Cystic fibrosis

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.

Verona Pharma and Nuance Pharma Announce $219 Million Strategic Collaboration to Develop and Commercialize Ensifentrine in Greater China

Thursday, June 10, 2021 - 7:00am

Ensifentrine also activates the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR), which is beneficial in reducing mucous viscosity and improving mucociliary clearance.

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.
  • The Company is evaluating nebulized ensifentrine in its Phase 3 clinical program ENHANCE (Ensifentrine as a Novel inHAled Nebulized COPD thErapy) for COPD maintenance treatment.
  • Since its inception in 2014, Nuance Pharma has assembled a portfolio of promising clinical-stage drug candidates for respiratory, pain and iron deficiency anemia.

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.

BiomX to Present Preclinical Results with BX004 in Cystic Fibrosis at the 44th European Cystic Fibrosis Conference

Wednesday, June 9, 2021 - 11:30am

BiomX Inc. (NYSE American: PHGE) (BiomX or the Company), a clinical-stage microbiome company advancing novel natural and engineered phage therapies that target specific pathogenic bacteria, today announced that preclinical results with BX004 in Cystic Fibrosis (CF) will be presented at the 44th European Cystic Fibrosis Conference (ECFS).

Key Points: 
  • BiomX Inc. (NYSE American: PHGE) (BiomX or the Company), a clinical-stage microbiome company advancing novel natural and engineered phage therapies that target specific pathogenic bacteria, today announced that preclinical results with BX004 in Cystic Fibrosis (CF) will be presented at the 44th European Cystic Fibrosis Conference (ECFS).
  • In vitro results demonstrate that BiomXs phage cocktail, BX004, was able to penetrate biofilm and was efficacious in significantly reducing bacterial levels of Pseudomonas aeruginosa encased in biofilm, relative to two different antibiotics.
  • Encouraged by these promising preclinical results, we look forward to advancing BX004 in CF to a Phase 1b/2a trial with results from Part 1 expected in the first quarter of 2022.
  • This press release contains express or implied forward-looking statements within the meaning of the safe harbor provisions of the U.S.

First-of-Its-Kind Study Uses Smartphone to Track Cystic Fibrosis in Real Life

Wednesday, June 2, 2021 - 2:30pm

The study will rely on observations that individuals living with cystic fibrosis make about their health in their daily lives to understand the impact of Trikafta when taken in the real world.

Key Points: 
  • The study will rely on observations that individuals living with cystic fibrosis make about their health in their daily lives to understand the impact of Trikafta when taken in the real world.
  • People ages 12 or older living with cystic fibrosis, who are taking Trikafta are eligible to participate.
  • The HERO-2 study is being conducted by Indiana University School of Medicine with funding from the Cystic Fibrosis Foundation.
  • The Cystic Fibrosis Foundation is the world's leader in the search for a cure for cystic fibrosis.

Hunterian Medicine Awarded Therapeutics Development Award from the Cystic Fibrosis Foundation

Thursday, May 27, 2021 - 5:47pm

Hunterian Medicine LLC , a gene-editing and gene therapy company, has been awarded a Therapeutics Development Award by the Cystic Fibrosis Foundation .

Key Points: 
  • Hunterian Medicine LLC , a gene-editing and gene therapy company, has been awarded a Therapeutics Development Award by the Cystic Fibrosis Foundation .
  • "We are especially grateful to receive the Cystic Fibrosis Foundations support for our cystic fibrosis gene therapy program," said Dr. Vinny Jaskula-Ranga, President and CEO of Hunterian Medicine.
  • The vision behind the Path to a Cure initiative is just another example of what sets the Cystic Fibrosis Foundation apart."
  • As part of the award, Hunterian will collaborate with cystic fibrosis experts from the Johns Hopkins University School of Medicine and the Johns Hopkins Cystic Fibrosis Center.

Eloxx Pharmaceuticals Announces Funding Award from Cystic Fibrosis Foundation for Development of Ribosome Modulating Agents

Thursday, May 27, 2021 - 1:30pm

We are honored to receive this funding from the Cystic Fibrosis Foundation in our ongoing commitment to identify and develop new treatments for CF.

Key Points: 
  • We are honored to receive this funding from the Cystic Fibrosis Foundation in our ongoing commitment to identify and develop new treatments for CF.
  • We have tremendous confidence in the potential of RMAs designed with TURBO-ZMto treat rare genetic diseases, said Sumit Aggarwal, President and Chief Executive Officer.
  • Mr. Aggarwal continued, In addition, the development of a Ribosome Modulating Agent for the treatment of CF with support from the Cystic Fibrosis Foundation complements our ongoing ELX-02 program, currently in Phase 2 clinical trials.
  • Eloxx Pharmaceuticals, Inc.is engaged in the science of ribosome modulation, leveraging both its innovative TURBO-ZMchemistry technology platform in an effort to develop novel Ribosome Modulating Agents (RMAs) and its library of Eukaryotic Ribosome Selective Glycosides (ERSGs).

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

Thursday, May 27, 2021 - 1:30pm

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.

Key Points: 
  • 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.

CF Foundation Funds Three New Research Awards to Advance its Path to a Cure Initiative

Thursday, May 27, 2021 - 1:30pm

Once identified, the compounds will go through chemical testing to select those that can be developed into drugs.

Key Points: 
  • Once identified, the compounds will go through chemical testing to select those that can be developed into drugs.
  • Nearly 4,000 people with CF in the U.S. have one or more nonsense mutations, which are challenging targets for drug discovery and development.
  • The CF Foundation launched its $500 million Path to a Cure initiative in October 2019.
  • The Foundation funds more CF research than any other organization, and nearly every CF drug available today was made possible because of Foundation support.