AAV-Based In Vivo Gene Therapy for Rare to Common Disease, and Innovative Gene Regulation Technologies

This campaign has been commissioned by an international Japanese pharmaceutical company who combat diseases using a combination of ground‑breaking technology and biology, with an extensive track record of academic collaboration. They are interested in AAV-based in vivo gene therapy through research programs for rare to common disease, and innovative gene regulation technologies.

1) AAV‑based in vivo gene therapy program for rare to common disease

Approaches of Interest:

• Therapeutic target: Mechanism of action is proven or well-understood
• Target disease:...