来自Spirovant Sciences的Katherine Excoffon和合著者描述了SP-101，一种AAV基因治疗载体，已被优化用于有效的人类气道细胞转导，并且包含一个功能性和可调节的缩短的人类CFTR迷你基因(hCFTRΔR)。研究人员发现，hCFTRΔR mRNA在接受单次吸入阿霉素(一种AAV转导增强剂)的雪貂 ...

该研究证明，FAST-PLV作为核酸递送平台，可以在体外和体内有效递送和表达mRNA和DNA ... Entos公司CEO John Lewis 表示，AAV和LNP是目前基因药物的主要递送 ...

不过，恩泽康泰CTO赵立波博士指出，外泌体药物递送的学术研究从2023年下旬进入了一个全新的阶段：外泌体创新疗法药效成分的多样性得以展示，例如在递送mRNA和AAV方面都有重磅的研究成果得以发表；另一方面，在明确了外泌体给药系统中的技术壁垒后 ...

“华兴资本医疗与生命科技行业周报”定期发布，专注从资本角度解读一周数据，并提供最新行业观察。医疗与生命科技是华兴资本多年来关注并深耕的领域，目前已为近200个融资及并购项目担任财务顾问。医疗与生命科技行业创新不断涌现，已经发展成为最活跃的行业之一，华 ...

Exosomal mRNA expression patterns were significantly altered in patients with MPO-AAV compared to healthy controls.

The successful mRNA vaccine efforts have spurred a new generation ... for clinical translation of these novel LNP formulations. AAV-based therapies, by contrast, have shown the potential for ...

mRNA technology was quickly adopted in the COVID-19 vaccine race and has undoubtedly proven its worth. Less than a year after the emergence of COVID-19, an entirely new type of vaccine based on ...

“Almost all gene therapies approved today are based on the AAV virus but the big caveat is that you need to give really high ...

Exciting results for MeiraGTx Holdings plc's GAD gene therapy for Parkinson's Disease, with promising phase 1/2 data and ...

This title is part of a longer publication history. The full run of this journal will be searched. TITLE HISTORY A title history is the publication history of a journal and includes a listing of the ...

On Thursday, Cathie Wood-led Ark Invest made a significant move by offloading shares of Moderna Inc (NASDAQ:MRNA). This decision comes amidst a backdrop of mixed analyst sentiments and Moderna’s ...

Moderna, Inc. engages in the development of transformative medicines based on messenger ribonucleic acid (mRNA). Its product pipeline includes the following modalities: prophylactic vaccines ...