Rocket Pharmaceuticals Inc. ( RCKT ) shares are up during Friday’s premarket session following the FDA’s accelerated approval of Kresladi (marnetegragene autotemcel), the first gene therapy for children with severe leukocyte adhesion deficiency-I (LAD-I).

FDA Accelerated Approval Drives Investor Sentiment

This significant development is boosting investor sentiment as the company aims to enhance financial flexibility through the monetization of a Rare Pediatric Disease Priority Review Voucher granted by the FDA, which could further support its growth strategy.

The first FDA-approved gene therapy for this ultra-rare condition underscores Rocket’s commitment to addressing critical pediatric health needs.

How Kresladi Works

Kresladi is an autologous hematopoietic stem cell-based gene therapy that treats severe LAD-I, a rare, inherited immune deficiency caused by mutations in the ITGB2 gene that prevent white blood cells from effectively fighting infections.

Kresladi uses the patient’s own hematopoietic (blood) stem cells (HSCs), which clinicians genetically modify to introduce functional copies of the ITGB2 gene.

After conditioning, clinicians infuse a single intravenous dose of Kresladi to address the underlying cause of severe LAD-I by restoring CD18 and CD11a cell surface expression in white blood cells, including neutrophils.

Clinical Data Shows Strong Survival And Safety Profile

The approval is supported by positive clinical efficacy and safety data from the global Phase 1/2 study of Kresladi, which demonstrated 100% overall survival at 12 months post-infusion (and for the entire duration of follow-up) for all enrolled patients.

All primary and secondary endpoints were met, and Kresladi was well tolerated in all patients with no treatment-related serious adverse events.

The data showed substantial reductions in the incidence of significant infections compared to pre-treatment levels, along with evidence of improvement in skin lesions and restoration of wound-healing capabilities.

Technical Analysis

Currently, Rocket Pharmaceuticals ( RCKT ) is trading 12.15% above its 20-day simple moving average (SMA) and 39.7% above its 100-day SMA, demonstrating a strong short-term bullish trend.

Shares have decreased by 40.48% over the past 12 months and are currently positioned closer to their 52-week highs than lows, suggesting a recovery phase in the stock’s price action.

The RSI is at 55.68, which is considered neutral territory, indicating that the stock is neither overbought nor oversold at this time.

Meanwhile, MACD is at 0.1168, below its signal line at 0.1807, indicating bearish pressure on the stock.

The combination of neutral RSI and bearish MACD suggests mixed momentum, indicating that while the stock is showing some strength, there are underlying bearish signals that traders should monitor closely.

Key Resistance: $5.50

Key Support: $4.50

Analyst Consensus & Recent Actions: The stock carries a Buy Rating with an average price target of $32.25. Recent analyst moves include:

Goldman Sachs: Sell (Raises Target to $3.00) (Mar. 2)

Cantor Fitzgerald: Overweight (Raises Target to $10.00) (Feb. 27)

RCKT Stock Price Activity: Rocket Pharmaceuticals ( RCKT ) shares were up 10.66% at $5.19 during premarket trading on Friday, according to Benzinga Pro data.

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