ASH 2022 – Orchard gets a Sanfilippo boost

ASH 2022 – Orchard gets a Sanfilippo boost

Source: 
EP Vantage
snippet: 

There are no approved therapies for the rare lysosomal storage disorder Sanfilippo syndrome, or mucopolysaccharidosis type III. Several gene therapy players are hoping to change this, though – and one of these, Orchard Therapeutics, had promising early data at Ash on Monday.