Cystic Fibrosis
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Cystic Fibrosis Rescued by Reprogramming the Cell’s Secretory Machinery
1/3/2024
Porosome Therapeutics, Inc. announced the publishing of a study that brings promise for a cure to cystic fibrosis.
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Sionna Therapeutics to Present Updates on Clinical Progress in Cystic Fibrosis at 42nd Annual J.P. Morgan Healthcare Conference
1/3/2024
Sionna Therapeutics to Present Updates on Clinical Progress in Cystic Fibrosis at 42nd Annual J.P. Morgan Healthcare Conference.
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BiomX Announces Positive Topline Results from Part 2 of the Phase 1b/2a Trial Evaluating BX004 for Treatment of Chronic Pulmonary Infections in Patients with Cystic Fibrosis
11/29/2023
BiomX Inc. today announced positive safety and efficacy results from Part 2 of the Phase 1b/2a trial evaluating the Company’s novel phage cocktail, BX004, for the treatment of chronic pulmonary infections caused by Pseudomonas aeruginosa (or P. aeruginosa) in patients with cystic fibrosis (“CF”).
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Arcturus Therapeutics Receives Orphan Drug Designation from the U.S. FDA for ARCT-032, for the Treatment of Cystic Fibrosis
11/27/2023
Arcturus Therapeutics Holdings Inc. today announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for the Company’s product candidate ARCT-032 to treat cystic fibrosis (CF).
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Vertex to Present Data Highlighting Long-Term Benefits of TRIKAFTA® at the North American Cystic Fibrosis Conference
11/2/2023
Vertex to Present Data Highlighting Long-Term Benefits of TRIKAFTA ® at the North American Cystic Fibrosis Conference.
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Tract Bio Announces Publication in American Journal of Respiratory and Critical Care Medicine Highlighting Stem Cell Variants Driving Lung Inflammation in Modulator-Treated Cystic Fibrosis Patients
11/1/2023
Tract Bio today announced the publication of a research article in the American Journal of Respiratory and Critical Care Medicine (AJRCCM) highlighting the stem cell variants driving chronic lung inflammation in modulator-treated cystic fibrosis patients (CFTR modulators).
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Moligo Technologies and Nationwide Children's Hospital Collaborate to Advance Cystic Fibrosis Research Using Ultrapure Long Single-Stranded DNA
10/24/2023
Moligo Technologies announced today that it has signed a collaboration agreement with Nationwide Children's Hospital to provide long, single-stranded DNA for its research into gene therapies for cystic fibrosis.
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ATUM Partners with Anagram Therapeutics to Extend Collaboration to Develop Orally Delivered Enzyme Therapies for Malabsorption and Nutrient Metabolism Disorders
10/4/2023
ATUM today announced an expanded partnership with Anagram Therapeutics Inc., a clinical-stage biopharmaceutical company dedicated to improving the lives of people with cystic fibrosis (CF) and other rare diseases.
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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
9/26/2023
Arcturus Therapeutics Holdings Inc. (the announced today that Arcturus and the Cystic Fibrosis Foundation (CF Foundation) have extended their ongoing agreement.
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CF Foundation Invests Up to $9 Million in Arcturus Therapeutics to Develop a Messenger RNA Therapy
9/26/2023
The Cystic Fibrosis Foundation is investing up to $9 million in additional funds in Arcturus Therapeutics to test an inhaled messenger RNA (mRNA) therapy that could treat the underlying cause of cystic fibrosis in all people living with the disease, including those with two copies of rare and nonsense mutations.
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BiomX to Present Data from Ongoing Phase 1b/2a Study Evaluating BX004 for the Treatment of Chronic Pseudomonas aeruginosa Pulmonary Infections
9/7/2023
BiomX Inc. today announced that the Company will present data from Part 1 of its ongoing Phase 1b/2a study evaluating the novel phage product candidate, BX004, for the treatment of chronic Pseudomonas a eruginosa.
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Porosome Therapeutics, Inc. Files Comprehensive Patent for New Generation of Treatments for Cystic Fibrosis and Other Diseases
8/21/2023
Porosome Therapeutics, Inc. is the first company with a technology platform that develops novel therapies targeting the porosome, the universal cellular structure controlling secretion.
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The biopharma company reported sales of $2.24 billion in the second quarter for the cystic fibrosis treatment, beating analysts’ estimate, after it won FDA approval in children ages two to five in April.
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Alcresta Therapeutics Announces Publication of Cystic Fibrosis Foundation Position Paper Confirming Inline Enzyme Cartridges are Safe and Effective
7/25/2023
Alcresta Therapeutics, Inc. today announced the publication of a position paper in the May issue of the Journal of Cystic Fibrosis.
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First Wave BioPharma Announces Final Patient Dosed in Phase 2 SPAN Clinical Trial of Enhanced Adrulipase FormulationTopline data expected in July 2023
6/28/2023
First Wave BioPharma, Inc. today announced that the final patient was dosed in the Phase 2 SPAN clinical trial investigating an enhanced enteric microgranule delivery formulation of adrulipase for the treatment of exocrine pancreatic insufficiency (EPI) in patients with cystic fibrosis (CF).
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First Wave BioPharma to Attend the 36th National Cystic Fibrosis Education Conference (July 28-30)
6/15/2023
First Wave BioPharma, Inc. announced that it will attend the Cystic Fibrosis Research Institute’s 36th National Cystic Fibrosis Education Conference being held July 28-30, 2023, at the Grand Bay Hotel San Francisco in Redwood City, California.
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First Wave BioPharma Reaches Enrollment Target for Phase 2 SPAN Adrulipase Clinical Trial in Cystic Fibrosis
6/13/2023
First Wave BioPharma, Inc. announced that it has achieved the enrollment target in its ongoing Phase 2 SPAN clinical trial investigating an enhanced enteric microgranule delivery formulation of adrulipase for the treatment of exocrine pancreatic insufficiency in patients with cystic fibrosis.
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Drugs that act on the CFTR protein only work in patients who produce the protein in the first place. That leaves 6% of patients hanging.
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4DMT to Present Interim Data from 4D-710 Phase 1/2 AEROW Clinical Trial for Cystic Fibrosis Lung Disease at the European Cystic Fibrosis Society (ECFS) 46th Annual Meeting
5/24/2023
4D Molecular Therapeutics today announced an oral presentation of the interim data from the 4D-710 Phase 1/2 AEROW clinical trial for the treatment of cystic fibrosis lung disease at the European Cystic Fibrosis Society 46th Annual Meeting held in Vienna, Austria on June 7-10, 2023.
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Tavanta Therapeutics Announces New Preclinical Data for its Cystic Fibrosis Candidate TAVT-135 at Two Prominent Scientific Conferences
5/24/2023
Tavanta Therapeutics today announced presentations of new preclinical data for TAVT–135, a novel inhaled chloride ion transporter in development for pan-genotypic treatment of cystic fibrosis (CF).