Development of a novel, regenerative therapy to reverse synapse loss in Alzheimer's Disease

项目总结/摘要 SBIR计划的使命是“鼓励国内小企业参与联邦 具有商业化潜力的研究/研发（R/R&D）。一 NIA的使命是“支持对阿尔茨海默病的研究，并促进对阿尔茨海默病的研究。 AD的病因、治疗和诊断”。拟议的发展计划是必要的 使IND能够在FDA备案和接受，这是商业化的早期前奏。 这项发展赠款的三个具体目标是： 1. SPG 302（一种新型突触发生分子）口服剂型的GMP生产。 2.开展GLP安全药理学研究，并开展GLP IND使能毒理学研究， 大鼠和Beagle犬用SPG 302处理4周 3.准备并向FDA提交成功的IND申请。 拟议的研究旨在逐步优化时间和资源 通过安全性试验，导致IND提交（对于I期单次给药剂量递增，多次给药剂量递增） 递增剂量和食物相互作用研究）。 Spinogenix将与CRO合作，收集CMC、药理学和毒理学信息 准备并向FDA提交电子IND。

Project Summary/Abstract The mission of the SBIR program is to “encourage domestic small businesses to engage in Federal Research/Research and Development (R/R&D) that has the potential for commercialization.” A mission of the NIA is to “support research on Alzheimer’s disease, and promote research on the etiology, treatment, and diagnosis of AD”. The proposed development program is necessary to enable the filing and acceptance of an IND with the FDA, an early prelude to commercialization. The three specific aims of this development grant are: 1. GMP Manufacture of an oral dosage form of SPG302, a novel synaptogenic molecule. 2. Conduct GLP safety pharmacology studies and conduct GLP IND-enabling toxicology studies in rats and Beagle dogs for 4 weeks with SPG302 3. Prepare and submit a successful IND request to the FDA. The proposed studies are designed to optimize time and resources while moving step wise through safety tests, resulting in an IND submission (for Phase 1 single ascending dose, multiple ascending dose and food interaction studies) within 24 months of the grant being funded. Spinogenix will work with a CRO to assemble the CMC, pharmacology and toxicology information and to prepare and submit an electronic IND to the FDA.