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AGTC Catches Eyes, PTLA Bleeds As Antidote Sales Disappoint, RARE Jumps

(RTTNews) - Today's Daily Dose brings you news about Applied Genetic's X-linked retinitis pigmentosa trial results, FDA approval of the first targeted therapy to treat a rare mutation in patients with gastrointestinal stromal tumors, DBV Tech's long-term data from phase III open-label extension study of Viaskin Peanut, Portola Pharma's disappointing Q4 Andexxa global net revenues and Ultragenyx Pharma's phase I/II study results of DTX301 in ornithine transcarbamylase deficiency.

Read ona?¦

Shares of Applied Genetic Technologies Corp. (AGTC) surged as much as over 130% on Thursday, following positive interim six-month data from its ongoing phase I/II clinical trial of AAV gene therapy product candidate expressing the RPGR protein for the treatment of X-linked retinitis pigmentosa.

X-linked retinitis pigmentosa (XLRP) is an inherited ocular disease caused by mutations in the RPGR gene and is characterized by progressive vision loss in boys and young men.

The patients experienced a durable improvement in visual function six months after dosing in the trial, according to the Company.
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