic Fibrosis Foundation to identify potential gene editing approaches to treat certain patients with cystic fibrosis (CF).
This award is part of the Cystic Fibrosis Foundations Path to a Cure initiative that was launched in October 2019 to address and treat the underlying cause of CF.
As part of the $400,000 award from the Foundation, Life Edit will explore its large collection of adenine base editors, or A-base editors, that can potentially be used to correct the six most common, Class I, cystic fibrosis nonsense mutations to restore CFTR function in vivo.
As part of the agreement, Life Edit will benefit from materials, resources, and expertise from the Cystic Fibrosis Foundation.