Rocket Pharmaceuticals wins FDA nod for KRESLADI gene therapy

Rocket Pharmaceuticals ( (RCKT) ) has issued an update.

On March 27, 2026, Rocket Pharmaceuticals announced that the U.S. Food and Drug Administration granted accelerated approval to KRESLADI, an autologous hematopoietic stem cell-based gene therapy for pediatric patients with severe leukocyte adhesion deficiency-I caused by biallelic ITGB2 variants who lack a matched sibling donor. The decision makes KRESLADI the first FDA-approved gene therapy for severe LAD-I, with continued approval dependent on verification of clinical benefit through longer-term follow-up and a post-marketing registry, and it underscores the therapy’s potential to address a life-threatening, ultra-rare immunodeficiency marked by high early-childhood mortality.

With KRESLADI’s approval, Rocket received a Rare Pediatric Disease Priority Review Voucher, which the company plans to monetize to strengthen its financial flexibility and support shareholder value. The approval, supported by years of research and clinical collaboration and partly funded by the California Institute for Regenerative Medicine, enhances Rocket’s position in the gene therapy space while offering a new treatment option for a very small population of severely affected children and signaling growing regulatory support for targeted genetic therapies in primary immunodeficiencies.

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 Neutral.

The score is held back primarily by weak financial performance (no revenue, persistent losses, and ongoing cash burn with equity erosion risk). Technicals are supportive due to strong trend and positive MACD, but extremely overbought RSI/Stochastics temper the technical score. Valuation is constrained by negative earnings and no provided dividend yield.

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

More about Rocket Pharmaceuticals

Rocket Pharmaceuticals, Inc. is a fully integrated biotechnology company focused on advancing a pipeline of genetic therapies for rare disorders with high unmet medical need. Listed on Nasdaq under the ticker RCKT and based in Cranbury, N.J., the company concentrates on gene therapies for ultra-rare pediatric diseases, targeting conditions with limited or no effective treatment options.

Average Trading Volume: 2,781,017

Technical Sentiment Signal: Sell

Current Market Cap: $509.2M

See more data about RCKT stock on TipRanks’ Stock Analysis page.