Chameleon Biosciences, a gene therapy ... whether an AAV-based therapy targets the kidney, the liver, the lung, the retinal pigment epithelium or the heart. The clinical challenges associated ...

and are a popular platform for gene therapy due to their safety and efficacy features. Adeno-associated viral (AAV) vectors in particular have high transduction rates and reduced toxicity concerns ...

A new, streamlined AAV purification process that is both universal and scalable could ease commercialization constraint.

DOI: 10.1056/NEJMoa2410597 Johnny Mahlangu, Could Lentivirus Overcome the AAV Gene-Therapy Challenges in Hemophilia A?, New England Journal of Medicine (2024). DOI: 10.1056/NEJMe2414214 ...

Dyno was founded in 2018 and applies artificial intelligence (AI) and quantitative in vivo experiments to address gene therapy’s delivery challenge. Naturally occurring AAV capsids lack precise ...

It includes AGTC’s lead candidate AGTC-501 (laruparetigene zosaparvovec), an AAV gene therapy for X-linked retinitis pigmentosa (XLRP), which results from mutations in the RPGR gene, causing ...

During her talk, Ms. Bhattacharya will provide a corporate update on Nanoscope's recent and upcoming progress across pipeline programs. She will specifically cover Nanoscope's lead asset MCO-010, ...