Cystic fibrosis

#AI #Autoimmune disease #Breast cancer #Clinical trial #Cystic fibrosis #Diabetes #FDA #Fractyl Health #Gene therapy #GLP-1 #Institute of Cancer Research #Oncology #Phesi

This week in Drug Discovery (18 – 22 May)

News round-up for 18 – 22 May by Bruno Quinney, Content Team at DDW.    This week, a report showed patients in Europe face barriers to patient access. Elsewhere, Wellcome prizes are facilitating research in both tropical disease and mental health.    The… Read More

#News

#AI #Autoimmune disease #Breast cancer #Clinical trial #Cystic fibrosis #Diabetes #FDA #Fractyl Health #Gene therapy #GLP-1 #Institute of Cancer Research #Oncology #Phesi

DDW Highlights: 19 May 2026

The latest episode of the DDW Highlights Podcast is now available to listen to below. DDW’s Bruno Quinney narrates five key stories of the previous week to keep DDW subscribers up-to-date on the latest industry news. Last week, research uncovered new treatment pathways for hard to treat breast cancers. Elsewhere,… Read More

#Podcasts

#AI #Autoimmune disease #Breast cancer #Clinical trial #Cystic fibrosis #Diabetes #FDA #Fractyl Health #Gene therapy #GLP-1 #Institute of Cancer Research #Oncology #Phesi

This week in Drug Discovery (11 – 15 May) 

News round-up for 11 – 15 May by Bruno Quinney, Content Team at DDW.  This week, research has uncovered new treatment pathways for hard to treat breast cancers. Elsewhere, therapies given the green light could combat type 2 diabetes and generalised myasthenia gravis.  The top stories:   … Read More

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Mother helps child to use a nebuliser
#Clinical trial #Cystic fibrosis #Enterprise Therapeutics

Cystic fibrosis treatment could improve lung function 

A new treatment for cystic fibrosis (CF) could improve lung function in patients, results suggest.  ETD001, developed by biopharmaceutical company Enterprise Therapeutics, was trialled in a Phase II study as an inhaled treatment in the 10% of people with CF with the highest unmet medical need, who do… Read More

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Double stranded DNA molecule with mutation in a gene
#Cystic fibrosis #European Commission #Gene mutation #Vertex

EC approves Vertex’s CFTR modulator for cystic fibrosis

The European Commission (EC) has granted approval for Alyftrek (deutivacaftor/tezacaftor/vanzacaftor) for the treatment of people with cystic fibrosis (CF) ages six years and older who have at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.  “Thousands of people with CF across… Read More

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#Clinical trial #Cystic fibrosis #Events #Vertex

Data shows improved quality of life with CFTR modulators

Vertex Pharmaceuticals has shared data demonstrating the positive clinical and quality of life benefits of treatment with cystic fibrosis transmembrane conductance regulator (CFTR) modulators and in particular, Alyftrek (deutivacaftor/tezacaftor/vanzacaftor).  These data were presented at the European Cystic Fibrosis Society’s (ECFS) 48th European Cystic Fibrosis Conference held 4-7… Read More

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Double stranded DNA molecule with mutation in a gene
#Clinical trials #Cystic fibrosis #Drug delivery #Drug development #Europe #Kither Biotech

In Conversation With: Dimitrios Goundis, Kither Biotech

In the In Conversation With series, a part of the free DDW podcast, DDW speaks with members of the drug discovery industry about their work and how it helps turn science into business.    In this episode, Megan Thomas is in conversation with Dimitrios Goundis, CEO… Read More

#News

Child with cystic fibrosis on ventilator
#Boehringer Ingelheim #Cystic fibrosis #Gene therapy #Imperial College London #Phase II trial

Inhaled gene therapy for cystic fibrosis enters the clinic

Boehringer Ingelheim, IP Group, the UK Respiratory Gene Therapy Consortium (GTC) and OXB have announced the start of LENTICLAIR 1, a Phase I/II trial of gene therapy BI 3720931.  The therapy has been designed to improve disease outcomes in people with cystic fibrosis (CF), regardless of gene… Read More

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Bacteria in Petri dishes
#Antibiotics #Antimicrobial resistance (AMR) #Cystic fibrosis #Funding #Glox Therapeutics #LifeArc

Glox awarded share of £3m cystic fibrosis funding

Glox Therapeutics has been awarded a share of the £3 ($3.6) million funding from the Collaborative Discovery Programme (CDP), launched by the Cystic Fibrosis Antimicrobial Resistance (CF AMR) Syndicate with funding from LifeArc.   Glox Therapeutics will receive up to £500,000 of the fund to accelerate the development… Read More

#Business & Investment #News

#Cystic fibrosis #Investment #LifeArc #UK

UK cystic fibrosis network to accelerate new therapeutics

LifeArc and Cystic Fibrosis Trust are jointly investing £15 million to establish a network of research hubs that will accelerate the discovery of novel diagnostics and therapeutics for lung infections. The Translational Innovation Hub Network for Lung Health and Infection will consist of four Innovation Hubs,… Read More

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#Cystic fibrosis #Financing #Funding #Neuroscience #Oncology #Rare disease

This week in drug discovery (30 Sept – 4 Oct) 

News round-up for 30 Sept – 4 Oct by DDW Senior Digital Content Editor Diana Spencer. The theme of the news highlights this week is funding, with companies raising considerable levels of private investment, alongside new funding grants to advance the development of therapeutics for lung infections in… Read More

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#Antimicrobial resistance (AMR) #Cystic fibrosis #Funding #LifeArc #Medicines Discovery Catapult (MDC)

£3m accelerates new drugs for lung infections in cystic fibrosis

The Cystic Fibrosis Antimicrobial Resistance (CF AMR) Syndicate has announced the first cohort of drug discovery projects to receive its Collaborative Discovery Programme (CDP) funding. The £3 ($3.9) million programme, funded by LifeArc, will support six early-stage novel antimicrobial projects over two years to accelerate new… Read More

#Business & Investment #News

Child with cystic fibrosis on ventilator
#Cystic fibrosis #Gene mutation #Phase I trial #Sionna Therapeutics

Two Phase I trials to assess NBD1 stabilisers for cystic fibrosis

The first subjects have been dosed in two separate Phase I clinical trials evaluating the safety, tolerability, and pharmacokinetics of Sionna Therapeutics’ SION-719 and SION-451 in healthy volunteers. SION-719 and SION-451 are product candidates from the company’s series of highly potent nucleotide-binding domain 1 (NBD1) stabilisers… Read More

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#Cystic fibrosis #Enterprise Therapeutics #Phase II trial

Clinical trial will assess novel approach to cystic fibrosis

Enterprise Therapeutics announced it has dosed the first person with cystic fibrosis (pwCF) in its Phase IIa trial of ETD001. ETD001, a low molecular weight compound with first-in-class potential, targets the epithelial sodium channel (ENaC) in the airway epithelium to increase the hydration and clearance of… Read More

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Child with cystic fibrosis on ventilator
#Animal models #Cystic fibrosis #Enterprise Therapeutics #Preclinical development

ENaC blocker enhances mucus clearance in cystic fibrosis models

A preclinical study in cystic fibrosis sheep models has demonstrated that low doses of the ENaC blocker ETD001 can enhance airway mucus clearance with a long duration of action. The study indicates that ETD001, at a dose level that was well tolerated in healthy volunteers, provides… Read More

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