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Pharma & Biotech

Wedbush sees Rocket Pharmaceuticals as significantly undervalued as cardiac gene therapy pipeline advances

The broker maintains its 'outperform' rating and $16 price target on shares trading at $4.97

Wedbush Securities has reiterated its bullish stance on Rocket Pharmaceuticals, the rare disease gene therapy company, after its fourth-quarter update confirmed that patient dosing in its pivotal Phase 2 study of RP-A501 for Danon disease will resume in the first half of 2026.

Danon disease is a rare and life-threatening inherited condition affecting the heart, muscles and brain, for which no approved gene therapy yet exists.

Wedbush analyst Yun Zhong said that with a minimum four-week interval required between dosing of the three additional patients due to receive treatment, all patient dosing could be completed by the third quarter of 2026, potentially allowing the US Food and Drug Administration to complete its data review by year end.

The broker also highlighted the expansion of the company's cardiac pipeline, with a third programme, RP-A701 targeting BAG3-dilated cardiomyopathy, a genetic form of heart failure, set to enter Phase 1 clinical testing in mid-2026 with Fast Track designation already granted.

Cash of approximately $188.9 million at the end of the fourth quarter supports a funding runway into the second quarter of 2027, which Wedbush said could extend to 2028 if the company's lentiviral therapy Kresladi receives FDA approval by its regulatory deadline of 28 March 2026.

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