News
SpliSense Successfully Completed Phase 1 Study of SPL84, RNA-Based Therapy, for the Treatment of Cystic Fibrosis
BU Researchers See Future Where Lung Disease Is Treatable, and Damaged Lungs are Regenerated
Novel Approach Developed for Engrafting Engineered Cells Into Injured Lung Tissue
Porosome Therapeutics, Inc. Files Comprehensive Patent for New Generation of Treatments for Cystic Fibrosis and Other Diseases
FDA Grants BiomX’s BX004 Clinical Program in Cystic Fibrosis Fast Track Designation
This fish delivers a nasty sting. Could it also save lives?
FDA Approves Kalydeco (ivacaftor) for Infants with Cystic Fibrosis as Young as 4 Months
https://www.cff.org/News/News-Archive/2020/FDA-Approves-Kalydeco-for-Infants-as-Young-as-4-Months/
FDA Approves Kalydeco for Infants as Young as 4 Months
The U.S. Food and Drug Administration has approved the use of Kalydeco® (ivacaftor) for infants with cystic fibrosis as young as four months who have certain mutations.
Vertex to Present New Data at European and North American Virtual Cystic Fibrosis Conferences Highlighting Long-Term Use of CFTR Modulators
https://www.businesswire.com/news/home/20200924005175/en/Vertex-to-Present-New-Data-at-European-and-North-American-Virtual-Cystic-Fibrosis-Conferences-Highlighting-Long-Term-Use-of-CFTR-Modulators
Vertex to Present New Data at European and North American Virtual Cystic Fibrosis Conferences Highlighting Long-Term Use of CFTR Modulators
Moderna and Vertex Establish New Collaboration to Treat Cystic Fibrosis Using Gene Editing
https://www.businesswire.com/news/home/20200916005915/en/Moderna-and-Vertex-Establish-New-Collaboration-to-Treat-Cystic-Fibrosis-Using-Gene-Editing
Moderna and Vertex Establish New Collaboration to Treat Cystic Fibrosis Using Gene Editing
Second collaboration between Moderna and Vertex based on Moderna’s proprietary mRNA and LNP technology
Collaboration will leverage Vertex’s investments and capabilities in genetic technologies for CF
Moderna to receive $75 million upfront, with potential for additional development, regulatory and commercial milestones and royalty payments
A year out from FDA approval, Vertex looks to expand top-selling CF drug to pediatrics
A year out from FDA approval, Vertex looks to expand top-selling CF drug to pediatrics
A year out from FDA approval, Vertex looks to expand top-selling CF drug to pediatrics
Vertex Seeks Nod for Cystic Fibrosis Drugs for Rare Mutations
https://finance.yahoo.com/news/vertex-seeks-nod-cystic-fibrosis-144902840.html
Vertex Seeks Nod for Cystic Fibrosis Drugs for Rare Mutations
Krystal Biotech’s KB407 Granted Orphan Drug Designation by the FDA to Treat Patients With Cystic Fibrosis
http://ir.krystalbio.com/news-releases/news-release-details/krystal-biotechs-kb407-granted-orphan-drug-designation-fda-treat
Krystal Biotech’s KB407 Granted Orphan Drug Designation by the FDA to Treat Patients With Cystic Fibrosis
Enterprise Therapeutics Doses First Subjects in Phase 1 Trial for First-in-Class Cystic Fibrosis Therapy ETD002
https://www.businesswire.com/news/home/20200817005196/en/Enterprise-Therapeutics-Doses-Subjects-Phase-1-Trial
Enterprise Therapeutics Doses First Subjects in Phase 1 Trial for First-in-Class Cystic Fibrosis Therapy ETD002
ETD002 is a novel TMEM16A chloride channel potentiator
Therapy applicable to all cystic fibrosis patients, independent of genotype
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
https://investors.eloxxpharma.com/news-releases/news-release-details/enrollment-us-phase-2-clinical-trial-cystic-fibrosis-has-resumed
Enrollment in the U.S. Phase 2 Clinical Trial in Cystic Fibrosis Has Resumed After Being Paused in Response to the COVID-19 Pandemic
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
https://www.globenewswire.com/news-release/2020/08/11/2076697/0/en/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-.html
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
Arrowhead Pharmaceuticals Initiates Dosing Phase 1/2 Study of ARO-ENaC for Treatment of Cystic Fibrosis
https://ir.arrowheadpharma.com/news-releases/news-release-details/arrowhead-pharmaceuticals-initiates-dosing-phase-12-study-aro
https://www.businesswire.com/news/home/20200811005244/en/Arrowhead-Pharmaceuticals-Initiates-Dosing-Phase-12-Study
Arrowhead Pharmaceuticals Initiates Dosing Phase 1/2 Study of ARO-ENaC for Treatment of Cystic Fibrosis
Aug 11, 2020 at 7:30 AM EDT
Curious clues in war between bacteria in cystic fibrosis patients
https://www.sciencedaily.com/releases/2020/08/200808085752.htm
Curious clues in war between bacteria in cystic fibrosis patients
Date:
August 8, 2020
Source:
University of North Carolina Health Care
Summary:
Several different kinds of bacteria can cause lung infections in people with cystic fibrosis. Pseudomonas aeruginosa, which can cause pneumonia, typically infects infants or young children and persists for life, while Burkholderia cepacia complex species only infect teenagers and adults. Although Burkholderia infections are rare, when they do take hold, they are deadly. Now, scientists have discovered a reason for this pathogen’s apparent age discrimination.
Cystic Fibrosis Foundation and Longwood Fund Launch CF Incubator – Nonprofit commits $20 million to fund projects and accelerate genetic therapies for CF
https://www.businesswire.com/news/home/20200805005130/en/Cystic-Fibrosis-Foundation-Longwood-Fund-Launch-CF
Cystic Fibrosis Foundation and Longwood Fund Launch CF Incubator
Nonprofit commits $20 million to fund projects and accelerate genetic therapies for CF
August 05, 2020 06:00 AM Eastern Daylight Time
BETHESDA, Md.–(BUSINESS WIRE)–Today the Cystic Fibrosis Foundation announced a collaboration with Longwood Fund, a biotech-focused venture capital firm, to accelerate innovative therapies for cystic fibrosis. Together, Longwood and the CF Foundation are dedicating resources to advance projects with transformative potential and incentivize early stage companies to prioritize CF drug discovery and development. The Foundation has committed $20 million to the collaboration as part of its Path to a Cure.
Eloxx Pharmaceuticals Receives U.S. Orphan Drug Designation for ELX-02 for the Treatment of Cystic Fibrosis
https://www.globenewswire.com/news-release/2020/08/04/2072353/0/en/Eloxx-Pharmaceuticals-Receives-U-S-Orphan-Drug-Designation-for-ELX-02-for-the-Treatment-of-Cystic-Fibrosis.html
Eloxx Pharmaceuticals Receives U.S. Orphan Drug Designation for ELX-02 for the Treatment of Cystic Fibrosis
ELX-02 had previously been granted orphan medicinal product designation for the treatment of Cystic Fibrosis by the European Medicines Agency
Researchers to investigate real-world effects of new cystic fibrosis medications
https://www.news-medical.net/news/20200730/Researchers-to-investigate-real-world-effects-of-new-cystic-fibrosis-medications.aspx
Researchers to investigate real-world effects of new cystic fibrosis medications
World-first technology breathes new life into cystic fibrosis detection and treatment
https://medicalxpress.com/news/2020-07-world-first-technology-life-cystic-fibrosis.html
World-first technology breathes new life into cystic fibrosis detection and treatment
A newly discovered disease may lead to better treatment of cystic fibrosis
https://www.sciencedaily.com/releases/2020/06/200605140505.htm
A newly discovered disease may lead to better treatment of cystic fibrosis
Date:
June 5, 2020
Source:
University of Münster
Summary:
Cystic fibrosis is the most frequent severe inherited disorder worldwide. Researchers have now discovered a novel disease that might lead to a better understanding of cystic fibrosis and new treatment options in the future.
CF Foundation Announces up to $5M Award to Support First-Ever Controlled Clinical Study of Phage Therapy in Cystic Fibrosis
https://www.businesswire.com/news/home/20200318005142/en/CF-Foundation-Announces-5M-Award-Support-First-Ever
CF Foundation Announces up to $5M Award to Support First-Ever Controlled Clinical Study of Phage Therapy in Cystic Fibrosis
Study reaffirms the Foundation’s commitment to advance solutions to the growing challenge of antibiotic resistance and evaluate the use of phage to treat infections for people with CF
Translate Bio Receives FDA Fast Track Designation for MRT5005, an inhaled mRNA therapeutic, for the Treatment of Cystic Fibrosis
https://www.biospace.com/article/releases/translate-bio-receives-fda-fast-track-designation-for-mrt5005-for-the-treatment-of-cystic-fibrosis/
Translate Bio Receives FDA Fast Track Designation for MRT5005 for the Treatment of Cystic Fibrosis
Feb 26, 2020
— First inhaled mRNA therapeutic remains on track to report results from its ongoing multiple-ascending dose (MAD) portion of Phase 1/2 clinical trial in the third quarter of 2020 —
Arrevus Receives FDA Orphan Drug Designation for ARV-1801 (sodium fusidate)
https://apnews.com/7d09e75058954f41be956ff39a3ed623
Arrevus Receives FDA Orphan Drug Designation for ARV-1801 (sodium fusidate)
February 18, 2020
New treatment kills off infection that can be deadly to cystic fibrosis patients
https://www.sciencedaily.com/releases/2020/01/200127075247.htm
New treatment kills off infection that can be deadly to cystic fibrosis patients
Enterprise Therapeutics Publishes Paper on Novel Therapeutic Approach for Treatment of All Cystic Fibrosis Patients
https://www.businesswire.com/news/home/20200108005300/en/
Enterprise Therapeutics Publishes Paper on Novel Therapeutic Approach for Treatment of All Cystic Fibrosis Patients
“TMEM16A Potentiation: A Novel Therapeutic Approach for the Treatment of Cystic Fibrosis” published in American Journal of Respiratory and Critical Care Medicine
Paper demonstrates first in class TMEM16A chloride channel potentiators, to accelerate mucociliary clearance
Co-authored by researchers from University of Sussex, University of North Carolina and University of Miami
A new era in cystic fibrosis treatment
https://penntoday.upenn.edu/news/new-era-cystic-fibrosis-treatment
A new era in cystic fibrosis treatment
New drug appears to slow effects of cystic fibrosis, offering hope to long-suffering patients
https://www.chicagotribune.com/lifestyles/health/sc-hlth-new-cystic-fibrosis-drug-0101-20191220-r3jjqdw3ozaknbdrxdfpzxrauu-story.html
New drug appears to slow effects of cystic fibrosis, offering hope to long-suffering patients
Landmark Cystic Fibrosis Treatment Changing Lives After Chicago Clinical Trial
Landmark Cystic Fibrosis Treatment Changing Lives After Chicago Clinical Trial
Landmark Cystic Fibrosis Treatment Changing Lives After Chicago Clinical Trial
The new drug just received FDA approval two months ago
Nationwide Children’s: New cystic fibrosis treatment could treat 90% of patients
https://local12.com/news/local/new-cystic-fibrosis-treatment-could-treat-90-of-patients
Nationwide Children’s: New cystic fibrosis treatment could treat 90% of patients
Corbus Pharmaceuticals Completes Enrollment of Phase 2b Study of Lenabasum for Treatment of Cystic Fibrosis
https://www.globenewswire.com/news-release/2019/11/18/1948686/0/en/Corbus-Pharmaceuticals-Completes-Enrollment-of-Phase-2b-Study-of-Lenabasum-for-Treatment-of-Cystic-Fibrosis.html
Corbus Pharmaceuticals Completes Enrollment of Phase 2b Study of Lenabasum for Treatment of Cystic Fibrosis
New cystic fibrosis drug therapy seen as major breakthrough in treatment
https://www.boston25news.com/news/trending-now/new-cystic-fibrosis-drug-seen-as-major-breakthrough-in-treatment/1004055824
New cystic fibrosis drug therapy seen as major breakthrough in treatment
Cystic fibrosis patients, doctors celebrate FDA’s ‘life-changing’ approval of new treatment
https://www.foxnews.com/health/cystic-fibrosis-celebrate-fdas-approval-trikafta
Cystic fibrosis patients, doctors celebrate FDA’s ‘life-changing’ approval of new treatment
‘A game-changer’: How Vertex delivered on cystic fibrosis
‘A game-changer’: How Vertex delivered on cystic fibrosis
FDA approves ‘miracle’ treatment for cystic fibrosis
https://abcnews.go.com/Health/fda-approves-miracle-treatment-cystic-fibrosis/story?id=66444291
FDA approves ‘miracle’ treatment for cystic fibrosis
Boehringer Ingelheim Announces First Patient Enrolled in Phase II Trial of Novel Cystic Fibrosis Treatment (BALANCE-CF™ 1)
https://www.prnewswire.com/news-releases/boehringer-ingelheim-announces-first-patient-enrolled-in-phase-ii-trial-of-novel-cystic-fibrosis-treatment-balance-cf-1-300937922.html
Boehringer Ingelheim Announces First Patient Enrolled in Phase II Trial of Novel Cystic Fibrosis Treatment (BALANCE-CF™ 1)
– Investigational compound BI ENaC inhibitor delivered twice daily via the Respimat®, a handheld inhaler
Scientists Hope They Have Discovered New Form of CF Therapy
Scientists Hope They Have Discovered New Form of CF Therapy
AzurRx BioPharma Announces Positive Results from Phase 2 Trial with MS1819 in Cystic Fibrosis Patients
https://www.globenewswire.com/news-release/2019/09/25/1920526/0/en/AzurRx-BioPharma-Announces-Positive-Results-from-Phase-2-Trial-with-MS1819-in-Cystic-Fibrosis-Patients.html
AzurRx BioPharma Announces Positive Results from Phase 2 Trial with MS1819 in Cystic Fibrosis Patients
Eloxx Pharmaceuticals Announces Cystic Fibrosis Foundation To Provide Funding and Support for ELX-02 U.S. Phase 2 Clinical Trial
https://investors.eloxxpharma.com/news-releases/news-release-details/eloxx-pharmaceuticals-announces-cystic-fibrosis-foundation-cf
Eloxx Pharmaceuticals Announces Cystic Fibrosis Foundation (CF Foundation) To Provide Funding and Support for ELX-02 U.S. Phase 2 Clinical Trial
Focusing on cystic fibrosis, Translate Bio to raise $90M
https://www.biopharmadive.com/news/focusing-on-cystic-fibrosis-translate-bio-to-raise-90m/563197/
Focusing on cystic fibrosis, Translate Bio to raise $90M
RESEARCH SPOTLIGHT: G3BP1, p62 and USP10 could be therapeutic targets for ubiquitinated protein aggregation disorders such as cystic fibrosis (CF)
https://www.nature.com/articles/s41598-019-46237-1
G3BP1, p62 and USP10 could be therapeutic targets for ubiquitinated protein aggregation disorders such as cystic fibrosis (CF)
Armata Pharmaceuticals Announces Development of New Synthetic Phage Candidate Targeting Pseudomonas aeruginosa
https://finance.yahoo.com/news/armata-pharmaceuticals-announces-development-synthetic-130000947.html
Armata Pharmaceuticals Announces Development of New Synthetic Phage Candidate Targeting Pseudomonas aeruginosa
Santhera Announces Publication of Phase I Clinical Data with POL6014 in Journal of Cystic Fibrosis
https://www.globenewswire.com/news-release/2019/09/10/1913144/0/en/Santhera-Announces-Publication-of-Phase-I-Clinical-Data-with-POL6014-in-Journal-of-Cystic-Fibrosis.html
Santhera Announces Publication of Phase I Clinical Data with POL6014 in Journal of Cystic Fibrosis
Developing Novel Therapies for the Treatment of Respiratory Diseases
https://www.technologynetworks.com/drug-discovery/blog/developing-novel-therapies-for-the-treatment-of-respiratory-diseases-323640
Developing Novel Therapies for the Treatment of Respiratory Diseases
Succinate in cystic fibrosis lungs feeds deadly virus
https://www.drugtargetreview.com/news/48104/succinate-cystic-fibrosis-lungs-feeds-deadly-virus/
FDA Accepts New Drug Application for VX-445 (Elexacaftor), Tezacaftor and Ivacaftor Combination Treatment
https://www.biospace.com/article/releases/fda-accepts-new-drug-application-for-vx-445-elexacaftor-tezacaftor-and-ivacaftor-combination-treatment-/
FDA Accepts New Drug Application for VX-445 (Elexacaftor), Tezacaftor and Ivacaftor Combination Treatment
RESEARCH SPOTLIGHT: A potential new target for the therapeutic removal of neutrophils from the lung in chronic inflammatory disease
https://www.ncbi.nlm.nih.gov/pubmed/31417560?dopt=Abstract
Knocking out cystic fibrosis: CRISPR-Cas may treat the genetic cause
https://medicalxpress.com/news/2019-08-cystic-fibrosis-crispr-cas-genetic.html
Knocking out cystic fibrosis: CRISPR-Cas may treat the genetic cause
Eloxx Pharmaceuticals Will Launch Phase 2 ELX-02 Trial for CF Patients Carrying G542X Mutation
Eloxx Pharmaceuticals Will Launch Phase 2 ELX-02 Trial for CF Patients Carrying G542X Mutation
Eloxx Pharmaceuticals Will Launch Phase 2 ELX-02 Trial for CF Patients Carrying G542X Mutation
Translate Bio Announces Interim Results from Phase 1/2 Clinical Trial of MRT5005 in Patients with Cystic Fibrosis
https://investors.translate.bio/news-releases/news-release-details/translate-bio-announces-interim-results-phase-12-clinical-trial
Translate Bio Announces Interim Results from Phase 1/2 Clinical Trial of MRT5005 in Patients with Cystic Fibrosis
Vertex Names New CEO to Advance Therapies to 90% of CF Patients
Vertex Names Reshma Kewalramani to Succeed Leiden as CEO, Advance Therapies to 90% of CF Patients
Vertex Names New CEO to Advance Therapies to 90% of CF Patients
Proteostasis Therapeutics Initiates Dosing in Global Phase 2 Study in Cystic Fibrosis
http://ir.proteostasis.com/news-releases/news-release-details/proteostasis-therapeutics-initiates-dosing-global-phase-2-study
Proteostasis Therapeutics Initiates Dosing in Global Phase 2 Study in Cystic Fibrosis
Hinge-like protein may open new doors in cystic fibrosis treatment
https://www.sciencedaily.com/releases/2019/07/190709153612.htm
Hinge-like protein may open new doors in cystic fibrosis treatment
Date:
July 9, 2019
Source:
Rockefeller University
Summary:
Drugs known as potentiators alleviate some symptoms of cystic fibrosis. Researchers recently figured out how these compounds work–a finding that may lead to better drugs that patients can more easily afford.
Eluforsen improved CFTR function in cell and animal models of p.Phe508del-CFTR-mediated cystic fibrosis
https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0219182
https://www.ncbi.nlm.nih.gov/pubmed/31251792?dopt=Abstract
Related Articles
Evaluation of eluforsen, a novel RNA oligonucleotide for restoration of CFTR function in in vitro and murine models of p.Phe508del cystic fibrosis.
PLoS One. 2019;14(6):e0219182
Authors: Beumer W, Swildens J, Leal T, Noel S, Anthonijsz H, van der Horst G, Kuiperij-Boersma H, Potman M, van Putten C, Biasutto P, Platenburg G, de Jonge H, Henig N, Ritsema T
Abstract
Cystic fibrosis (CF) is caused by mutations in the gene encoding the epithelial chloride channel CF transmembrane conductance regulator (CFTR) protein. The most common mutation is a deletion of three nucleotides leading to the loss of phenylalanine at position 508 (p.Phe508del) in the protein. This study evaluates eluforsen, a novel, single-stranded, 33-nucleotide antisense oligonucleotide designed to restore CFTR function, in in vitro and in vivo models of p.Phe508del CF. The aims of the study were to demonstrate cellular uptake of eluforsen, and its efficacy in functional restoration of p.Phe508del-CFTR both in vitro and in vivo. In vitro, the effect of eluforsen was investigated in human CF pancreatic adenocarcinoma cells and human bronchial epithelial cells. Two mouse models were used to evaluate eluforsen in vivo. In vitro, eluforsen improved chloride efflux in CF pancreatic adenocarcinoma cell cultures and increased short-circuit current in primary human bronchial epithelial cells, both indicating restoration of CFTR function. In vivo, eluforsen was taken up by airway epithelium following oro-tracheal administration in mice, resulting in systemic exposure of eluforsen. In female F508del-CFTR mice, eluforsen significantly increased CFTR-mediated saliva secretion (used as a measure of CFTR function, equivalent to the sweat test in humans). Similarly, intranasal administration of eluforsen significantly improved nasal potential difference (NPD), and therefore CFTR conductance, in two CF mouse models. These findings indicate that eluforsen improved CFTR function in cell and animal models of p.Phe508del-CFTR-mediated CF and supported further development of eluforsen in human clinical trials, where eluforsen has also been shown to improve CFTR activity as measured by NPD.
PMID: 31251792 [PubMed – in process]
PubMed:31251792
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
https://www.globenewswire.com/news-release/2019/07/08/1879728/0/en/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-Insuffi.html
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
Aridis Pharmaceuticals Receives Orphan Drug Designation for AR-501
https://www.prnewswire.com/news-releases/aridis-pharmaceuticals-receives-orphan-drug-designation-for-ar-501-300875809.html
Emerging gene therapies for cystic fibrosis
https://www.tandfonline.com/doi/abs/10.1080/17476348.2019.1634547?journalCode=ierx20
https://www.ncbi.nlm.nih.gov/pubmed/31215818?dopt=Abstract
Emerging gene therapies for cystic fibrosis.
Expert Rev Respir Med. 2019 Jun 19;:
Authors: Miah KM, Hyde SC, Gill DR
Abstract
Introduction: Cystic fibrosis (CF) remains a life-threatening genetic disease, with few clinically effective treatment options. Gene therapy and gene editing strategies offer the potential for a one-time CF cure, irrespective of the CFTR mutation class. Areas covered: We review emerging gene therapies and gene delivery strategies for the treatment of CF particularly viral and non-viral approaches with potential to treat CF. Expert opinion: It was initially anticipated that the challenge of developing a gene therapy for CF lung disease would be met relatively easily. Following early proof-of-concept clinical studies, CF gene therapy has entered a new era with innovative vector designs, approaches to subvert the humoral immune system and increase gene delivery and gene correction efficiencies. Developments include integrating adenoviral vectors, rapamycin loaded nanoparticles and lung-tropic lentiviral vectors. The characterisation of novel cell types in the lung epithelium, including pulmonary ionocytes, may also encourage cell type-specific targeting for CF correction. We anticipate preclinical studies to further validate these strategies, which should pave the way for clinical trials. We also expect gene editing efficiencies to improve to clinically translatable levels, given advancements in viral and non-viral vectors. Overall, gene delivery technologies look more convincing in producing an effective CF gene therapy.
PMID: 31215818 [PubMed – as supplied by publisher]
PubMed:31215818
FDA expands approval of treatment for cystic fibrosis to include patients ages 6 and older
https://www.fda.gov/news-events/press-announcements/fda-expands-approval-treatment-cystic-fibrosis-include-patients-ages-6-and-older
FDA expands approval of treatment for cystic fibrosis to include patients ages 6 and older
Airway regeneration using iPS cell-derived airway epithelial cells with Cl- channel function
https://www.tandfonline.com/doi/full/10.1080/19336950.2019.1628550
https://www.ncbi.nlm.nih.gov/pubmed/31198082?dopt=Abstract
Airway regeneration using iPS cell-derived airway epithelial cells with Cl- channel function.
Channels (Austin). 2019 Dec;13(1):227-234
Authors: Yoshie S, Omori K, Hazama A
Abstract
induced pluripotent stem (iPS) cells can be differentiated into various cell types, including airway epithelial cells, since they have the capacity for self-renewal and pluripotency. Thus, airway epithelial cells generated from iPS cells are expected to be potent candidates for use in airway regeneration and the treatment of airway diseases such as cystic fibrosis (CF). Recently, it was reported that iPS cells can be differentiated into airway epithelial cells according to the airway developmental process. These studies demonstrate that airway epithelial cells generated from iPS cells are equivalent to their in vivo counterparts. However, it has not been evaluated in detail whether these cells exhibit physiological functions and are fully mature. Airway epithelial cells adequately control water volume on the airway surface via the function of Cl- channels. Reasonable environments on the airway surface cause ciliary movement with a constant rhythm and maintain airway clearance. Therefore, the generation of functional airway epithelial cells/tissues with Cl- channel function from iPS cells will be indispensable for cell/tissue replacement therapy, the development of a reliable airway disease model, and the treatment of airway disease. This review highlights the generation of functional airway epithelial cells from iPS cells and discusses the remaining challenges to the generation of functional airway epithelial cells for airway regeneration and the treatment of airway disease.
PMID: 31198082 [PubMed – in process]
PubMed:31198082
A generic drug from Argentina offers cystic fibrosis families hope
https://www.newscientist.com/article/2205916-a-generic-drug-from-argentina-offers-cystic-fibrosis-families-hope/
A generic drug from Argentina offers cystic fibrosis families hope
Proteostasis Therapeutics Receives Orphan Drug Designation in the EU for PTI-428 for the Treatment of Cystic Fibrosis
https://finance.yahoo.com/news/proteostasis-therapeutics-receives-orphan-drug-110000228.html
Proteostasis Therapeutics Receives Orphan Drug Designation in the EU for PTI-428 for the Treatment of Cystic Fibrosis
Vertex Selects Triple Combination Regimen of VX-445, Tezacaftor and Ivacaftor to Submit for Global Regulatory Approvals in Cystic Fibrosis
https://investors.vrtx.com/news-releases/news-release-details/vertex-selects-triple-combination-regimen-vx-445-tezacaftor-and
Cystic Fibrosis Patients Turn to Experimental Phage Therapy
http://archive.is/sjG3o#selection-257.0-261.134
Cystic Fibrosis Patients Turn to Experimental Phage Therapy
Phages have not been approved by the Food and Drug Administration, but there is growing interest in the treatment for cystic fibrosis.
L-iminosugars show therapeutic potential as anti-inflammatory agents in cystic fibrosis
An excerpt from a recent paper (April 25, 2019) titled, 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:
Biological results herein obtained place ent-1 and congeners among the earliest examples of l-iminosugars acting as anti-inflammatory agents for therapeutic applications in Cystic Fibrosis.
The paper describes how certain iminosugars were able to dramatically reduce the inflammatory response induced by P. aeruginosa… and do it extremely efficiently:
The remarkably low dosage [40-fold vs. counterpart] of the l-iminosugar, combined with its inability to act as inhibitor for most glycosidases, is expected to limit the onset of undesired effects, which are typically associated with the administration of its d-counterpart.
For more information, check out the recent paper (links below).
https://www.sciencedirect.com/science/article/pii/S0223523419303794
https://www.ncbi.nlm.nih.gov/pubmed/31075609?dopt=Abstract
Further reading on iminosugars:
A Systematic Review of the Clinical Efficacy and Safety of CFTR Modulators in Cystic Fibrosis
https://www.nature.com/articles/s41598-019-43652-2
A Systematic Review of the Clinical Efficacy and Safety of CFTR Modulators in Cystic Fibrosis
ABO-401 Gene Therapy Shows Potential to Restore CFTR Function in Preclinical Study
Identification of GLPG/ABBV-2737, a Novel Class of Corrector, Which Exerts Functional Synergy With Other CFTR Modulators
https://www.frontiersin.org/articles/10.3389/fphar.2019.00514/full
Groundbreaking research for cystic fibrosis on brink of finding universal treatment
https://nbc16.com/news/local/new-treatment-for-cystic-fibrosis-on-the-brink-of-breakthrough-at-osu
Great Strides Being Made in Treating Cystic Fibrosis
https://www.kaaltv.com/news/great-strides-being-made-in-treating-cystic-fibrosis/5341778/
By next year, another drug cocktail is expected to be available. “Over 90% of cystic fibrosis patients will finally have a drug that is moderately-effective to significantly-effective at improving their outcome,” said Dr. Wylam.
Researcher gets $3.3 million grant to develop universal treatment for cystic fibrosis
https://www.eurekalert.org/pub_releases/2019-04/osu-rg043019.php
FDA Approves KALYDECO (ivacaftor) as First and Only CFTR Modulator to Treat Eligible Infants with CF as Early as Six Months of Age
https://www.biospace.com/article/releases/fda-approves-kalydeco-ivacaftor-as-first-and-only-cftr-modulator-to-treat-eligible-infants-with-cf-as-early-as-six-months-of-age/
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
https://www.biospace.com/article/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/
How Will We Be Treating Cystic Fibrosis 10 Years From Now?
Possible new treatment for cystic fibrosis [amphotericin]
https://www.wivb.com/news/national/possible-new-treatment-for-cystic-fibrosis/1948622917
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
https://www.dailymail.co.uk/health/article-6933001/Gene-editing-cure-cystic-fibrosis-birth-study-suggests.html
New test could lead to personalized treatments for cystic fibrosis
https://medicalxpress.com/news/2019-04-personalized-treatments-cystic-fibrosis.html
Synspira Therapeutics Receives Orphan Designation for SNSP113 for the Treatment of Cystic Fibrosis in the European Union
https://www.prnewswire.com/news-releases/synspira-therapeutics-receives-orphan-designation-for-snsp113-for-the-treatment-of-cystic-fibrosis-in-the-european-union-300829745.html
Common virus linked to faster disease progression in cystic fibrosis
https://www.eurekalert.org/pub_releases/2019-04/elf-cvl040419.php
Developing a first-in-class regenerative gene therapy for cystic fibrosis
Treating cystic fibrosis patients before birth could safeguard organs
https://www.sciencenews.org/article/treating-cystic-fibrosis-patients-birth-could-safeguard-organs
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.”
https://www.globenewswire.com/news-release/2019/03/27/1773995/0/en/Eloxx-Pharmaceuticals-Presents-Positive-New-Data-for-Lead-Investigational-Drug-ELX-02-at-the-European-Cystic-Fibrosis-Society-ECFS-Basic-Science-Conference.html
https://apnews.com/Globe%2520Newswire/0477890fdeef1e23fb57e43611e83f77
Proteostatis stock plunges on cystic fibrosis triple combo data
https://www.bizjournals.com/boston/news/2019/03/25/proteostatis-stock-plunges-on-cystic-fibrosis.html
Proteostasis Therapeutics Announces Broad New Dataset from Proprietary Combination and Add-On CFTR Modulator Studies in Cystic Fibrosis Patients
https://www.prnewswire.com/news-releases/proteostasis-therapeutics-announces-broad-new-dataset-from-proprietary-combination-and-add-on-cftr-modulator-studies-in-cystic-fibrosis-patients-300817507.html
New method catches cystic fibrosis inflammatory enzyme in the act
https://cen.acs.org/analytical-chemistry/diagnostics/New-method-catches-cystic-fibrosis/97/web/2019/03
Two UIs work together on cystic fibrosis breakthrough
https://www.press-citizen.com/story/news/2019/03/18/new-reserach-limiting-infection-cystic-fibrosis-patients/3161934002/
New University of Illinois Research Points To Possible Cystic Fibrosis Treatment – Interview with Professor Martin Burke
https://will.illinois.edu/news/story/new-u-of-i-research-points-to-possible-cystic-fibrosis-treatment
Amphotericin B improves cystic fibrosis symptoms in cultured cells and pigs
https://cen.acs.org/biological-chemistry/Amphotericin-B-improves-cystic-fibrosis/97/i11
Antifungal drug could help cystic fibrosis patients for whom common treatments don’t work
https://www.sciencemag.org/news/2019/03/antifungal-drug-could-help-cystic-fibrosis-patients-whom-common-treatments-don-t-work
Pore-forming small molecules offer a promising way to tackle cystic fibrosis
https://www.nature.com/articles/d41586-019-00781-y
Vertex’s cystic fibrosis therapy meets goal of late-stage studies
https://uk.reuters.com/article/vertex-pharms-study/vertexs-cystic-fibrosis-therapy-meets-goal-of-late-stage-studies-idUKL3N20S4CR
Vertex cystic fibrosis triple combo therapy succeeds in important trials
https://www.reuters.com/article/us-vertex-pharms-study/vertex-cystic-fibrosis-triple-combo-therapy-succeeds-in-important-trials-idUSKCN1QN1LI
Window of opportunity for treatment of early cystic fibrosis lung infections
https://www.sciencedaily.com/releases/2019/03/190304121505.htm
European HIT-CF Project to Explore ELX-02 as Treatment for Rare CF Mutations
Translate Bio provides update on lead mRNA therapeutic MRT5005, currently in development for the treatment of cystic fibrosis (CF)
https://globenewswire.com/news-release/2019/02/27/1743808/0/en/Translate-Bio-Provides-Updates-on-Cystic-Fibrosis-CF-and-Ornithine-Transcarbamylase-OTC-Deficiency-Programs.html
MaRS Innovation adds to growing portfolio with two new LAB150 projects to create novel therapeutics for Cystic Fibrosis and Respiratory Syncytial Virus
https://globenewswire.com/news-release/2019/02/27/1743264/0/en/MaRS-Innovation-adds-to-growing-portfolio-with-two-new-LAB150-projects-to-create-novel-therapeutics-for-Cystic-Fibrosis-and-Respiratory-Syncytial-Virus.html
Eloxx Pharmaceuticals evaluating investigational compound ELX-02 for treatment of Cystic Fibrosis in patients with nonsense mutations
https://globenewswire.com/news-release/2019/02/26/1742890/0/en/Eloxx-Pharmaceuticals-Announces-Participation-in-the-HIT-CF-Project-on-Cystic-Fibrosis.html
Eloxx Pharmaceuticals Announces Participation in the HIT-CF Project on Cystic Fibrosis
Eloxx is evaluating ELX-02, its lead investigational compound, for the treatment of Cystic Fibrosis in patients with nonsense mutations [Read more…] about Eloxx Pharmaceuticals evaluating investigational compound ELX-02 for treatment of Cystic Fibrosis in patients with nonsense mutations
Correct antibiotic dosing could preserve lung microbial diversity in cystic fibrosis
https://www.sciencedaily.com/releases/2019/02/190222084242.htm [Read more…] about Correct antibiotic dosing could preserve lung microbial diversity in cystic fibrosis
Novel gene therapy approach creates new route to tackle rare, inherited diseases including cystic fibrosis
https://www.eurekalert.org/pub_releases/2019-02/uoih-ngt021719.php