Previous efforts to circumvent the problem of the size limitation imposed by the AAV vector have focused mainly on altering the candidate genes into ‘mini-expression cassettes’ suitable for ...

The majority are in the very early preclinical stage of development, but applications for regulatory approval have been ...

While the company is prioritizing RJVA-001 and a lead program in obesity, it's pausing some R&D efforts and laying off 17 percent of its workforce.

Viralgen and Axovia Therapeutics are set to form a partnership to progress the development and manufacturing of an ...

Viral vectors introduce genes and gene editing sequences into cells, and are a popular platform for gene therapy due to their safety and efficacy features. Adeno-associated viral (AAV) vectors in ...

That therapy uses an adeno-associated virus (AAV) as a vector rather than a lentivirus, a vector type that features in approved gene therapies such as Roche's Luxturna and Novartis' Zolgensma.

First demonstration of single AAV muscle editing in non-human primates: NanoCas achieved in vivo editing efficiencies of up to 30% when targeting dystrophin in the skeletal muscle of cynomolgus ...

Genethon & Eukarÿs enter strategic partnership to develop a breakthrough technology to reduce the biomanufacturing cost of gene therapies: Evry, France Friday, January 24, 2025, ...

To address these challenges, researchers are developing novel viral vector designs, such as self-inactivating vectors, tissue-specific promoters, and targeted gene editing approaches. Additionally, ...

New Vericheck ddPCR Empty-Full Capsid Kits for AAV Serotypes 2 and 8 Provide Precise Measurement of Viral Titer and Capsid ...

Merck's expertise spans viral vector types including adeno-associated virus, lentivirus and adenovirus. The company provides contract testing services and extensive contract development and ...