FDA places clinical hold on Regenxbio's gene therapy for Hunter syndrome

Aug 24 (Reuters) - Regenxbio said on Monday the U.S. FDA has placed its experimental gene therapy for a rare inherited ...
BioSpace

REGENXBIO’s Hunter syndrome gene therapy hit by FDA hold, stymieing resubmission plans

The FDA has placed a clinical hold on RGX-121 after small nodules or cystic masses were detected in the spines of five ...
BioPharma Dive

Safety concerns spur FDA to again halt testing of Regenxbio gene therapy

Investigators detected masses on the spines of five patients who received its Hunter syndrome treatment, leading to the ...

Regenxbio: Sell-Off On Hunter Setback Presents A Risky Buy Opportunity

Regenxbio (RGNX) analysis: high-volatility “risky buy” amid FDA hold, cash runway to 2027, and big gene therapy catalysts ...

GC Biopharma's Hunter Syndrome Treatment 'Hunterase' Receives Drug Approval in India and Taiwan

GC Biopharma (006280.KS), a leading global pharmaceutical company based in South Korea, announced today that its Hunter ...
Hosted on MSN

Enzyme replacement therapy offers hope for ultra-rare Hunter syndrome

Ongoing clinical research at UNC could lead to a first-of-its-kind enzyme replacement therapy for Hunter syndrome, an ultra-rare disorder that causes progressive multisystem disease and neurologic ...
NewsBytes

India approves Hunterase for rare genetic disorder Hunter syndrome

Hunter syndrome, or MPS II, is a rare genetic disorder caused by an enzyme deficiency. It leads to progressive damage in the ...
FierceBiotech

Denali eyes approval after Hunter syndrome drug clears safety measures, reduces biomarkers in open-label study

After winning a breakthrough therapy designation for its Hunter syndrome enzyme replacement therapy, Denali Therapeutics is climbing closer to its goal of accelerated approval by unveiling data ...