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$150 Million

Orchard Therapeutics Ltd.


Lead Placement Agent, February 2021

Orchard Therapeutics Ltd.

Orchard Therapeutics is a commercial-stage, fully-integrated biopharmaceutical company dedicated to transforming the lives of patients with fatal rare diseases through autologous ex vivo lentiviral gene therapies. The Company’s gene therapy approach seeks to transform a patient’s own, or autologous, hematopoietic stem cells (HSCs) into a gene-modified drug product to treat the patient’s disease. By utilizing a non-replicating lentiviral vector, Orchard introduces a functional copy of a missing or faulty gene into the patient’s autologous HSCs through an ex vivo process, resulting in a drug product that can then be re-introduced into the patient. Gene modified HSCs have also demonstrated the ability to cross the blood brain barrier, engraft in the brain as microglia and to deliver and express genes and proteins within the CNS.