Disease Hospitals Therapies Virus 
Drug Discovery World
OCTOBER 2, 2024
Three drugs have been granted ‘rare paediatric disease designation’ (RPDD) in the US by the Food and Drug Administration (FDA). The FDA grants RPDD for serious or life-threatening diseases that affect fewer than 200,000 people in the United States and primarily affect patients less than 18 years of age.
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
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
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. Patients affected by the disease are found to also experience an imbalance of glutathione regulation, which is important for liver detoxification.
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. Encouraging results An improvement in neurocognitive assessments compared with natural progression of the disease in one of the children at 18-months post-treatment.
Drug Discovery World
SEPTEMBER 17, 2024
Where do you work, and can you tell us what you’re working on at the moment? I work at Poolbeg Pharma, where we are developing novel treatments for diseases with unmet medical needs and large addressable markets, with programmes in oncology, infectious diseases and endocrinology.
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