Morgan Stanley sees this gene therapy stock doubling from here

Rocket Pharmaceuticals could dream as a trial with blockbuster sales potential enters a pivotal phase, according to Morgan Stanley. Analyst Michael Ulz initiated the stock at overweight with a $45 price target. That target share price implies the stock will rally 107.1% from Tuesday’s close. Ulz called Rocket a “leading gene therapy company” that can find the best treatments for a given disease. He said the recent expansion of its cardiovascular pipeline can help the company stand out given competitors’ focus on liver diseases and provide long-term value. In the meantime, he said the hematology, or blood disease, area of ​​the company is underappreciated and can give the company near-term revenues. Both areas of the business should have new data in the next year. Ulz said he’s specifically watching the RP-A501, a “first and best-in-class” treatment for Damon disease, which is a rare and fast-moving disease that impacts organ systems and has led to heart failure and death by age 20. Aside from Rocket’s potential alternative, the only current treatment is a heart transplant. RP-A501, a one-time gene therapy treatment, has shown “promising” phase one results among adults and children, Ulz said. Rocket has gotten feedback from the Food and Drug Administration signaling an accelerated path to the market, he said, and is expecting a second-phase study to begin in the second quarter of this year. The drug, according to the analyst, could rake in more than $1.3 billion, on a risk-adjusted basis. “We view RPA501 (AAV) as a key driver, with blockbuster sales potential, based on very promising Ph1 data supporting advancement into a pivotal study expected in 2Q23,” he said in a Wednesday note to clients. The stock has fluctuated between gains and losses in recent years. Before losing 60.2% and 10.4% in 2021 and 2022, respectively, it gained 53.6%. and 141% in 2019 and 2020. It’s up about 12% this year. RCKT 5Y mountain Rocket Pharmaceuticals—CNBC’s Michael Bloom contributed to this report.

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