Gaurav Shah, Rocket Pharma CEO

Rock­et Phar­ma­ceu­ti­cals says FDA agrees to 12-pa­tient study for ac­cel­er­at­ed ap­proval, ex­pects to raise $175M

Rock­et Phar­ma­ceu­ti­cals an­nounced Wednes­day that it ex­pects to raise $175 mil­lion from a stock sale, a day af­ter the Cran­bury, NJ-based biotech said the FDA agreed to its plan to ap­ply for ac­cel­er­at­ed ap­proval of its Danon dis­ease gene ther­a­py based on a 12-pa­tient study.

Rock­et’s stock $RCKT jumped about 42% on Wednes­day morn­ing, eclips­ing $21 a share.

Rock­et is de­vel­op­ing a one-time gene ther­a­py for Danon dis­ease — a rare X-linked dis­or­der caused by mu­ta­tions in the LAMP2 gene that leads to the weak­en­ing of the heart and skele­tal mus­cles, as well as in­tel­lec­tu­al dis­abil­i­ty. Males with Danon dis­ease gen­er­al­ly live un­til they are around 20 years old, while fe­males live to around 35 or 40. Rock­et’s treat­ment de­liv­ers a func­tion­al ver­sion of the LAMP2 gene via a vi­ral cap­sid called an AAV.

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