Assessment of an AAV gene therapy for an ophthalmic indication

Challenge:

Alacrita conducted an assessment of an AAV gene therapy for an ophthalmic indication, focusing on market potential, technical and regulatory issues and identification of key risks.

Solution:

Starting with the prospective licensor’s internal due diligence, Alacrita used its in-house expertise in the AAV gene therapy arena to assess the strength of the target dataset and identify key risks associated with the below:

• 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

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

Gene Therapy Consulting

Alacrita frequently supports client programs in the gene therapy space, with our expertise spanning a range of therapeutic areas and functional disciplines.