CONFIDENTIAL planned 2022 milestones – focus on regulatory milestones and commercialization Sickle Cell DiseaseBeta-thalassemia Cerebral Adrenoleukodystrophy Complete manufacturing of commercial drug product validation lots MID-2022 Confirm vector and drug product analytical comparability Q4 2022 BLA submission Q1 2023 Currently evaluating what impact, if any, partial clinical hold may have on BLA target submission date Ad Comm DATE TO BE ANNOUNCED FDA PDUFA date AUGUST 19, 2022 Commercial launch 2022 Ad Comm DATE TO BE ANNOUNCED FDA PDUFA date SEPTEMBER 16, 2022 Commercial launch 2022 1