Vertex Pharma has teamed up with Orum Therapeutics to develop a conditioning regimen, used to prepare patients to receive gene-editing medicines, that it hopes will offer a more tolerable alternative to current chemotherapy options.Vertex is paying $15 million upfront to get the ball rolling on the collaboration, which could be worth up to $945 million if it exercises options to use the technology for three developmental programmes and all of them hit various milestones.The deal focuses on the development of degrader-antibody conjugates (DACs), a fairly new therapeutic category that combines the properties of antibody-drug conjugates (ADCs) and protein degrader technologies, although the targets and mechanisms that are involved in the alliance remain under wraps.Intensive chemo is used before treatment with gene-editing medicines, including Vertex's CRISPR Therapeutics-partnered Casgevy (exagamglogene autotemcel) which became the first based on the CRISPR/Cas9 gene-editing technology to be approved towards the end of last year.

Vertex Pharma与Orum Therapeutics合作开发了一种调理方案，用于为患者接受基因编辑药物做好准备，希望能为目前的化疗方案提供一种更耐受的替代方案。Vertex正在预付1500万美元以推动合作，如果Vertex在三个开发项目中使用该技术，并且所有这些项目都达到了不同的里程碑，那么合作的价值可能高达9.45亿美元。该协议侧重于开发降解物-抗体偶联物（DAC），这是一种相当新的治疗类别，结合了抗体-药物偶联物（ADC）和蛋白质降解技术的特性，尽管联盟中涉及的目标和机制仍不明确。在基因编辑药物治疗之前使用强化化疗，包括Vertex的CRISPR Therapeutics与Casgevy（exagamglogene autotemcel）合作，该药物成为去年年底批准的第一种基于CRISPR/Cas9基因编辑技术的药物。

The $2.2 million, one-shot regimen is used to treat sickle cell disease (SCD) and transfusion-dependent beta thalassaemia (TDT).A few days before Casgevy is infused, patients undergo myeloablative chemo to wipe out their bone marrow, removing unhealthy blood stem cells and making room for the new, edited versions.

这项耗资220万美元的一次性治疗方案用于治疗镰状细胞病（SCD）和输血依赖性β地中海贫血（TDT）。在输注卡西维前几天，患者接受清髓化疗以清除骨髓，去除不健康的造血干细胞，并为新的编辑版本腾出空间。

The chemo has to be very aggressive, so it can be hard to tolerate, is often unsuitable for frail patients, and carries other risks like infertility.A gentler conditioning regimen could make Casgevy and other gene-editing therapies more accessible for patients, along with potentially other interventions that rely on myeloablative chemo like some cell and gene therapies.It's worth mentioning that the Orum deal comes as Vertex has sued th.

化疗必须非常积极，因此很难耐受，通常不适合体弱的患者，并带有其他风险，如不孕症。更温和的预处理方案可以使Casgevy和其他基因编辑疗法更容易为患者接受，以及可能依赖于清髓化疗的其他干预措施，如某些细胞和基因疗法。值得一提的是，Orum交易是Vertex起诉th的时候达成的。