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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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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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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. For HCC patients with concurrent inferior vena cava tumour thrombus, treatment options are limited and overall prognosis is poor. months postoperatively.

Therapies 162
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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. Three additional patients are currently within the normal cognitive development range at nine to 18 months post-treatment, but require longer follow-up to assess outcomes.

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. BPM31510IV BPM31510IV has become the first treatment for primary coenzyme Q10 (CoQ10) deficiency to receive RPDD designation.

Disease 147
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Research reveals therapeutic potential of mRNA in rare diseases

Drug Discovery World

Researchers have used messenger RNA (mRNA) to create an effective therapy for a rare liver disease in preclinical studies, demonstrating the technology’s potential therapeutic use in people. Successful industry/academia collaboration Fewer than 5% of rare diseases have approved therapies and most of these treatments use gene therapy.

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. months after initial treatment. 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