Amid a reckoning for the gene therapy field, Vertex Pharmaceuticals has joined a growing list of companies paring back their ...

Ver­tex is pulling back on its ge­net­ic med­i­cine re­search, end­ing all work re­lat­ed to ade­no-as­so­ci­at­ed virus­es, ...

Siren Biotechnology is trying to raise $5 million via the type of crowdfunding round that’s rarely used by young drugmakers.

A new study in Human Gene Therapy describes a machine learning (ML) model that can be used as a surrogate for laborious in ...

Multidisciplinary coordination is crucial for effective gene therapy administration in Duchenne muscular dystrophy, focusing on institutional readiness and patient-centered care. Guidelines ...

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 ...

Also Read: uniQure/ CSL Behring’s Hemophilia Gene Therapy Shows Sustained Efficacy and Safety At Four Years According to a 2021 study in Neuroepidemiology, approximately 70,000 people have been ...

and clinical developments that are driving today's explosion of gene therapy advances. Developing AAV capsids with improved yield, or fitness, is a key strategy for reducing manufacturing costs in ...

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 ...

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When discussing challenges in gene therapy, delivery of the genetic payload ... we’ll talk to experts in delivery with a focus on AAV vectors. We’ll discuss the successes the field has ...