New In Vivo Data in Humanized Mouse Model Demonstrate that Prime Editing Can Correct Predominant Mutations Accounting for Approximately 60% of Patients with RHO adRP Up to 70% Precise Correction at RHO p.P23H Mutation and up to 65% at RHO p.V345L and p.P347L, Well Above Threshold Required to Stop Disease Progression in Photoreceptors No Detectable AAV Genome Integration and No Detectable Off-target Edits Observed Data Presented Today in an Oral Presentation at RD2023 CAMBRIDGE, Mass., Oct.

人源化小鼠模型的新体内数据表明，Prime编辑可以纠正主要突变，约占RHO adRP患者的60%，RHO p.P23H突变精确纠正率高达70%，RHO p.V345L和p.P347L精确纠正率高达65%，远高于阻止光感受器疾病进展所需的阈值-没有可检测的AAV基因组整合，也没有可检测的脱靶编辑-今天在马萨诸塞州剑桥市RD2023的口头报告中提供的观察数据。

24, 2023 (GLOBE NEWSWIRE) -- Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company committed to delivering a new class of differentiated, one-time curative genetic therapies, today reported new preclinical data demonstrating the ability of Prime Editors to efficiently and precisely correct the predominant mutations that cause rhodopsin associated autosomal dominant retinitis pigmentosa (RHO adRP).

242023（GLOBE NEWSWIRE）-Prime Medicine，Inc。（纳斯达克股票代码：PRME），一家生物技术公司，致力于提供一类新的分化，一次性治疗性遗传疗法，今天报道了新的临床前数据，证明了主要编辑能够有效和精确地纠正导致视紫红质相关的常染色体显性视网膜色素变性（RHO-adRP）的主要突变。

The data were presented today at the International Symposium on Retinal Degeneration 2023 Congress (RD2023) in Costa del Sol, Spain. RHO adRP is a rare inherited retinal disease that causes progressive vision loss in early adolescence, leading to eventual blindness in adulthood due to photoreceptor degeneration.

这些数据今天在西班牙Costa del Sol的视网膜变性2023大会（RD2023）国际研讨会上发表。RHO adRP是一种罕见的遗传性视网膜疾病，可在青春期早期导致进行性视力丧失，并由于感光细胞变性而最终导致成年失明。

It results from mutations in the gene RHO, which encodes rhodopsin, the light-sensitive G protein-coupled receptor involved in phototransduction in rods, a type of photoreceptor, and leads to the progressive loss of rods and, subsequently, cones in the retina. “The data presented today are the first proof-of-concept data for Prime Editing’s application in treating ophthalmological indications, and highlight the ability of Prime’s novel, dual AAV delivery platform to efficiently deliver Prime Editors to the eye,” said Jeremy Duffield, M.D., Ph.D., Chief Scientific Officer of.

它是由基因RHO的突变引起的，该基因编码视紫红质，视紫红质是一种光敏G蛋白偶联受体，参与视杆（一种感光体）的光转导，并导致视杆以及视锥细胞逐渐丧失。“今天提供的数据是Prime Editing在治疗眼科适应症中应用的第一个概念验证数据，突出了Prime新颖的双AAV交付平台有效地将主要编辑提供给眼睛的能力，”Jeremy Duffield博士，博士，首席科学官。