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Swiss scientists identify a virus that kills dormant bacteria

Drug Discovery World

Researchers have found a virus that kills dormant bacteria, a discovery that could help to combat infections that can’t be treated with antibiotics. Studies have shown that combination therapy with this phage and an antibiotic eradicates many dormant germs in pure culture and in a mouse model. They have named their new phage Paride.

Virus 130
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FDA approves first gene therapy for Duchenne muscular dystrophy

Drug Discovery World

Sarepta Therapeutics’ Elevidys has become the first gene therapy for Duchenne muscular dystrophy (DMD) to gain marketing authorisation in the US. The Food and Drug Administration (FDA) has granted accelerated approval to Elevidys (delandistrogene moxeparvovec-rokl), an adeno-associated virus (AAV) based gene therapy for the treatment of DMD.

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CAR T therapy successfully treats solid tumours in China

Drug Discovery World

CARsgen has published evidence of over seven years of disease-free survival in advanced hepatocellular carcinoma (HCC) following treatment with its CAR-GPC3 T cell therapy. However, both patients maintained a tumour-free status during long-term follow-up after receiving a combination of local and CAR T cell therapies.

Therapies 162
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Gene therapy startup emerges with green light for first-of-its-kind trial

BioPharma Drive: Drug Pricing

Spun out of research at Nationwide Children’s Hospital, Vironexis claims it will be the first company to start a study testing a cancer drug delivered via the adeno-associated viruses commonly used in genetic medicines.

Therapies 112
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Gene therapy for ‘childhood dementia’ shows promise

Drug Discovery World

An investigational gene therapy for Sanfilippo syndrome – which leads to a form of childhood dementia – has shown promising early results in a proof-of-concept study. The post Gene therapy for ‘childhood dementia’ shows promise appeared first on Drug Discovery World (DDW).

Therapies 130
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Three drugs secure ‘rare paediatric disease designation’ in the US

Drug Discovery World

ETD001 ETD001 is a novel, first in class blocker of the epithelial sodium channel (ENaC) aimed at treating people with cystic fibrosis (pwCF) without current effective therapies. The drug entered Phase II clinical trials in July 2024, which are expected to complete in 2025.

Disease 147
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Cancer vaccine shows sustained improvement in survival rates

Drug Discovery World

The combination therapy was also shown to reduce the risk of distant metastasis or death by 62% compared to Keytruda alone in these patients. The global Phase III clinical trial is being led by University College London Hospitals NHS Foundation Trust in the UK and will include around 1,100 people.

Vaccine 147