Browsing Tag
gene therapy
71 posts
JW Therapeutics says China pharma deals remain insulated from Beijing’s sensitive technology scrutiny
JW Therapeutics says China pharma deals remain open despite Beijing tech scrutiny. See why biotech licensing still matters.
How skeletal endpoints are becoming central to Gaucher disease innovation strategies
FLT201 data shifts focus to skeletal endpoints in Gaucher disease. Discover what this means for gene therapy strategy and market competition.
What clinicians and regulators will watch next after Itvisma’s positive CHMP opinion
Novartis Pharma AG’s Itvisma gains CHMP backing. Discover what clinicians, regulators, and investors will watch next in SMA gene therapy.
Why VG801 could reshape endpoint strategy in inherited retinal disease development
VG801 shows early vision gains in Stargardt disease. Discover how VeonGen Therapeutics is redefining endpoints and regulatory strategy.
Can Ocugen, Inc. disrupt chronic eye injections with a single gene therapy procedure? (NASDAQ: OCGN)
Ocugen, Inc. reports Phase 2 gene therapy results that could reshape retinal treatment economics. Discover what this means for investors and competitors.
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.