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Rocket Pharmaceuticals Withdraws FDA Application for Gene Therapy

Story Highlights
  • Rocket Pharmaceuticals withdrew its FDA application for a Fanconi Anemia gene therapy.
  • The withdrawal aligns with strategic priorities and does not reflect safety concerns.
  • Looking for the best stocks to buy? Follow the recommendations of top-performing analysts.
Rocket Pharmaceuticals Withdraws FDA Application for Gene Therapy

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Rocket Pharmaceuticals ( (RCKT) ) just unveiled an update.

On October 3, 2025, Rocket Pharmaceuticals announced the voluntary withdrawal of its Biologics License Application for mozafancogene autotemcel, a gene therapy for Fanconi Anemia, from the FDA. This decision aligns with the company’s strategic focus on projects with clearer regulatory paths and does not reflect any safety concerns. Rocket had already ceased new investments in this therapy and withdrawn its European application in July 2025. The company is open to future partnerships to advance the therapy, preserving the option to re-engage with regulators later.

The most recent analyst rating on (RCKT) stock is a Buy with a $8.00 price target. To see the full list of analyst forecasts on Rocket Pharmaceuticals stock, see the RCKT Stock Forecast page.

Spark’s Take on RCKT Stock

According to Spark, TipRanks’ AI Analyst, RCKT is a Underperform.

Rocket Pharmaceuticals’ overall score reflects its position as a biotechnology firm in the development phase, with significant financial risks due to its lack of revenue and reliance on external financing. Technical indicators suggest potential volatility, while the negative P/E ratio highlights current valuation challenges. The company’s strong equity position is a positive factor, but ongoing cash burn remains a critical concern.

To see Spark’s full report on RCKT stock, click here.

More about Rocket Pharmaceuticals

Rocket Pharmaceuticals operates in the biotechnology industry, focusing on developing gene therapies for rare and devastating diseases. The company prioritizes programs with clear regulatory and commercial pathways.

Average Trading Volume: 3,692,847

Technical Sentiment Signal: Sell

Current Market Cap: $341M

Learn more about RCKT stock on TipRanks’ Stock Analysis page.

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