Positive results for patients enrolled in CIRM-funded trial of a rare pediatric disease

Leukocyte Adhesion Deficiency-I (LAD-I) is a rare pediatric disease that prevents patients from combating infections. This leads to recurring bacterial and fungal infections that respond poorly to antibiotics, require frequent hospitalizations, and can be fatal. It is caused by a mutation in a specific gene that causes low levels of a protein called CD18. The low levels of CD18 affect the immune system’s ability to work efficiently and reduces 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 corrected version of the mutated 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 expression to 4-10 percent is associated with survival into adulthood. 

Rocket presented promising results from four patients enrolled in the trial at the Clinical Immunology Society 2021 Annual Meeting.

Patient 1001 was 9 years-of-age at enrollment and had been followed for 18-months after treatment. Patient 1004 was 3 years-of-age at enrollment and had been followed for 9-months. Patients 2006 and 2005 were 7 months- and 2 years-of-age at enrollment and had been followed for 3-months.

Key findings from trial include the following:

  • RP-L201 was well tolerated and no safety issues reported with infusion or treatment.
  • Patient 1001 demonstrated CD18 expression of about 40 percent and resolution of skin lesions with no new lesions reported 18-months post-treatment.
  • Patient 1004 demonstrated CD18 expression of about 28 percent 9-months post-treatment.
  • Patient 2006 demonstrated CD18 expression of about 70 percent 3-months post-treatment.
  • Patient 2005 demonstrated CD18 expression of about 51 percent 3-months post-treatment.

In a news release, Jonathan Schwartz, M.D., Chief Medical Officer and Senior Vice President of Rocket expressed optimism for these findings.

“Today’s positive updates on our LAD-I program add to the growing body of encouraging evidence that RP-L201 may provide durable clinical benefit for patients with severe LAD-I who face recurrent, life-threatening infections from birth.”

To access the poster used for this presentation, visit Rocket’s website linked here.

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.

CIRM funded trial for LAD-I announces positive results

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 has announced positive results from a CIRM-funded 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.  

The two patients enrolled in the CIRM funded trial have shown restored levels of CD18. Previous studies have indicated that an increase in CD18 to 4-10% is associated with survival into adulthood. The two patients demonstrated CD18 levels that exceeded this threshold.

In a news release, Jonathan Schwartz, M.D. Chief Medical Officer and Senior Vice President of Rocket, elaborated on these positive results.

“Patients with LAD-I have markedly diminished expression of the integrin CD18 and suffer from life-threatening bacterial and fungal infections. Natural history studies indicate that an increase in CD18 expression to 4-10% is associated with survival into adulthood. The two patients enrolled in our Phase 1 trial demonstrated restored CD18 expression substantially exceeding this threshold. In addition, we continue to observe a durable treatment effect in the patient followed through one year, with improvement of multiple disease-related skin lesions after therapy and no further requirements for prophylactic anti-infectives.”