Amid a reckoning for the gene therapy field, Vertex Pharmaceuticals has joined a growing list of companies paring back their ...

Liver-directed AAV-based gene therapy offers promise for people living with hemophilia B or other genetic disorders. 6,7,11–18 However, the long-term safety and efficacy of this approach remain ...

such as the dorsal root ganglia or the liver, which may help avoid toxicities commonly associated with AAV delivery. Using TRACER, Voyager is also identifying capsids with the capacity for strong ...

An “inverse vaccine” that delivers deactivated allergens to the liver was able to reduce reactions in mice with allergic asthma for at least a year after treatment. Should the approach ...

Removing EPO signaling in mouse liver tumors leads to tumor regression along with sensitivity to immunotherapy.

AAV4 also mostly avoids the liver, a major target organ for most ... Walkey et al, A comprehensive atlas of AAV tropism in the mouse, Molecular Therapy (2025). DOI: 10.1016/j.ymthe.

Blocking the activity of the protein turns formerly “cold,” or immune-resistant, liver tumors in mice into “hot” tumors teeming with cancer-fighting immune cells. When combined with an ...

'Cold' tumors are resistant to common immunotherapies. Researchers have uncovered a master regulator that can be manipulated to prevent tumor growth in mice. A protein identified nearly 40 years ...