Rocket Refocuses Pipeline As Gene Therapy Sentiment Sours

Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a firm focused on developing cardiovascular gene therapy, announced a strategic corporate reorganization to extend its runway.

The restructuring and reprioritization initiative focuses Rocket’s resources on the adeno-associated virus (AAV) cardiovascular platform, comprised of clinical programs in Danon disease, PKP2-associated arrhythmogenic cardiomyopathy (PKP2-ACM), and BAG3-associated dilated cardiomyopathy (BAG3-DCM), as well as submitting the completed responses to the CRL for KRESLADI (formerly RP-L201) for severe leukocyte adhesion deficiency-I (LAD-I).

Also Read: Rocket Pharma’s Gene Therapy Future Clouded By Regulatory Uncertainty

The transition reflects the company’s focus on its late-stage AAV pipeline, a strategy previously announced in January 2025.

The company will reduce its workforce by approximately 30%. Along with other planned cost-saving initiatives, Rocket expects this will reduce it’s 12-month operating expenses by nearly 25%.

Rocket anticipates that its existing cash resources will fund operations into the second quarter of 2027.

Based on business and strategic priorities and as part of its realignment, Rocket anticipates delays associated with the Fanconi Anemia (FA; RP-L102) and Pyruvate Kinase Deficiency (PKD; RP-L301) programs.

Regulatory approval of RP-L102 in 2026 is unlikely.

In May, the FDA placed a clinical hold on the company’s Phase 2 pivotal trial of RP-A501, investigational gene therapy for Danon disease.

Analyst Reaction: Bank Of America (BofA) Securities’ Jason Zemansky downgraded Rocket from Buy to Neutral and reduced the price forecast from $9 to $4.

The analyst states that the gene therapy for Danon disease was the primary driver of Rocket Pharmaceuticals’ performance. But after recent high-profile failures in the gene therapy space, investor confidence has weakened.

BofA writes the company is indirectly admitting that the outlook for its lead program remains uncertain in the near term. While the cost savings may be beneficial in the long run, near-term gains appear to be limited.

Without a clear timeline and with continued concerns around the gene therapy class, investors may remain cautious.

Price Action: RCKT stock is down 2.80% at $3.12 at the last check on Friday.

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