A phase I/II open label study to assess safety, feasibility and efficacy of ex vivo expanded, autologous haematopoietic stem and progenitor cell populations that contain CD34+ cells transduced with a lentiviral vector encoding the TCIRG1 cDNA in children with autosomal recessive osteopetrosis caused by mutations in the TCIRG1 gene.

You may be able to join if

• 1. Diagnosis of autosomal recessive osteopetrosis caused by mutations in the TCIRG1 gene, defined by one of the following: Clinical Trial Protocol Document FORM SOP-SC-008.02 Revision 2 Page 16 of 39 EU CT number: 2024-518972-30-00 Protocol number: ARO-FT024-01 Protocol date: 12/02/2025 Protocol version: 1.0 TEMP SOP-GEN-000.05 Rev.4 a. Clinical features of osteopetrosis and documented pathogenic/likely pathogenic biallelic variants (homozygosity or compound heterozygosity, whereby at least 1 allele must contain a known pathogenic mutation) in the TCIRG1 gene causing malignant infantile osteopetrosis. b. If a patient presents with clinical features suggestive of severe osteopetrosis (e.g., generalized osteosclerosis, club-shaped long bones, skull base sclerosis, recurrent fractures and osteomyelitis, cranial nerve entrapment leading to visual and/or hearing loss, bone marrow insufficiency) and at least one pathogenic/likely pathogenic mutation of the TCIRG1 gene.
• 2. Age: ≥ 28 days and ≤ 2 years old
• 3. Body weight: ≥ 4 kg