bluebird bio (BLUE) Investor Presentation - Slideshow

Core Program Milestones - Bluebird bio aims for 3 U.S approvals by the end of 2023 [7] - The company plans a commercial launch for beti-cel in early Q4 2022, pending FDA decision [8, 11] - A BLA submission for lovo-cel is planned for Q1 2023 [8, 16, 21] - Potential therapy availability for eli-cel is anticipated in Q4 2022 [9, 23, 34] Beti-cel (Beta-thalassemia) - Approximately 1,500 patients in the U.S are potentially addressable [12] - 90% of patients achieved transfusion independence (TI) and normal or near-normal hemoglobin levels in the LTF-303 study [14] - Patients spent up to 170 hours a year managing transfusions [12] - The average lifetime medical care cost per patient is $6.4 million [12] Lovo-cel (Sickle Cell Disease) - Over 20,000 patients in the US may be addressed by gene therapy [7, 17] - 1 in 365 Black or African American babies is born with sickle cell disease [17] - Patients experienced complete resolution of severe vaso-occlusive events (VOEs) through up to 36 months of follow-up [20] Eli-cel (Cerebral Adrenoleukodystrophy) - Approximately 40 patients are diagnosed with CALD in the U.S each year [22] - 90.6% (29/32) MFD-free survival at 24 months (ALD-102) [29] Financial Status - Restructuring is expected to deliver up to $160 million in cost savings over the next two years [32] - The company anticipates a 35-40% reduction in operating costs by year-end 2022 [32] - As of March 31, 2022, the company had $312 million in cash, cash equivalents, and marketable securities [32]