Assessment of an AAV gene therapy for an opthalmic indication

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

  • 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.

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