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 ...

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 ...

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 ...

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 ...

REGENXBIO Inc. (Nasdaq: RGNX) today announced data from its RGX-121 (clemidsogene lanparvovec) program for the treatment of mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, will ...

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 ...

(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 ...

MPS includes a group of rare ... the company is progressing RGX-202, a gene therapy for Duchenne muscular dystrophy. Following positive data from a Phase I/II trial, Regenxbio has aligned with ...

Detailed price information for Denali Therapeutics Inc (DNLI-Q) from The Globe and Mail including charting and trades.