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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