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Rocket Pharmaceuticals Announces Positive Data From RP-L102, RP-L201 Gene Therapy Programs

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  • Rocket Pharmaceuticals Inc (NASDAQ: RCKT) has announced positive data from its Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), and Pyruvate Kinase Deficiency (PKD) gene therapy programs.

  • Data were presented at the American Society of Gene and Cell Therapy Annual Meeting.

  • RP-L102 for FA showed evidence of preliminary engraftment in at least six of nine patients.

  • Although preliminary, four out of the five patients anticipated necessary for a positive trial outcome have initially met the minimum 10% MMC resistance threshold in the bone marrow on at least one occasion, including two patients at 6-months post-treatment.

  • RP-L102 demonstrated a favorable safety profile. One patient experienced a Grade 2 transient infusion-related reaction.

  • All four LAD-I patients with follow-up between 3 to 18 months after RP-L201 treatment had CD18 expression that exceeded the 4-10% threshold associated with survival into adulthood and consistent peripheral blood vector copy number, demonstrating durable clinical benefit.

  • All of these patients have been free of serious infections since hospital discharge following RP-L201 therapy.

  • RP-L301 for Pyruvate Kinase Deficiency showed no serious safety issues or infusion-related complications observed up to 9-months post-treatment. RP-L301 continued to be well-tolerated.

  • Price Action: RCKT shares are up 2.3% at $42.57 during the market session on the last check Thursday.

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