Safety and Efficacy of PMT Therapy of hPAP

Inclusion criteria

• ✓. Male or female with a confirmed diagnosis of hPAP defined as:
• ✓. Diffuse ground glass opacification of the lungs visualized on a chest computed tomogram (CT)
• ✓. History of prior receipt of WLL therapy or moderate hPAP lung disease severity requiring therapy in the opinion of the Clinical Site Investigator and/or Sponsor
• ✓. Able to undergo bone marrow collection by routine clinical aspiration
• ✓. 18 years of age or older on the date the Informed consent form (ICF) is signed
• ✓. Females who have been post-menopausal for \>2 years or females of child-bearing potential after a confirmed menstrual period using a highly efficient method of contraception (as described in Section 11.4.2) for the period from 3 months prior to the first administration of gene-corrected macrophages until 12 months after the last administration of gene-corrected macrophages. Females of child-bearing potential must have a negative serum pregnancy test at Screening (Visit 1), at bone marrow collection (Visit 2), and immediately before each administration of gene-corrected macrophages (Visits 3, 5, 7), and must not be lactating.
• ✓. Males of reproductive potential must agree to use condoms for the period from the 1st administration of gene-corrected macrophages until 12 months after the last dose of gene-corrected macrophages, have a partner who is not of child-bearing potential (i.e. men or females who have been post-menopausal for \>2 years), or have a female partner who is using adequate contraception as described in Section 11.4.2.
• ✓. Signed written informed consent form (ICF)

Exclusion criteria