Positive results from CIRM-funded LAD-I trial presented at the 62nd American Society of Hematology Annual Meeting

Gaurav Shah, M.D., CEO and President of Rocket Pharmaceuticals

Leukocyte Adhesion Deficiency-I (LAD-I) is a rare pediatric disease caused by a mutation in a specific gene that causes low levels of a protein called CD18. Due to low levels of CD18, the adhesion of immune cells is affected, which negatively impacts the body’s ability to combat infections.

Rocket Pharmaceuticals is conducting a CIRM-funded ($6.56 M) clinical trial that is testing a treatment that uses a gene therapy called RP-L201. The therapy uses a patient’s own blood stem cells and inserts a functional version of the gene.  These modified stem cells are then reintroduced back into the patient. The goal is to establish functional immune cells, enabling the body to combat infections. Previous studies have indicated that an increase in CD18 to 4-10% is associated with survival into adulthood. 

The company presented interim data from the trial at the 62nd American Society of Hematology (ASH) Annual Meeting in the form of an oral presentation. The data presented is from three pediatric patients with severe LAD-I, which is defined by CD18 expression of less than 2%. The patients were all treated with RP-L201. Patient One was 9-years of age at enrollment and had been followed for 12-months as of a cutoff date of November 2020. Patient Two was 3-years of age at enrollment and had been followed for over 6-months. Patient Three was 7-months of age at enrollment and was recently treated with RP-L201.

Key highlights from the presentation include:

  • RP-L201 was well tolerated, no safety issues reported with infusion or post-treatment
  • All patients achieved hematopoietic (blood) reconstitution within 5-weeks
    • 12 months post-treatment, Patient One demonstrated durable CD18 expression of approximately 40%,
    • 6-months post-treatment, Patient Two demonstrated CD18 expression of 23%
    • 2-months post-treatment, Patient Three demonstrated CD18 expression of 76%

In a press release from Rocket, Gaurav Shah, M.D., CEO and President of Rocket, expressed excitement about these results.

“…we continue to see encouraging evidence of efficacy for RP-L201 for the treatment of LAD-I. Patients have shown sustained CD18 expression of 23% to 40%, far exceeding the 4-10% threshold associated with survival into adulthood…”

To view the presentations at the conclusion of the oral presentation, click the link here.