Browsing Tag
gene therapy
71 posts
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.
MeiraGTx signs $475m ophthalmology gene therapy collaboration with Eli Lilly
MeiraGTx Holdings plc signed a USD 475 million gene therapy deal with Eli Lilly and Company to develop AAV-AIPL1 and apply riboswitch tech in ophthalmology.
uniQure moves closer to FDA approval as AMT-130 gene therapy shows progress for Huntington’s disease
Find out how uniQure’s AMT-130 regulatory path for Huntington’s disease is evolving after the FDA’s latest feedback on its data sufficiency.
Breakthrough hope: Benitec Biopharma’s BB-301 delivers promising results in rare muscular dystrophy trial
Find out how Benitec Biopharma’s BB-301 gene therapy is showing early success in restoring swallowing function for OPMD patients.
Can Lilly’s milestone-heavy deal for Adverum reshape how Big Pharma acquires gene therapy platforms?
Lilly’s $12.47-per-share buyout of Adverum Biotechnologies puts milestone CVRs at the center of biotech M&A. But can this deal model truly balance risk and reward?
Lilly’s $262m Adverum Biotechnologies buyout marks a major push into ocular gene therapy
Find out how Eli Lilly’s $262 million Adverum Biotechnologies buyout is advancing gene-therapy innovation in vision care!