- FDA places clinical hold on Regenxbio’s RGX-121 gene therapy after spine MRI findings, delaying refiling plansThe U.S. Food and Drug Administration has issued a clinical hold on Regenxbio’s gene therapy candidate RGX-121 after routine MRI scans revealed asymptomatic changes in the spines of trial participa…
- Spruce Biosciences advances toward Q4 FDA filing for ultra-rare Sanfilippo B therapySpruce Biosciences reported progress toward filing its enzyme replacement therapy, tralesinidase alfa (TA-ERT), for Sanfilippo syndrome type B, an ultra-rare lysosomal disorder. The company said t…