Cirrus’ lead program is a novel AAV gene therapy designed to restore IRAK-M, a central regulator of the eye’s immune homeostasis, so as to ...

A new study in Human Gene Therapy describes a machine learning (ML) model that can be used as a surrogate for laborious in vitro experiments. This in silico approach aims to increase the fitness of ...

Gene therapy (introducing genetic material into living cells to fix, replace, enhance, or block a faulty gene) is rapidly gaining traction as a strategy for the treatment of genetic diseases. The ...

The US Pharmacopeia (USP) has recognized mass photometry as a key orthogonal method for characterizing its new adeno-associated virus (AAV) reference standards, as referenced in the draft general ...

Groundbreaking study demonstrates that targeted oligodendrocyte gene therapy leads to decreased N-acetylaspartate (NAA) levels and increased ...

WALTHAM, Mass.--(BUSINESS WIRE)--Affinia Therapeutics (“Affinia”), an innovative gene therapy company with a pipeline of first-in-class and/or best-in-class adeno-associated virus (AAV) gene therapies ...

It will also make preclinical gene therapy studies in mice more efficient and reproducible, given that a lot of the homework on the best AAV for a given cell type has already been done." This work was ...

“We’re excited to share our latest data with the scientific community at ASGCT, which remains the most prestigious venue for showcasing gene therapy innovations,” said Dr. Nicole K. Paulk, PhD, Siren ...

PHILADELPHIA--(BUSINESS WIRE)--Latus Bio, Inc. (Latus), a biotechnology company pioneering advances in tissue-targeted AAV gene therapy, today announces the publication of a seminal research ...

In a preclinical study, UC Davis Comprehensive Cancer Center scientists developed a highly targeted gene therapy that could revolutionize treatment for cancers linked to a common herpesvirus, with ...

At a median 13 years after a single infusion of AAV-mediated gene therapy, factor IX expression remained stable in 10 patients with severe hemophilia B. The median annualized bleeding rate decreased ...