Cystic fibrosis transmembrane conductance regulator

ReCode Therapeutics Presents Preclinical Data Using SORT-LNP™ and RNA Platforms to Rescue CFTR Function at the 44th European Cystic Fibrosis Conference (ECFS)

Friday, June 11, 2021 - 1:00pm

These data showcase the potential of our differentiated RNA-based molecular therapies to treat cystic fibrosis.

Key Points: 
  • These data showcase the potential of our differentiated RNA-based molecular therapies to treat cystic fibrosis.
  • By leveraging our LNP platform to deliver genomic medicines, we have demonstrated the ability to restore cystic fibrosis transmembrane conductance regulator (CFTR) function through both tRNA and mRNA approaches, said David Lockhart, Ph.D., CEO & President, ReCode Therapeutics.
  • RCT223 was shown to restore CFTR function for at least 72 hours in patient-derived hBE cells after a single administration.
  • The Companys pipeline includes lead programs for patients with life-limiting genetic respiratory diseases, including cystic fibrosis and primary ciliary dyskinesia.

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

Friday, June 11, 2021 - 10:00am

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

Key Points: 
  • 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.

Vertex Announces U.S. FDA Approval for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in Children With Cystic Fibrosis Ages 6 through 11 With Certain Mutations

Wednesday, June 9, 2021 - 4:04pm

We can now reach approximately 1,500 newly eligible children in the U.S., and we continue to pursue approval for this expanded indication in other countries.

Key Points: 
  • We can now reach approximately 1,500 newly eligible children in the U.S., and we continue to pursue approval for this expanded indication in other countries.
  • The regimen was generally well tolerated, and safety data were similar with those observed in previous studies of patients ages 12 years and older.
  • For more information on TRIKAFTA, patient assistance programs or to find additional eligibility details, visit TRIKAFTA.com , VertexGPS.com or vertextreatments.com .
  • CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene.

SpliSense Secures $28.5 Million in Series B Financing

Thursday, May 13, 2021 - 12:30pm

b'JERUSALEM, May 13, 2021 /PRNewswire/ -- SpliSense , a biopharmaceutical company developing transformative mRNA-altering therapies for cystic fibrosis (CF) and other genetic pulmonary diseases, today announced that it has closed a $28.5 million Series B funding round.

Key Points: 
  • b'JERUSALEM, May 13, 2021 /PRNewswire/ -- SpliSense , a biopharmaceutical company developing transformative mRNA-altering therapies for cystic fibrosis (CF) and other genetic pulmonary diseases, today announced that it has closed a $28.5 million Series B funding round.
  • SpliSense utilizes short, precisely targeted proprietary RNA stretches called ASOs to correct various mutations in the CFTR mRNA.
  • Nissim Darvish, MD, Chairman of the Board, SpliSense, "SpliSense couples Prof. Kerem\'s pioneering research on the genetic underpinnings of CF together with a strong track record in developing ASO therapies and inhaled treatments.
  • SpliSense\' pipeline includes innovative therapies in various development stages, from discovery to IND enabling studies.

Vertex Announces European Commission Approval for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in Combination With Ivacaftor to Treat Cystic Fibrosis Patients 12 Years and Older With At Least One F508del Mutation in the CFTR gene

Wednesday, April 28, 2021 - 12:30pm

CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene.

Key Points: 
  • CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene.
  • Children must inherit two defective CFTR genes \xe2\x80\x94 one from each parent \xe2\x80\x94 to have CF.
  • While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation.
  • Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.\n2 EMA.

Global CFTR Modulators Drug Market Size Forecast Clinical Trials Research Development Report 2026

Thursday, April 22, 2021 - 11:02am

b'"Global CFTR Modulators Market Opportunity, Dosage, Price & Clinical Trials Insight 2026" Report Highlights:\nGlobal CFTR Modulators Market Opportunity: > US$ 20 Billion\nGlobal CFTR Modulators Market Growth: 42% CAGR (2017 -2020)\nGlobal CFTR Modulators Market Growth In 2020: 55%\n"Global CFTR Modulators Market Opportunity, Dosage, Price & Clinical Trials Insight 2026" provides comprehensive insight on clinical and non-clinical factors that are driving the global CFTR Modulators market and its impact on the global pharmaceutical market landscape.

Key Points: 
  • b'"Global CFTR Modulators Market Opportunity, Dosage, Price & Clinical Trials Insight 2026" Report Highlights:\nGlobal CFTR Modulators Market Opportunity: > US$ 20 Billion\nGlobal CFTR Modulators Market Growth: 42% CAGR (2017 -2020)\nGlobal CFTR Modulators Market Growth In 2020: 55%\n"Global CFTR Modulators Market Opportunity, Dosage, Price & Clinical Trials Insight 2026" provides comprehensive insight on clinical and non-clinical factors that are driving the global CFTR Modulators market and its impact on the global pharmaceutical market landscape.
  • Several pharmaceutical companies including Vertex Pharmaceuticals, AbbVie, 4D Molecular Therapeutics and others are currently developing a wide range of CFTR modulators based on different mechanism of action which are present in clinical trials.
  • In coming years, market will see a rapid approval of several CFTR modulator combinations of different mechanism of action which will also propel the growth of market.
  • As per our analysis, the global CFTR modulator market will witness high growth rates during the forecasted period.\n'

Global CFTR Modulators Drug Market Size Forecast Clinical Trials Research Development Report 2026

Thursday, April 22, 2021 - 11:16am

b'"Global CFTR Modulators Market Opportunity, Dosage, Price & Clinical Trials Insight 2026" Report Highlights:\nGlobal CFTR Modulators Market Opportunity: > US$ 20 Billion\nGlobal CFTR Modulators Market Growth: 42% CAGR (2017 -2020)\nGlobal CFTR Modulators Market Growth In 2020: 55%\n"Global CFTR Modulators Market Opportunity, Dosage, Price & Clinical Trials Insight 2026" provides comprehensive insight on clinical and non-clinical factors that are driving the global CFTR Modulators market and its impact on the global pharmaceutical market landscape.

Key Points: 
  • b'"Global CFTR Modulators Market Opportunity, Dosage, Price & Clinical Trials Insight 2026" Report Highlights:\nGlobal CFTR Modulators Market Opportunity: > US$ 20 Billion\nGlobal CFTR Modulators Market Growth: 42% CAGR (2017 -2020)\nGlobal CFTR Modulators Market Growth In 2020: 55%\n"Global CFTR Modulators Market Opportunity, Dosage, Price & Clinical Trials Insight 2026" provides comprehensive insight on clinical and non-clinical factors that are driving the global CFTR Modulators market and its impact on the global pharmaceutical market landscape.
  • Several pharmaceutical companies including Vertex Pharmaceuticals, AbbVie, 4D Molecular Therapeutics and others are currently developing a wide range of CFTR modulators based on different mechanism of action which are present in clinical trials.
  • In coming years, market will see a rapid approval of several CFTR modulator combinations of different mechanism of action which will also propel the growth of market.
  • As per our analysis, the global CFTR modulator market will witness high growth rates during the forecasted period.\n'

Vertex Receives Australian TGA Approval for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to Treat People With Cystic Fibrosis Ages 12 Years and Older Who Have At Least One F508del Mutation

Wednesday, March 24, 2021 - 11:21pm

It is caused by a defective and/or missing CFTR protein resulting from mutations in the CFTR gene.

Key Points: 
  • It is caused by a defective and/or missing CFTR protein resulting from mutations in the CFTR gene.
  • Cystic fibrosis is a complex, progressive, devastating disease that causes severe damage to the lungs, digestive system and other organs in the body.
  • The approval of any new treatment option for people living with cystic fibrosis is always welcome news.
  • In Australia, TRIKAFTA is indicated for the treatment of cystic fibrosis (CF) in patients ages 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Arcturus Therapeutics to Advance ARCT-032, an Aerosolized LUNAR® mRNA-based Therapeutic, as a Development Candidate for Cystic Fibrosis Lung Disease

Wednesday, December 30, 2020 - 12:00pm

We are pleased to have advanced ARCT-032 as a novel mRNA-based development candidate for CF Lung Disease.

Key Points: 
  • We are pleased to have advanced ARCT-032 as a novel mRNA-based development candidate for CF Lung Disease.
  • ARCT-032, based on our proprietary LUNAR technology, is designed to result in the efficient expression of a functional Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein in the lungs.
  • ARCT-032 has the potential to provide benefit to all CF patients, regardless of their underlying genetic mutations.
  • ARCT-032 will utilize Arcturus' LUNAR lipid-mediated aerosolized platform to deliver CFTR messenger RNA to the lungs.

Vertex Announces FDA Approvals of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor), SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) and KALYDECO® (ivacaftor) for Use in People With CF With Certain Rare Mutations

Monday, December 21, 2020 - 7:30pm

The full list of mutations for TRIKAFTA, SYMDEKO and KALYDECO can be found within the updated full Prescribing Information for each respective product.

Key Points: 
  • The full list of mutations for TRIKAFTA, SYMDEKO and KALYDECO can be found within the updated full Prescribing Information for each respective product.
  • In addition, people with CF and their families can search eligibility for Vertex CF medicines through vertextreatments.com .
  • CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene.
  • KALYDECO, SYMDEKO, or TRIKAFTA may affect the way other medicines work, and other medicines may affect how KALYDECO, SYMDEKO, or TRIKAFTA work.