Development of Nogo Receptor Decoy for the Treatment of Spinal Cord Injury

脊髓损伤（SCI）会导致身体部分或大部分瘫痪和感觉丧失。许多SCI患者是永久性残疾，一生都依赖轮椅和其他辅助设备。从SCI完全恢复通常是不可能的，因为脊髓的轴突通常受损，不能在成人中再生。 主要合作者开发了一种治疗候选药物（AXER-204），刺激皮质脊髓和中缝脊髓轴突生长。此外，已经表明，在急性、亚急性和慢性脊柱挫伤中可以实现功能恢复。在慢性SCI动物模型中证明疗效的临床前研究为启动AXER-204的临床试验提供了基础。该项目的目的是进一步开发这种疗法，为在人体临床试验中进行测试做准备。 BrIDG团队正在合作完成以下关于AXER-204的研究： - 非GMP和良好生产规范（GMP）材料的合成 - 处方开发 - 药代动力学/吸收、分布、代谢和排泄（PK/ADME）研究 - IND导向毒理学

Spinal cord injuries (SCIs) cause paralysis and loss of feeling in some or most of the body. Many people with SCIs are permanently disabled, relying on wheelchairs and other assistive devices for their entire lives. Complete recovery from SCI is normally not possible because the axons of the spinal cord are usually damaged and cannot regrow in adults. The lead collaborators have developed a therapeutic candidate (AXER-204) that stimulates corticospinal and raphespinal axon growth. Additionally, it has been shown that functional recovery can be achieved in acute, subacute and chronic spinal contusion. Preclinical studies that demonstrate efficacy in an animal model of chronic SCI provide the basis for launching clinical trials of AXER-204. This projects aim is to further develop this therapy to prepare it for testing in human clinical trials. The BrIDGs team is collaborating on the completion of the following studies on AXER-204: - Synthesis of non-GMP and Good Manufacturing Practice (GMP) material - Formulation development - Pharmacokinetic/absorption, distribution, metabolism, and excretion (PK/ADME) studies - IND-directed toxicology