A Study Investigate the Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Response of SLN124 in Adults With Alpha/Beta-thalassaemia and Very Low- and Low-risk Myelodysplastic Syndrome

Exclusion Criteria

• •Adult with haemoglobin S/alpha-thalassaemia or haemoglobin S/beta-thalassaemia or adult with secondary MDS, i.e., MDS that is known to have arisen because of chemical injury or treatment with chemotherapy and/or radiation for another disease.
• •History of multiple drug allergies or history of allergic reaction to an oligonucleotide or GalNAc, or intolerance to s.c. injections.
• •Known infection with HIV, or active infectious hepatitis A, B, or C virus.
• •Any conditions which, in the opinion of the Investigator, would make the subject unsuitable for enrolment in the study or could interfere with the subject's participation in, or completion of the study.
• •History or clinical evidence of alcohol or illegal drug misuse within 2 years before screening.
• •Currently using ESA, or plan to use ESA at any point during the study.
• •Require daily treatment with 1 or more non-steroidal anti-inflammatory drugs during the study period. Paracetamol will be permitted for use as an antipyretic and/or analgesic.
• •Treatment, or change in treatment with prohibited medications as specified in the protocol
• •Treatment with ICT where the subject has not been on a stable dose for at least 8 weeks before screening or it is planned to initiate ICT therapy during the study.
• •Clinically significant cardiac disease
• •Clinically significant pulmonary disease
• •For subjects with thalassaemia:
• •Treatment, or change in treatment with prohibited medications as specified in the protocol
• •currently and anticipated to receiving more than 5 units of RBCs during the 24 weeks to 6 weeks period before first dose of study drug.
• •For subjects with very low / low-risk MDS:
• •Previous allogeneic or autologous stem cell transplantation.
• •Currently or planned to receive treatment with a corticosteroid for MDS within 8 weeks before screening.
• •Currently or planned to receive treatment with haematopoietic growth factors (e.g., eltrombopag, romiplostim) within 8 weeks before screening.