相关临床试验
5
0 进行中
药物批准
0
批准总数
监管机构
0
监管机构数
成立时间
2015
终止
4
80.0%
撤回
1
20.0%
暂无批准数据
- Alltrna has received approval to begin a Phase 1 trial of AP003 in healthy volunteers in Australia, which the company describes as the first tRNA therapy to enter human clinical trials. - AP003 is a lipid nanoparticle-encapsulated tRNA delivered by IV infusion that is designed to read through premature stop signals caused by nonsense mutations. - The company targets the Arg-to-stop mutation, the most common nonsense mutation, within a universe of roughly 19 conserved stop signals that recur across many genetic diseases. - Alltrna plans an adult phenylketonuria study to show protein restoration and biomarker reduction before a basket trial in multiple inborn errors of metabolism.
- Three-year-old Oliver Chu became the first person globally to receive groundbreaking gene therapy for Hunter syndrome, a rare genetic disorder that typically causes death before age 20. - Nine months post-treatment, Oliver is producing hundreds of times the normal amount of the missing enzyme and showing dramatic improvements in speech, mobility, and cognitive development. - The Manchester-based trial involves five boys worldwide and represents a potential breakthrough for treating rare genetic conditions affecting over 3.5 million people in the UK. - The gene therapy uses modified stem cells to produce the missing iduronate-2-sulfatase enzyme, with modifications allowing it to cross the blood-brain barrier effectively.
- Aera Therapeutics appointed William Querbes, Ph.D., as chief scientific officer to lead advancement of genetic medicine delivery platforms and pipeline expansion. - Dr. Querbes brings over 20 years of experience in genetic medicines development, including leading the discovery of GIVLAARI, the first FDA-approved RNAi therapeutic using GalNAc conjugate technology. - The appointment strengthens Aera's leadership team as the company develops proprietary lipid and protein nanoparticle platforms to expand genetic medicine applications across multiple therapeutic areas.
• Pompe disease, a rare genetic disorder, is seeing advancements with over 20 therapies in the pipeline. • Key players like Amicus Therapeutics and Spark Therapeutics are developing novel treatments, including gene therapies and enzyme replacements. • Clinical trials are underway, evaluating the safety and efficacy of drugs like Cipaglucosidase alfa and SPK-3006. • These emerging therapies target various mechanisms, such as alpha-glucosidase replacement and glycogen synthase kinase modulation.
