Browsing Tag
gene therapy
66 posts
A single appeals court ruling puts gene therapy patent economics back in play for Regenxbio Inc. and Sarepta Therapeutics Inc.
Regenxbio Inc. wins a key appeals court reversal against Sarepta Therapeutics Inc. Discover what this means for gene therapy IP and investors.
Will Sarepta’s Elevidys succeed where others stalled? All eyes on EMBARK trial’s 3-year data drop
Find out how Sarepta’s 3-year Elevidys trial data could reshape investor sentiment, regulatory outlook, and Duchenne gene therapy strategy.
Positive early Phase 2 data strengthen Ocugen’s case for OCU410 in the fast-evolving dry AMD treatment market
Find out how Ocugen’s early Phase 2 data for OCU410 could reshape competition and investor sentiment in the fast-growing dry AMD treatment market.
AAVantgarde clears key execution hurdle as LUCE-1 Phase 1/2 study of AAVB-081 completes enrollment
Find out how AAVantgarde Bio’s LUCE-1 enrollment milestone reshapes the gene therapy landscape for Usher syndrome type B retinitis pigmentosa.
Five-year durability data show Hemgenix is transforming haemophilia B care with a single dose
Five‑year results show Hemgenix delivers long‑lasting bleed protection and stable clotting factor levels for haemophilia B patients. Read more on the breakthrough here.
Sangamo Therapeutics wins FDA fast-track status for gene therapy ST-503 targeting small fiber neuropathy
Find out how Sangamo Therapeutics’ FDA fast-track win for ST-503 could reshape small fiber neuropathy treatment and shift biotech investor sentiment.
Will ST‑503 be the non‑opioid breakthrough in pain therapy? Sangamo gets Fast Track nod
Sangamo Therapeutics gains FDA Fast Track for ST-503, a gene therapy targeting chronic neuropathic pain. Find out what this means for non-opioid innovation.
Sangamo Therapeutics shares surge 13% after FDA accepts rolling BLA for ST‑920 in Fabry disease
Sangamo Therapeutics shares surged 13% as the FDA accepted a rolling BLA for ST‑920 in Fabry disease. Learn what this milestone means and what’s next.
Why the FDA’s warning on Elevidys matters for Duchenne muscular dystrophy treatment
Sarepta’s Elevidys gene therapy now carries an FDA boxed warning. Find out what it means for Duchenne patients, investors, and gene therapy safety.
Neurogene (NASDAQ: NGNE) advances Rett syndrome gene therapy with Embolden trial launch and positive pediatric data
Neurogene reports progress on NGN-401 Rett syndrome gene therapy with pivotal trial launch and strong pediatric data. See what’s ahead in 2026.