People with thalassemia and sickle cell anemia got a boost with FDA approval in the final quarter of 2023 of two ex vivo gene editing therapies with mechanistically distinct approaches: in Vertex and CRISPR Therapeutics’ Casgevy (exagamglogene autotemcel), gene editing reactivates fetal hemoglobin production, whereas bluebird bio’s Lyfgenia (lovotibeglogene autotemcel) produces a modified anti-sickling hemoglobin (HbA^T87Q). Casgevy also received approval across Europe. Expected pricing of $2–3 million per patient treated may stifle the global impact, given the distribution of the disease.

The outlook for those with difficult-to-treat small cell lung cancer improved with the emergence of Amgen’s tarlatamab, a T cell–engaging bispecific antibody with affinities for CD3 and delta-like ligand 3, a marker overexpressed on SCLC cells. In a phase 2 trial, objective responses were seen in 40% of those receiving the low dose (10 mg) of the bispecific, whereas the rate was just 32% in high-dose recipients. If tarlatamab’s phase 2 data succeed before the FDA in June 2024 in the priority review, it will become the first bispecific antibody approved for a solid cancer indication.

FDA 于 2023 年最后一个季度批准了两种体外基因编辑疗法，其治疗方法在机制上截然不同：在 Vertex 和 CRISPR Therapeutics 的 Casgevy (exagamglogene autotemcel) 中，基因编辑重新激活胎儿血红蛋白的产生，从而使地中海贫血和镰状细胞性贫血患者得到了提振。而 bluebird bio 的 Lyfgenia (lovotibeglogene autotemcel) 则生产改良的抗镰状血红蛋白 (HbA ^T87Q )。Casgevy 也获得了整个欧洲的批准。考虑到该疾病的分布情况，每位治疗患者的预计定价为 2-300 万美元，这可能会抑制全球影响。

随着安进公司 tarlatamab 的出现，难治性小细胞肺癌患者的前景得到改善，tarlatamab 是一种 T 细胞双特异性抗体，对 CD3 和 δ 样配体 3（一种在 SCLC 细胞上过度表达的标记物）具有亲和力。在一项 2 期试验中，接受低剂量（10 毫克）双特异性抗体的受试者中有 40% 出现客观反应，而高剂量受试者中这一比例仅为 32%。如果 tarlatamab 的 2 期数据在 2024 年 6 月 FDA 优先审查中获得成功，它将成为第一个被批准用于实体癌症适应症的双特异性抗体。