Remove Hospitals Remove Therapies Remove Trials Remove Virus
article thumbnail

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.

article thumbnail

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
Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

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
article thumbnail

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
article thumbnail

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
article thumbnail

Meet the Researcher: Liam Tremble

Drug Discovery World

I am the lead scientist on our oncology and infectious disease programmes, where I manage and oversee everything related to drug discovery and work closely with our VP of Clinical Operations on our clinical trials. Administration of these cancer immunotherapies is therefore restricted only to tertiary cancer centres.

Research 221
article thumbnail

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