Cystic fibrosis transmembrane conductance regulator

Cystic Fibrosis Foundation Announces Investment in SalioGen Therapeutics to Fund Genetic Therapies Research

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Wednesday, January 5, 2022
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The Cystic Fibrosis Foundation announced today that it has invested in SalioGen Therapeutics to support the companys preclinical research into a novel genetic therapy for cystic fibrosis.

Key Points: 
  • The Cystic Fibrosis Foundation announced today that it has invested in SalioGen Therapeutics to support the companys preclinical research into a novel genetic therapy for cystic fibrosis.
  • Gene therapy involves inserting a new version of the CFTR DNA into the cells of people with cystic fibrosis.
  • The Cystic Fibrosis Foundation is the world's leader in the search for a cure for cystic fibrosis.
  • The Foundation funds more CF research than any other organization, and nearly every CF drug available today was made possible because of Foundation support.

Verona Pharma Completes Enrollment in ENHANCE-1 48-week Subset

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Wednesday, January 5, 2022
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LONDON and RALEIGH, N.C., Jan. 05, 2022 (GLOBE NEWSWIRE) -- Verona Pharma plc (Nasdaq: VRNA) (“Verona Pharma” or the “Company”), a clinical-stage biopharmaceutical company focused on respiratory diseases, announces enrollment completed in the 48-week subset of the ENHANCE-1 trial in December 2021. The ENHANCE-2 trial has completed screening with 788 subjects randomized as of January 4, 2022, and full enrollment is expected by the end of January 2022. The Phase 3 ENHANCE (“Ensifentrine as a Novel inHAled Nebulized COPD thErapy”) program is evaluating nebulized ensifentrine for the maintenance treatment of chronic obstructive pulmonary disease (“COPD”).

Key Points: 
  • Ensifentrine also activates the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR), which is beneficial in reducing mucous viscosity and improving mucociliary clearance.
  • Verona Pharma is a clinical-stage biopharmaceutical company focused on developing and commercializing innovative therapies for the treatment of respiratory diseases with significant unmet medical needs.
  • Verona Pharma is closely monitoring the potential impact of the COVID-19 pandemic on its operations and clinical trials, in particular the timelines and costs of its Phase 3 ENHANCE clinical program.
  • These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this press release.

SpliSense Announces FDA and EMA Grant Orphan Drug Designation to SPL84-23-1 for the Treatment of Cystic Fibrosis

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Tuesday, January 4, 2022
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"The orphan designations we received for our lead drug candidate both from the FDA and EMA are important milestones toward addressing the urgent, unmet needs of patients living with cystic fibrosis," said Gili Hart, PhD, CEO, SpliSense.

Key Points: 
  • "The orphan designations we received for our lead drug candidate both from the FDA and EMA are important milestones toward addressing the urgent, unmet needs of patients living with cystic fibrosis," said Gili Hart, PhD, CEO, SpliSense.
  • SpliSense utilizes short, precisely targeted proprietary RNA stretches called ASOs to correct various mutations in the CFTR mRNA.
  • Cystic fibrosis (CF) is a genetic disease that leads to respiratory infections and disabilities and affects over 90,000 people worldwide.
  • The Initial of focus SpliSense is on high unmet need, orphan indications (CF) with subsequent expansion to larger, non-orphan pulmonary indications (muco- obstructive diseases).

Vertex Announces Reimbursement Agreement in Spain for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in Combination With Ivacaftor to Treat People With Cystic Fibrosis 12 Years and Older With At Least One F508del Mutation in the CFTR Gene

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Friday, November 19, 2021
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The agreement covers people with CF ages 12 years and older who have at least one copy of the F508del mutation, regardless of the other mutation type in the CFTR gene.

Key Points: 
  • The agreement covers people with CF ages 12 years and older who have at least one copy of the F508del mutation, regardless of the other mutation type in the CFTR gene.
  • The formalized agreement is an important milestone for people living with cystic fibrosis in Spain.
  • We are pleased that the Ministry of Health recognized the value of KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor.
  • CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene.

Global Trikafta Patent, Sales & Clinical Trials Insight to 2028 - Featuring Abbvie, GlaxoSmithKline and Roche Among Others - ResearchAndMarkets.com

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Wednesday, November 10, 2021
Health, Biotechnology, Safety, Government, Disease, Investor, Cystic fibrosis transmembrane conductance regulator, Growth, Mutation, Research, Genome, Data, CFTR, Cystic fibrosis, Vertex Pharmaceuticals, Patent, Patient, Prevalence, Pharmaceutical industry

Trikafta Patent, Sales & Clinical Trials Insight 2028 Report Analysis & Data Highlights:

Key Points: 
  • Trikafta Patent, Sales & Clinical Trials Insight 2028 Report Analysis & Data Highlights:
    The successful mapping of human genome and advances in molecular technology has led to evolution of targeted therapies.
  • Researchers have developed CFTR modulator agents which are small molecules which modulate the function of abnormal CFTR protein.
  • To date, four CFTR modulators have been approved for the treatment of cystic fibrosis.
  • The novel drug Trikafta was designed to increase the quantity and function of the F508del-CFTR protein at the cell surface.

ReCode Therapeutics Presents Preclinical Data from mRNA-based Cystic Fibrosis Program at 2021 North American Cystic Fibrosis Conference

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Wednesday, November 3, 2021
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ReCode Therapeutics , a biopharmaceutical company pioneering disease-modifying genetic medicines using its proprietary LNP delivery platform, presented new preclinical data today from the Companys inhaled mRNA-based therapeutic program for cystic fibrosis (CF) during an oral poster session at the 2021 North American Cystic Fibrosis Conference (NACFC) .

Key Points: 
  • ReCode Therapeutics , a biopharmaceutical company pioneering disease-modifying genetic medicines using its proprietary LNP delivery platform, presented new preclinical data today from the Companys inhaled mRNA-based therapeutic program for cystic fibrosis (CF) during an oral poster session at the 2021 North American Cystic Fibrosis Conference (NACFC) .
  • Todays data support the continued development of an inhaled mRNA therapy for the treatment of CF, and we look forward to advancing this program toward an IND submission.
  • ReCode Therapeutics is an integrated genetic medicines company developing disease-modifying therapeutics using its powerful LNP delivery technology to target organs and tissues beyond the liver.
  • The Companys pipeline includes lead programs for patients with life-limiting genetic respiratory diseases, including cystic fibrosis and primary ciliary dyskinesia.

Tessera Therapeutics Announces R&D Collaboration with Cystic Fibrosis Foundation as Part of a Portfolio of Complementary Technologies Advanced by Flagship’s Pioneering Medicines to Treat Cystic Fibrosis

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Wednesday, November 3, 2021
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Tessera Therapeutics today announced a treatment-focused collaboration with the Cystic Fibrosis Foundation using Tesseras Gene Writing technology to rewrite the genetic codes that cause cystic fibrosis.

Key Points: 
  • Tessera Therapeutics today announced a treatment-focused collaboration with the Cystic Fibrosis Foundation using Tesseras Gene Writing technology to rewrite the genetic codes that cause cystic fibrosis.
  • The Gene Writers are part of a portfolio of technologies that Pioneering Medicines has assembled to treat cystic fibrosis as part of a broader collaboration with the Cystic Fibrosis Foundation.
  • Were excited to work with the Cystic Fibrosis Foundation, the worlds leader in supporting breakthrough technologies for cystic fibrosis, said Geoffrey von Maltzahn, Ph.D, Co-Founder and CEO of Tessera, and General Partner at Flagship Pioneering.
  • The Cystic Fibrosis Foundations extensive network of scientists, companies, and clinicians knowledgeable about cystic fibrosis will help accelerate screening and development of our cystic fibrosis-targeting Gene Writers.

Calithera Presents Interim Data from Phase 1b Trial of Arginase Inhibitor CB-280 in Cystic Fibrosis at NACFC 2021

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Monday, November 1, 2021
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SOUTH SAN FRANCISCO, Calif., Nov. 01, 2021 (GLOBE NEWSWIRE) -- Calithera Biosciences, Inc. (Nasdaq: CALA), a clinical-stage biopharmaceutical company, today shared interim safety and efficacy results from a Phase 1b, randomized, double-blind, placebo-controlled, dose-escalation trial evaluating CB-280, the company’s investigational arginase inhibitor, in adults with cystic fibrosis (CF). The data were shared in a poster presentation at the North American Cystic Fibrosis Foundation Conference (NACFC; Abstract 529).

Key Points: 
  • The data were shared in a poster presentation at the North American Cystic Fibrosis Foundation Conference (NACFC; Abstract 529).
  • At NACFC, data were presented on the first 24 subjects (18 treated with CB-280, six with placebo) participating in the ongoing, Phase 1b dose-escalation trial in adults with CF.
  • At the completion of each dose cohort, unblinded data were reviewed by an Independent Data Safety Monitoring Committee (IDMC) convened by the Cystic Fibrosis Foundation (CFF).
  • A recorded presentation of the poster, A phase 1b, randomized, double-blind, placebo-controlled, dose escalation trial of CB-280, an arginase inhibitor, in patients with cystic fibrosis, will be available to NACFC registered attendees.

Spirovant to Present SP-101 Data at the 2021 North American Cystic Fibrosis Conference

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Thursday, October 28, 2021
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W05.5: Delivery of SP-101 restores CFTR function in human CF airway epithelial cultures and drives hCFTRΔR transgene expression in the airways of ferretsNovember 3, 2021, 11:00 AM - 1:00 PM ET Presenter: Katherine Excoffon, Ph.D., VP, Research, Spirovant Sciences Presented During: W05--PTC-GMS: Approaches for Delivery of Nucleotide Based Therapeutics

Key Points: 
  • PHILADELPHIA, Oct. 28, 2021 /PRNewswire/ -- Spirovant Sciences , a gene therapy company developing treatments and cures for inherited respiratory diseases including cystic fibrosis (CF), todayannounced that it will be delivering oral and poster presentations highlighting pre-clinical data for its compound, SP-101, at the 2021 North American Cystic Fibrosis Conference (NACFC 2021), November 2-5.
  • Spirovant is a gene therapy company focused on changing the course of cystic fibrosis and other respiratory diseases.
  • The company's current investigational gene therapy technologies are designed to overcome the historical barriers that have prevented effective genetic treatments for cystic fibrosis.
  • Spirovant's lead programs are in development for cystic fibrosis.

Health Union to Present Research on Quality of Life Impact at Three Conferences Throughout Fall 2021

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Monday, October 25, 2021
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PHILADELPHIA, Oct. 25, 2021 /PRNewswire/ -- Health Union has been accepted to present research at three prestigious conferences throughout fall 2021, focusing on the experiences and perspectives of people living with cystic fibrosis, hidradenitis suppurativa and spondyloarthritis.

Key Points: 
  • PHILADELPHIA, Oct. 25, 2021 /PRNewswire/ -- Health Union has been accepted to present research at three prestigious conferences throughout fall 2021, focusing on the experiences and perspectives of people living with cystic fibrosis, hidradenitis suppurativa and spondyloarthritis.
  • The research, leveraging data from the company's syndicated, condition-specific In America surveys, highlights Health Union's deep understanding of the entire patient journey, including significant condition impact on quality of life.
  • Health Union will present quantitative research on how patients perceive the impact of cystic fibrosis transmembrane conductance regulator (CFTR) medication Trikafta on their futures and different aspects of quality of life.
  • Health Union is presenting research at the International Society for Quality of Life Research's 28th Annual Conference.