Assessing an AAV gene therapy for an ophthalmic indication

Challenge:

We were asked to conduct an assessment of an AAV gene therapy for an ophthalmic indication, focusing on market potential, technical and regulatory issues and the identification of key risks. The resulting assessment would be used by the client to decide whether to proceed with a proposed transaction.

Solution:

Starting with the prospective licensor’s internal due diligence, we harnessed our considerable in-house expertise in the AAV gene therapy arena to assess the strength of the target dataset and key risks associated with the:

• scientific rationale and preclinical proof-of-concept dataset
• preclinical development package
• manufacturing/CMC approach for clinical trials and beyond
• clinical development strategy
• competitive position vs. current and pipeline products
• market potential

We concluded that key risks were associated with the potential longevity of the therapeutic effects and the proposed powering of the Phase III trial. As a result, the client did not proceed with the transaction.