Drug Discovery World
JANUARY 29, 2024
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.
Drug Discovery World
JUNE 23, 2023
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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Drug Discovery World
OCTOBER 17, 2023
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.
Drug Discovery World
FEBRUARY 27, 2023
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).
Drug Discovery World
JUNE 6, 2024
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.
DrugBank
JUNE 13, 2024
Gene Therapy: Reprogramming the Body's Cellular Code Gene therapy is an exciting field that treats diseases at their genetic roots. A key part of gene therapy is efficiently delivering the therapeutic genetic material directly into target cells.
Drug Discovery World
JANUARY 11, 2024
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.
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