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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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