Orchard Therapeutics ^{Priced, Nasdaq: ORTX}

We are a commercial-stage, fully-integrated biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through autologous ex vivo gene therapies. Our 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 through a single administration. We achieve this outcome by utilizing a lentiviral vector to introduce 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 at the bedside. Our portfolio currently includes Strimvelis, our commercial-stage gammaretroviral-based product for the treatment of ADA-SCID, five lentiviral product candidates in clinical-stage development and several other product candidates in preclinical development. We expect to submit a BLA with the FDA for our product candidate OTL-101, a lentiviral gene therapy for ADA-SCID, in 2020, followed by an MAA with the EMA. We also plan to submit an MAA with the EMA for our next most advanced clinical candidate, OTL-200 for the treatment of MLD, in 2020 followed by a BLA with the FDA.