Featured data showcase the transformative potential of HSC gene therapy to enable cross-correction and restore enzymatic ...

Immusoft Corporation, a cell therapy company dedicated to improving the lives of patients with rare diseases, today announced that it will feature oral and poster presentations at the upcoming ...

Haliza Md Kasim’s eyes widened. Despite having gone through this before with her first son, Yusof, and despite the doctors explaining that this was a genetic disease affecting boys, inherited from ...

With a regulatory decision expected before the end of this year, RGX-121 could become the first gene therapy approved for Hunter syndrome. Also known as mucopolysaccharidosis type II, Hunter ...

Giving gene therapy to newborn mice with Batten disease ... and similar neurodegenerative conditions in children, such as mucopolysaccharidoses, another group of rare inherited diseases caused ...

Giving gene therapy to newborn mice with Batten disease ... and similar neurodegenerative conditions in children such as the mucopolysaccharidoses, another group of rare inherited diseases caused ...

Regenxbio has entered into a strategic collaboration worth $810m with Japan-based Nippon Shinyaku to advance gene therapies targeting the rare metabolic disorder mucopolysaccharidosis (MPS).

Both are rare genetic disorders called mucopolysaccharidoses ... allows RegenxBio “to leverage our expertise in gene therapy manufacturing while also capturing milestones and a meaningful ...