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Dutch company ProQR announces the receipt of FDA authorisation to initiate Phase I/II study for Leber congenital amaurosis, type 10 (LCA10)

Dutch biotechnology company ProQR Therapeutcis N.V., (NASDAQ:PRQR), Leiden, The Netherlands, has announced the receipt of “fast-track” designation from the FDA for their clinical stage development of “QR-110”, an experimental gene editing treatment for Leber’s congenital amaurosis (LCA), type 10. Fast track designation is granted by the FDA to sponsors of treaments under development for serious […]

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Spark Therapeutics files rolling BLA with FDA for new gene therapy product to treat RPE-mediated retinal degeneration

Spark Therapeutics (NASDAQ:ONCE), Philadelphia, PA, has announced the filing of a rolling Biologics License Application (BLA) with the US FDA for their novel gene therapy treatment, “voretigene neparvovec”, for inherited retinal disease. The treatment, previously termed “SPK-RPE65”, is a replacement gene therapy treatment for RPE65-mediated inherited retinal degeneration. According to the company, the regulatory submission […]

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RegenxBio Inc. (NASDAQ:RGNX) raises additional investment funds as long term product for novel wet AMD study enters phase I clinical testing.

RegenxBio Inc. (NASDAQ:RGNX), Rockville, Maryland, USA, has announced the successful raising of $75.9 million through the sale of 3.7 million shares at $20.50/share in a follow-on funding underwritten by Morgan Stanley, Bank of America, Merrill Lynch, Piper Jaffray and Chardan Capital Markets. The clinical stage company claims to control a broad stable of international intellectual […]

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