The UK regulatory authorities have approved the first ever trial of a revolutionary gene therapy for young children diagnosed with Hunter syndrome, a devastating rare lysosomal storage disorder. Five ...

Hopes were high that a drug developed by Sangamo based on its zinc finger nuclease (ZFN) genome-editing technology could lead to the first therapy that could correct the genetic defect n MPS I ...

Long-term safety data with median follow-up of two years, and out to more than four years, demonstrate that tividenofusp alfa was generally well tolerated Regulatory submission for accelerated ...

Ultragenyx Pharmaceutical RARE reported new findings from an ongoing, pivotal study of its investigational candidate, UX111 (ABO-102) AAV gene therapy, for Sanfilippo syndrome type A (MPS IIIA). Per ...

After winning a breakthrough therapy designation for its Hunter syndrome enzyme replacement therapy, Denali Therapeutics is ...

Regenxbio Inc. remains undervalued despite its cutting-edge AAV gene therapy technology and promising assets like RGX-314 for wet AMD and RGX-121 for MPS II. RGX-314, partnered with AbbVie ...

(NASDAQ: DNLI), today announced the primary analysis of the Phase 1/2 study in 47 participants with Hunter syndrome (MPS II) in the 24-week ... recent Breakthrough Therapy designation, further ...

RGX-121 is poised to be the first gene therapy for MPS II with potential FDA approval as early as late 2025, and RGX-111 has demonstrated very promising results in phase 1/2 study. With Nippon ...

RGX-121 is poised to be the first gene therapy for MPS II with potential FDA approval as early as late 2025, and RGX-111 has demonstrated very promising results in Phase 1/2 study. With Nippon ...

Furthermore, the gene therapy candidate demonstrated a favorable safety profile and continues to be overall well tolerated in MPS II patients. REGENXBIO is currently gearing up to use CSF levels ...

(NASDAQ: RARE) today announced the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA or the Agency) seeking accelerated approval for UX111 (ABO-102) AAV ...

REGENXBIO's partnership with Nippon Shinyaku provides $110M upfront, ensuring a cash runway for gene therapy programs in MPS I & II, despite sacrificing some upside. REGENXBIO is rated a HOLD ...