This page tracks new and upcoming treatment options for cystic fibrosis (CF). What’s in development? What’s promising? What’s available soon? Current topics include ETD002, ELX-02, ARO-ENaC, MRT5005, TMEM16A, ABO-401, MS1819, CFTR modulators, VX-445, VX-659, POL6014, PTI-801, gene therapy, airway stem cells, ABBV-2222, BX004, ABCI, ARCT-032, RSP-1502, SPL84, and more.
Last updated: 1 year ago
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- Arcturus Therapeutics and Cystic Fibrosis Foundation Extend Agreement to Advance ARCT-032, an Investigational Messenger RNA (mRNA) Therapeutic to Treat Cystic Fibrosis Sep 26, 2023
- SpliSense Successfully Completed Phase 1 Study of SPL84, RNA-Based Therapy, for the Treatment of Cystic Fibrosis Sep 6, 2023
- BU Researchers See Future Where Lung Disease Is Treatable, and Damaged Lungs are Regenerated Aug 31, 2023
- Novel Approach Developed for Engrafting Engineered Cells Into Injured Lung Tissue Aug 29, 2023
- Porosome Therapeutics, Inc. Files Comprehensive Patent for New Generation of Treatments for Cystic Fibrosis and Other Diseases Aug 21, 2023
- FDA Grants BiomX’s BX004 Clinical Program in Cystic Fibrosis Fast Track Designation Aug 9, 2023
- This fish delivers a nasty sting. Could it also save lives? Jul 24, 2023
- FDA Approves Kalydeco (ivacaftor) for Infants with Cystic Fibrosis as Young as 4 Months Sep 25, 2020
- Vertex to Present New Data at European and North American Virtual Cystic Fibrosis Conferences Highlighting Long-Term Use of CFTR Modulators Sep 24, 2020
- Moderna and Vertex Establish New Collaboration to Treat Cystic Fibrosis Using Gene Editing Sep 16, 2020
- A year out from FDA approval, Vertex looks to expand top-selling CF drug to pediatrics Sep 11, 2020
- Vertex Seeks Nod for Cystic Fibrosis Drugs for Rare Mutations Sep 2, 2020
- Krystal Biotech’s KB407 Granted Orphan Drug Designation by the FDA to Treat Patients With Cystic Fibrosis Aug 17, 2020
- Enterprise Therapeutics Doses First Subjects in Phase 1 Trial for First-in-Class Cystic Fibrosis Therapy ETD002 Aug 17, 2020
- Enrollment in the U.S. Phase 2 Clinical Trial for ELX-02 in Cystic Fibrosis Has Resumed After Being Paused in Response to the COVID-19 Pandemic Aug 12, 2020
- AzurRx BioPharma Announces Positive Interim Data From First Patient Cohort in Phase 2 Clinical Trial of MS1819 in Combination with PERT Therapy in the Treatment of Cystic Fibrosis Patients with Severe Exocrine Pancreatic Insufficiency Aug 11, 2020
- Arrowhead Pharmaceuticals Initiates Dosing Phase 1/2 Study of ARO-ENaC for Treatment of Cystic Fibrosis Aug 11, 2020
- Curious clues in war between bacteria in cystic fibrosis patients Aug 8, 2020
- Cystic Fibrosis Foundation and Longwood Fund Launch CF Incubator – Nonprofit commits $20 million to fund projects and accelerate genetic therapies for CF Aug 5, 2020
- Eloxx Pharmaceuticals Receives U.S. Orphan Drug Designation for ELX-02 for the Treatment of Cystic Fibrosis Aug 4, 2020
- Researchers to investigate real-world effects of new cystic fibrosis medications Jul 30, 2020
- World-first technology breathes new life into cystic fibrosis detection and treatment Jul 13, 2020
- A newly discovered disease may lead to better treatment of cystic fibrosis Jun 5, 2020
- CF Foundation Announces up to $5M Award to Support First-Ever Controlled Clinical Study of Phage Therapy in Cystic Fibrosis Mar 18, 2020
- Translate Bio Receives FDA Fast Track Designation for MRT5005, an inhaled mRNA therapeutic, for the Treatment of Cystic Fibrosis Feb 26, 2020
- Arrevus Receives FDA Orphan Drug Designation for ARV-1801 (sodium fusidate) Feb 18, 2020
- New treatment kills off infection that can be deadly to cystic fibrosis patients Jan 27, 2020
- Enterprise Therapeutics Publishes Paper on Novel Therapeutic Approach for Treatment of All Cystic Fibrosis Patients Jan 8, 2020
- A new era in cystic fibrosis treatment Dec 24, 2019
- New drug appears to slow effects of cystic fibrosis, offering hope to long-suffering patients Dec 20, 2019
- Landmark Cystic Fibrosis Treatment Changing Lives After Chicago Clinical Trial Dec 17, 2019
- Nationwide Children’s: New cystic fibrosis treatment could treat 90% of patients Dec 13, 2019
- Corbus Pharmaceuticals Completes Enrollment of Phase 2b Study of Lenabasum for Treatment of Cystic Fibrosis Nov 18, 2019
- New cystic fibrosis drug therapy seen as major breakthrough in treatment Nov 1, 2019
- Cystic fibrosis patients, doctors celebrate FDA’s ‘life-changing’ approval of new treatment Nov 1, 2019
- ‘A game-changer’: How Vertex delivered on cystic fibrosis Oct 23, 2019
- FDA approves ‘miracle’ treatment for cystic fibrosis Oct 23, 2019
- Boehringer Ingelheim Announces First Patient Enrolled in Phase II Trial of Novel Cystic Fibrosis Treatment (BALANCE-CF™ 1) Oct 16, 2019
- Scientists Hope They Have Discovered New Form of CF Therapy Oct 9, 2019
- AzurRx BioPharma Announces Positive Results from Phase 2 Trial with MS1819 in Cystic Fibrosis Patients Sep 25, 2019
- Eloxx Pharmaceuticals Announces Cystic Fibrosis Foundation To Provide Funding and Support for ELX-02 U.S. Phase 2 Clinical Trial Sep 23, 2019
- Focusing on cystic fibrosis, Translate Bio to raise $90M Sep 18, 2019
- RESEARCH SPOTLIGHT: G3BP1, p62 and USP10 could be therapeutic targets for ubiquitinated protein aggregation disorders such as cystic fibrosis (CF) Sep 13, 2019
- Armata Pharmaceuticals Announces Development of New Synthetic Phage Candidate Targeting Pseudomonas aeruginosa Sep 12, 2019
- Santhera Announces Publication of Phase I Clinical Data with POL6014 in Journal of Cystic Fibrosis Sep 10, 2019
- Developing Novel Therapies for the Treatment of Respiratory Diseases Sep 5, 2019
- Succinate in cystic fibrosis lungs feeds deadly virus Aug 23, 2019
- FDA Accepts New Drug Application for VX-445 (Elexacaftor), Tezacaftor and Ivacaftor Combination Treatment Aug 20, 2019
- RESEARCH SPOTLIGHT: A potential new target for the therapeutic removal of neutrophils from the lung in chronic inflammatory disease Aug 19, 2019
- Knocking out cystic fibrosis: CRISPR-Cas may treat the genetic cause Aug 7, 2019
- Eloxx Pharmaceuticals Will Launch Phase 2 ELX-02 Trial for CF Patients Carrying G542X Mutation Aug 6, 2019
- Translate Bio Announces Interim Results from Phase 1/2 Clinical Trial of MRT5005 in Patients with Cystic Fibrosis Jul 31, 2019
- Vertex Names New CEO to Advance Therapies to 90% of CF Patients Jul 29, 2019
- Proteostasis Therapeutics Initiates Dosing in Global Phase 2 Study in Cystic Fibrosis Jul 25, 2019
- Hinge-like protein may open new doors in cystic fibrosis treatment Jul 9, 2019
- Eluforsen improved CFTR function in cell and animal models of p.Phe508del-CFTR-mediated cystic fibrosis Jul 9, 2019
- AzurRx BioPharma Announces Initiation of Phase 2 Clinical Study for MS1819-SD in combination with standard PERT for Cystic Fibrosis Patients with Severe Exocrine Pancreatic Insufficiency Jul 8, 2019
- Aridis Pharmaceuticals Receives Orphan Drug Designation for AR-501 Jul 1, 2019
- Emerging gene therapies for cystic fibrosis Jun 27, 2019
- FDA expands approval of treatment for cystic fibrosis to include patients ages 6 and older Jun 21, 2019
- Airway regeneration using iPS cell-derived airway epithelial cells with Cl- channel function Jun 14, 2019
- A generic drug from Argentina offers cystic fibrosis families hope Jun 7, 2019
- Proteostasis Therapeutics Receives Orphan Drug Designation in the EU for PTI-428 for the Treatment of Cystic Fibrosis Jun 4, 2019
- Vertex Selects Triple Combination Regimen of VX-445, Tezacaftor and Ivacaftor to Submit for Global Regulatory Approvals in Cystic Fibrosis May 30, 2019
- Cystic Fibrosis Patients Turn to Experimental Phage Therapy May 17, 2019
- L-iminosugars show therapeutic potential as anti-inflammatory agents in cystic fibrosis May 16, 2019
- A Systematic Review of the Clinical Efficacy and Safety of CFTR Modulators in Cystic Fibrosis May 10, 2019
- ABO-401 Gene Therapy Shows Potential to Restore CFTR Function in Preclinical Study May 10, 2019
- Identification of GLPG/ABBV-2737, a Novel Class of Corrector, Which Exerts Functional Synergy With Other CFTR Modulators May 9, 2019
- Groundbreaking research for cystic fibrosis on brink of finding universal treatment May 6, 2019
- Great Strides Being Made in Treating Cystic Fibrosis May 5, 2019
- Researcher gets $3.3 million grant to develop universal treatment for cystic fibrosis Apr 30, 2019
- FDA Approves KALYDECO (ivacaftor) as First and Only CFTR Modulator to Treat Eligible Infants with CF as Early as Six Months of Age Apr 30, 2019
- Abeona Therapeutics to Report New Preclinical Data Demonstrating Therapeutic Potential of ABO-401 for Treatment of Cystic Fibrosis at American Society of Gene and Cell Therapy Annual Meeting Apr 30, 2019
- How Will We Be Treating Cystic Fibrosis 10 Years From Now? Apr 29, 2019
- Possible new treatment for cystic fibrosis [amphotericin] Apr 23, 2019
- Gene editing could cure cystic fibrosis before birth, study suggests: Scientists use controversial CRISPR on mice to fix DNA defects that lead to the lung disease Apr 17, 2019
- New test could lead to personalized treatments for cystic fibrosis Apr 17, 2019
- Synspira Therapeutics Receives Orphan Designation for SNSP113 for the Treatment of Cystic Fibrosis in the European Union Apr 10, 2019
- Common virus linked to faster disease progression in cystic fibrosis Apr 7, 2019
- Developing a first-in-class regenerative gene therapy for cystic fibrosis Apr 2, 2019
- Treating cystic fibrosis patients before birth could safeguard organs Mar 27, 2019
- Eloxx Pharmaceuticals Presents Positive New Data for Lead Investigational Drug, ELX-02, at the European Cystic Fibrosis Society (ECFS) Basic Science Conference – “We believe these ground-breaking new data establish a solid basis for understanding the activity of ELX-02 and it’s potential for development in the treatment of the high unmet medical need cystic fibrosis patients with nonsense mutations.” Mar 27, 2019
- Proteostatis stock plunges on cystic fibrosis triple combo data Mar 25, 2019
- Proteostasis Therapeutics Announces Broad New Dataset from Proprietary Combination and Add-On CFTR Modulator Studies in Cystic Fibrosis Patients Mar 25, 2019
- New method catches cystic fibrosis inflammatory enzyme in the act Mar 19, 2019
- Two UIs work together on cystic fibrosis breakthrough Mar 18, 2019
- New University of Illinois Research Points To Possible Cystic Fibrosis Treatment – Interview with Professor Martin Burke Mar 14, 2019
- Amphotericin B improves cystic fibrosis symptoms in cultured cells and pigs Mar 13, 2019
- Antifungal drug could help cystic fibrosis patients for whom common treatments don’t work Mar 13, 2019
- Pore-forming small molecules offer a promising way to tackle cystic fibrosis Mar 13, 2019
- Vertex’s cystic fibrosis therapy meets goal of late-stage studies Mar 6, 2019
- Vertex cystic fibrosis triple combo therapy succeeds in important trials Mar 6, 2019
- Window of opportunity for treatment of early cystic fibrosis lung infections Mar 4, 2019
- European HIT-CF Project to Explore ELX-02 as Treatment for Rare CF Mutations Mar 4, 2019
- Translate Bio provides update on lead mRNA therapeutic MRT5005, currently in development for the treatment of cystic fibrosis (CF) Feb 27, 2019
- MaRS Innovation adds to growing portfolio with two new LAB150 projects to create novel therapeutics for Cystic Fibrosis and Respiratory Syncytial Virus Feb 27, 2019
- Eloxx Pharmaceuticals evaluating investigational compound ELX-02 for treatment of Cystic Fibrosis in patients with nonsense mutations Feb 26, 2019
- Correct antibiotic dosing could preserve lung microbial diversity in cystic fibrosis Feb 22, 2019
- Novel gene therapy approach creates new route to tackle rare, inherited diseases including cystic fibrosis Feb 19, 2019
- U of S research could advance treatment for Cystic Fibrosis Feb 7, 2019
- UC Berkeley professor collaborates on study to improve cystic fibrosis treatment Feb 5, 2019
- ‘We’re still waiting’: As cystic fibrosis drugs deliver new hope, not everyone is being swept up by scientific progress Feb 4, 2019
- Advances In Cystic Fibrosis Treatment Give Many Hope, But Others Have ‘Panic’ Of Being Left Behind Feb 4, 2019
- Discovery could improve cystic fibrosis treatment Jan 31, 2019
- ‘A hopeful time in CF’; Research leads to new understanding in cystic fibrosis treatment Jan 26, 2019
- Philadelphia gene therapy company awarded $4.5M from Cystic Fibrosis Foundation Jan 23, 2019
Research
- TMEM16A Mediated Mucus Production in Human Airway Epithelial Cells Oct 7, 2020
- Detargeting Lentiviral-Mediated CFTR Expression in Airway Basal Cells Using miR-106b Oct 6, 2020
- Long-term DHA supplementation in CF patients did not improve inflammatory biomarkers or clinical outcomes, compared with placebo Oct 1, 2020
- ELX-02: an investigational read-through agent for the treatment of nonsense mutation related genetic disease Sep 24, 2020
- Treating cystic fibrosis with mRNA and CRISPR Aug 16, 2020
- Novel lung tropic AAV capsids for therapeutic gene delivery Aug 16, 2020
- CFTR Modulator Therapy Enhances Peripheral Blood Monocyte Contributions to Immune Responses in People With Cystic Fibrosis Aug 13, 2020
- Non-viral mediated gene therapy in human cystic fibrosis airway epithelial cells recovers chloride channel functionality Aug 10, 2020
- Synthesis and biological evaluation of thiazole derivatives on basic defects underlying cystic fibrosis: a good starting point for the design of analogues with improved CFTR potentiator activity Aug 9, 2020
- Gene Therapy in Rare Respiratory Diseases: What Have We Learned So Far? Aug 8, 2020
- Diversified Synthetic Pathway of 1, 4-Dihydropyridines: A Class of Pharmacologically Important Molecules Aug 7, 2020
- Cystic Fibrosis Defective Response to Infection Involves Autophagy and Lipid Metabolism Aug 6, 2020
- Pancreatic enzyme replacement therapy for people with cystic fibrosis Aug 5, 2020
- Correction of CFTR function in intestinal organoids to guide treatment of Cystic Fibrosis Aug 3, 2020
- Proteomics and Metabolomics for Cystic Fibrosis Research Jul 30, 2020
- CFTR Modulators Dampen Aspergillus-Induced Reactive Oxygen Species Production by Cystic Fibrosis Phagocytes Jul 24, 2020
- Pendrin stimulates a chloride absorption pathway to increase CFTR-mediated chloride secretion from Cystic Fibrosis airway epithelia Jul 1, 2020
- GM1 as Adjuvant of Innovative Therapies for Cystic Fibrosis Disease Jun 24, 2020
- Highlights From the 33rd Annual North American Cystic Fibrosis Conference (NACFC) Jun 23, 2020
- Antisense oligonucleotide-mediated correction of CFTR splicing improves chloride secretion in cystic fibrosis patient-derived bronchial epithelial cells Jun 10, 2020
- TMEM16A Potentiation: A Novel Therapeutic Approach for the Treatment of Cystic Fibrosis Jan 3, 2020
- Improving outcomes of infections in cystic fibrosis in the era of CFTR modulator therapy Nov 12, 2019
- Anti-inflammatories and mucociliary clearance therapies in the age of CFTR modulators Nov 12, 2019
- S-Nitrosylation of CHIP Enhances F508Del CFTR Maturation Oct 9, 2019
- Transforming Growth Factor-β1 Selectively Recruits microRNAs to the RNA-Induced Silencing Complex and Degrades CFTR mRNA under Permissive Conditions in Human Bronchial Epithelial Cells Oct 5, 2019
- Inflammatory role of extracellular sphingolipids in Cystic Fibrosis Sep 26, 2019
- IL-1β dominates the promucin secretory cytokine profile in cystic fibrosis Sep 16, 2019
- Revealing the molecular signaling pathways of mucus stasis in cystic fibrosis Sep 16, 2019
- G3BP1, p62 and USP10 could be therapeutic targets for ubiquitinated protein aggregation disorders such as cystic fibrosis (CF) Sep 9, 2019
- Cystic Fibrosis Lung Disease and Immunometabolism: Targeting the NLRP3 Inflammasome Sep 4, 2019
- Whole-blood transcriptomic responses to lumacaftor/ivacaftor therapy in cystic fibrosis Aug 29, 2019
- Modulating phosphoinositide metabolism may promote therapeutic benefits for cystic fibrosis patients Aug 28, 2019
- CK19 stabilizes CFTR at the cell surface by limiting its endocytic pathway degradation Aug 26, 2019
- Scientists develop new type of mouse that will rapidly advance in vivo testing of new CF therapies Aug 14, 2019
- Neutrophil Extracellular Trap Formation: Physiology, Pathology, and Pharmacology Aug 14, 2019
- The impact of CFTR modulator therapies on CF airway microbiology Aug 12, 2019
- The role of endothelial cells in cystic fibrosis Aug 7, 2019
- Targeting IRE1α may improve the metabolic fitness of macrophages and other immune cells in CF Aug 2, 2019
- The CXCL12/CXCR4 Signaling Axis Retains Neutrophils at Inflammatory Sites in Zebrafish Jul 31, 2019
- Advances In Gene Therapy For Cystic Fibrosis Lung Disease Jul 23, 2019
- Discovering the chloride pathway in the CFTR channel Jul 20, 2019
- Lumacaftor-ivacaftor in the treatment of cystic fibrosis: design, development and place in therapy Jul 19, 2019
- Targeted Activation of Cystic Fibrosis Transmembrane Conductance Regulator: two molecular approaches to activate CFTR expression Jul 15, 2019
- Current trends and developments in the clinical translation of nanomedicine for lung diseases, including cystic fibrosis Jul 8, 2019
- CFTR-PTEN-dependent mitochondrial metabolic dysfunction promotes Pseudomonas aeruginosa airway infection Jul 3, 2019
- Enabling Synthesis of ABBV-2222, A CFTR Corrector for the Treatment of Cystic Fibrosis Jul 1, 2019
- The Emerging Role of Neutrophil Extracellular Traps in Respiratory Disease Jun 29, 2019
- Evaluation of eluforsen, a novel RNA oligonucleotide for restoration of CFTR function in in vitro and murine models of p.Phe508del cystic fibrosis Jun 28, 2019
- Emerging gene therapies for cystic fibrosis Jun 27, 2019
- Ivacaftor was safe and effective in severe cystic fibrosis patients with non-G551D gating mutations Jun 25, 2019
- Cystic fibrosis drug ivacaftor (Vx-770) stimulates CFTR channels at picomolar concentrations Jun 17, 2019
- Airway regeneration using iPS cell-derived airway epithelial cells with Cl- channel function Jun 14, 2019
- Augmentation of CFTR function in human bronchial epithelial cells via SLC6A14-dependent amino acid uptake: Implications for treatment of Cystic Fibrosis Jun 12, 2019
- Novel AAV-mediated gene delivery system corrects CFTR function in pigs Jun 11, 2019
- Antisense oligonucleotide eluforsen is safe and improves respiratory symptoms in F508DEL cystic fibrosis Jun 7, 2019
- Network Biology Identifies Novel Regulators of CFTR Trafficking and Membrane Stability Jun 4, 2019
- Focus on TRP channels in cystic fibrosis May 30, 2019
- Antibiofilm efficacy of nitric oxide-releasing alginates against cystic fibrosis bacterial pathogens May 28, 2019
- A Systematic Review of the Clinical Efficacy and Safety of CFTR Modulators in Cystic Fibrosis May 10, 2019
- Identification of GLPG/ABBV-2737, a Novel Class of Corrector, Which Exerts Functional Synergy With Other CFTR Modulators May 9, 2019
- Structural analogues of roscovitine rescue the intracellular traffic and the function of ER-retained ABCB4 variants in cell models Apr 30, 2019
- Restoration of F508-del Function by Transcomplementation: The Partners Meet in the Endoplasmic Reticulum – “These results come at an opportune time for developing a gene therapy for CF and offer new treatment options for a wide range of CF patients.” Apr 29, 2019
- Exploring the effect of chirality on the therapeutic potential of N-alkyl-deoxyiminosugars: anti-inflammatory response to Pseudomonas aeruginosa infections for application in CF lung disease Apr 25, 2019
- Impact of CFTR-modulating drugs on GH-IGF-1 axis impairment in adult patients with cystic fibrosis. Apr 20, 2019
- AMPs as Anti-biofilm Agents for Human Therapy and Prophylaxis Apr 13, 2019
- Microbial Metabolites in Cystic Fibrosis: a Target for Future Therapy? Apr 9, 2019
- Antibacterial Properties and Efficacy of a Novel SPLUNC1-Derived Antimicrobial Peptide, α4-Short, in a Murine Model of Respiratory Infection Apr 9, 2019
- Functional effects of the microbiota in chronic respiratory disease Apr 8, 2019
- Cytomegalovirus – an unrecognised potential contributor to cystic fibrosis disease progression? Apr 7, 2019
- Most important clinical papers in cystic fibrosis published in 2018 – with focus on CFTR modulator therapy Apr 5, 2019
- Bacteriophage PEV20 and Ciprofloxacin Combination Treatment Enhances Removal of Pseudomonas aeruginosa Biofilm Isolated from Cystic Fibrosis Patients Apr 4, 2019
- Cystic Fibrosis Transmembrane Conductance Regulator: A Possible New Target for Photodynamic Therapy Enhances Wound Healing Apr 2, 2019
- Therapeutic Potential of Specialized Pro-resolving Lipid Mediators (SPMs) for the Treatment of Inflammation in Cystic Fibrosis Apr 2, 2019
- Safety and efficacy of treatment with lumacaftor in combination with ivacaftor in younger patients with cystic fibrosis Mar 30, 2019
- Vitamin D inhibits pro-inflammatory cytokines in the airways of cystic fibrosis patients infected by Pseudomonas aeruginosa Mar 29, 2019
- Prevention of cystic fibrosis: The beginning of the end? Mar 27, 2019
- In utero and postnatal VX-770 administration rescues multiorgan disease in a ferret model of cystic fibrosis Mar 27, 2019
- Plasma membrane-localized TMEM16 proteins are indispensable for expression of CFTR and may therefore have an impact on disease severity in cystic fibrosis Mar 26, 2019
- Brevenal, a Marine Natural Product, is Anti-Inflammatory and an Immunomodulator of Macrophage and Lung Epithelial Cells Mar 20, 2019
- Cell-Selective Regulation of CFTR Gene Expression: Relevance to Gene Editing Therapeutics Mar 19, 2019
- Choline Supplementation in Cystic Fibrosis – The Metabolic and Clinical Impact Mar 18, 2019
- Autophagy suppresses the pathogenic immune response to dietary antigens in cystic fibrosis – “autophagy stimulation may mitigate the intestinal malfunction of CF patients” Mar 15, 2019
- Cystic fibrosis transmembrane conductance regulator modulators: Present and future in cystic fibrosis treatment Mar 15, 2019
- Lentiviral Vectors for the Treatment and Prevention of Cystic Fibrosis Lung Disease Mar 14, 2019
- A Practical Review of Proteasome Pharmacology – proteasome function a target for future therapeutic intervention in cystic fibrosis Mar 13, 2019
- Small-molecule ion channels increase host defences in cystic fibrosis airway epithelia Mar 13, 2019
- Clinical validation of an evidence-based method to adjust Pancreatic Enzyme Replacement Therapy through a prospective interventional study in paediatric patients with Cystic Fibrosis Mar 12, 2019
- Two CFTR mutations within codon 970 differently impact on the chloride channel functionality Mar 9, 2019
- Cystic Fibrosis Plasma Blunts the Immune Response to Bacterial Infection Mar 8, 2019
- SMAD Signaling Restricts Mucous Cell Differentiation In Human Airway Epithelium Mar 8, 2019
- The effects of cycled inhaled aztreonam on the cystic fibrosis (CF) lung microbiome Mar 8, 2019
- Pyruvate-depleting conditions induce biofilm dispersion and enhance the efficacy of antibiotics in killing biofilms in vitro and in vivo Mar 6, 2019
- Nonsense Mediated RNA Decay Pathway Inhibition Restores Expression and Function of W1282X CFTR Mar 5, 2019
- Small molecule-facilitated anion transporters in cells for a novel cystic fibrosis therapeutic approach Mar 1, 2019
- Emerging Therapeutic Approaches for Cystic Fibrosis – From Gene Editing to Personalized Medicine Feb 27, 2019
- Enhancing the Expression of CFTR Using Antisense Molecules Against MicroRNA miR-145-5p Feb 27, 2019
- Transcriptome Profiling and Molecular Therapeutic Advances in Cystic Fibrosis: Recent Insights Feb 26, 2019
- Tetrafunctional Block Copolymers Promote Lung Gene Transfer in Newborn Piglets Feb 26, 2019
- A novel membrane-disruptive antimicrobial peptide from frog skin secretion against cystic fibrosis isolates and evaluation of anti-MRSA effect using Galleria mellonella model Feb 22, 2019
- Changes in microbiome diversity following beta-lactam antibiotic treatment are associated with therapeutic versus subtherapeutic antibiotic exposure in cystic fibrosis Feb 22, 2019
Clinical Trials
- A Double-Blind, Active-Controlled, Multiple-Ascending Dose Study of Aerosolized RSP-1502 in Subjects With CF and Chronic PA Lung Infection Aug 29, 2023
- Study to Assess Amphotericin B Cystetic for Inhalation (ABCI) Doses in Healthy Volunteers & People With Cystic Fibrosis (ABCI) Aug 23, 2023
- Nebulized Bacteriophage Therapy in Cystic Fibrosis Patients With Chronic Pseudomonas Aeruginosa Pulmonary Infection Aug 14, 2023
- Safety, Tolerability, and Pharmacokinetics of ARCT-032 in Healthy Adult Subjects Apr 7, 2023
- A Study Evaluating the Long-term Safety of VX-445 Combination Therapy Jun 1, 2020
- Novel Therapeutic Approaches for Treatment of CF Patients With W1282X Premature Termination Codon Mutations May 29, 2020
- A Phase 3 Study of VX-445 Combination Therapy in Cystic Fibrosis (CF) Subjects Heterozygous for F508del and a Gating or Residual Function Mutation (F/G and F/RF Genotypes) May 15, 2020
- A Study Evaluating Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis and F/MF Genotypes Apr 20, 2020
- Glycerol Phenylbutyrate Corrector Therapy For CF (Cystic Fibrosis) Apr 8, 2020
- A Study Evaluating the Efficacy and Safety of VX-445/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects, Homozygous for F508del Mar 5, 2020
- Study to explore the use of off-label CFTR modulators that may affect CFTR function in patients with CFTR mutations that are not currently approved for these drugs Sep 30, 2019
- Study Assessing the Safety, Tolerability, Pharmacokinetics, Food Effect, and Drug-Drug Interactions of PTI-801 in Healthy Volunteers, and Safety, Tolerability, and Pharmacokinetics of PTI-801 in Subjects With Cystic Fibrosis Sep 9, 2019
- Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy Aug 15, 2019
- A DDI Study of FDL169 and FDL176 in Healthy Subjects Aug 1, 2019
- A Phase 2b Randomised, Placebo Controlled Study of OligoG in Patients With Cystic Fibrosis Jul 17, 2019
- A Clinical Study to Investigate Safety, Tolerability and Distribution of CHF 6333 After One or After Repeated Inhalation in Patients With Cystic Fibrosis (CF) Jul 8, 2019
- Human MesenchymAl Stem Cells Infusion in Patients With Cystic Fibrosis Jun 24, 2019
- Study Designed to Assess the Safety, Tolerability and PK of PTI-808 in Healthy Volunteers and in Adults With Cystic Fibrosis Jun 18, 2019
- Glycerol Phenylbutyrate Corrector Therapy [Ravicti] For CF (Cystic Fibrosis) Jun 11, 2019
- A Phase 2 Study of ABBV-3067 Alone and in Combination With ABBV-2222 May 31, 2019
- Novel Therapeutic Approaches for Treatment of CF Patients With W1282X Premature Termination Codon Mutations May 17, 2019
- Dose Escalation Study of ALX-009 in Healthy Men and Cystic Fibrosis (CF) and Non-CF Bronchiectasis Patients May 1, 2019
- A Phase IIa, Randomized, Placebo-controlled, Double-blind, Cross-over Study to Evaluate Safety and Efficacy of Subcutaneous Administration of Anakinra in Patients With Cystic Fibrosis Apr 25, 2019
- iPS Cell Response to CFTR Modulators Apr 16, 2019
- A Study to Evaluate the Safety and Efficacy of VX-121 Combination Therapy in Subjects With Cystic Fibrosis Apr 11, 2019
- A Phase 2 Study to Evaluate Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis Apr 11, 2019
- Beta-cell Response to Incretin Hormones in Cystic Fibrosis Apr 10, 2019
- Study of VX-121 in Healthy Subjects and in Subjects With Cystic Fibrosis Apr 8, 2019
- A Study of AeroVanc for the Treatment of MRSA Infection in CF Patients Apr 4, 2019
- Safety, Tolerability and Efficacy of S-1226 in Cystic Fibrosis Apr 4, 2019
- Effect of Losartan in Cystic Fibrosis (CF) Apr 4, 2019
- Trial of Inhaled Molgramostim in CF Subjects With NTM Infection Apr 3, 2019
- Validation of Respiratory Epithelial Functional Assessment to Predict Clinical Efficacy of Orkambi Mar 28, 2019
- Study to Evaluate the Safety & Tolerability of MRT5005 Administered by Nebulization in Adults With Cystic Fibrosis Mar 21, 2019
- An Efficacy and Safety Study of SPX-101 Inhalation Solution in Subjects With Cystic Fibrosis Mar 19, 2019
- The Effect of PC945 on Aspergillus Fumigatus Lung Infection in Patients With Cystic Fibrosis Mar 12, 2019
- A Phase 1/2a Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of IONIS-ENaCRx in Patients With Cystic Fibrosis Mar 8, 2019
- A Study to Evaluate the Safety and Efficacy of Long-term Treatment With TEZ/IVA in CF Subjects With an F508del CFTR Mutation Mar 1, 2019
- SAD and MAD of Inhaled AR-501 in Health Adults and P. Aeruginosa Infected Cystic Fibrosis Subjects Feb 26, 2019
- A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy Feb 18, 2019
- A Study Evaluating the Long Term Safety and Efficacy of VX-659 Combination Therapy Feb 18, 2019
- OPTION: A Trial to Assess the Safety & Efficacy of MS1819 in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis (OPTION) Feb 15, 2019
- Safety and Tolerability Study of Allogeneic Mesenchymal Stem Cell Infusion in Adults With Cystic Fibrosis (CEASE-CF) Feb 12, 2019
- Study of LAU-7b in the Treatment of Cystic Fibrosis in Adults (APPLAUD) Feb 8, 2019
- Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic Fibrosis Feb 6, 2019
- Study Designed to Assess the Safety, Tolerability and PK of PTI-808 in Healthy Volunteers and in Adults With Cystic Fibrosis Jan 31, 2019
- A Phase 2b Randomised, Placebo Controlled Study of OligoG in Patients With Cystic Fibrosis (CFmised, Double-blind, Parallel-group Study of Alginate Oligosaccharide) Jan 30, 2019
- Safety, Tolerability, and Pharmacokinetics of PTI-808, PTI-801, and PTI-428 Combination Therapy in Subjects With Cystic Fibrosis Jan 30, 2019
- Novel Therapeutic Approaches for Treatment of CF Patients With W1282X Premature Termination Codon Mutations Jan 28, 2019
- Personalized Theratyping Trial Jan 21, 2019
- Study of VX-121 in Healthy Subjects and in Subjects With Cystic Fibrosis Jan 15, 2019
- Human MesenchymAl Stem Cells Infusion in Patients With Cystic Fibrosis (HAPI) Dec 24, 2018
- A DDI Study of FDL169 and FDL176 in Healthy Subjects Nov 28, 2018
- Clinical Study to Investigate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of POL6014 in Patients With CF Nov 20, 2018
- Safety, Pharmacokinetics and Pharmacodynamics Study of Inhaled QBW276 in Patients With Cystic Fibrosis Oct 19, 2018
- Study to Evaluate the Safety & Tolerability of MRT5005 Administered by Nebulization in Adults With Cystic Fibrosis (RESTORE-CF) Sep 27, 2018
Projects
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