The MyPEAK-1 trial investigator Milind Desai said: “TN-201 is the first gene therapy to be tested in HCM patients whose disease is caused by mutations to the MYBPC3 gene. For patients with this ...

It’s called gene therapy. Gene therapy seeks out the root cause of disease by targeting a single gene within a person’s cells. “As techniques advance, treatment becomes more and more ...

In the complex process of producing a gene therapy, bioprocessors depend on assays to monitor the attributes of the product at various stages. Scientists at Thermo Fisher Scientific focus on ...

In a fascinating turn of science and narrative, small mice have assisted scientists to go back in time. By inserting a human-specific gene into their genetic code, scientists potentially opened up ...

A new study in Human Gene Therapy describes a machine learning (ML) model that can be used as a surrogate for laborious in vitro experiments. This in silico approach aims to increase the fitness ...

High dose and low dose AMT-130 demonstrated an 80% and 30% slowing of disease progression vs external control, respectively. The Food and Drug Administration has granted Breakthrough Therapy ...

Benefits of the gene therapy persisted over several years of follow-up. Presymptomatic children with MLD appeared to have the best outcomes. Severe motor impairment or death was significantly less ...

The gene therapy delandistrogene moxeparvovec-rokl showed clinically meaningful benefits and disease stabilization at 2 years in patients with Duchenne muscular dystrophy (DMD). Patients with ...

in Bengaluru and CMC Vellore has resulted in the country's first human gene therapy trial for haemophilia, officials announced on Thursday. Haemophilia is a genetic blood disorder in which blood ...

Artis officially emerged from stealth on Wednesday, marking its launch as a San Diego-based cell and gene therapy CDMO with the acquisition of personalized medicine compatriot Landmark Bio.