A leading biotechnology company with a focus on development and commercialization of gene therapies for bleeding and other debilitating disorders had developed a next-generation AAV gene therapy platform. The company wanted to build its pipeline with other drugs against autoimmune and complement-mediated diseases. Along with support from investors, they were in the process of evaluating a long list of potential indications in terms of technical feasibility with their platform technology, and approached Alacrita to support additional appraisal of short-listed indications for prioritization.


Alacrita supported the client by engaging an experienced consultant with a background in building and delivering innovative therapeutic portfolios within the immunology/inflammatory disease field through both internal and in-licensed projects. Based on his expert knowledge around the scientific, clinical and commercial landscape in immunology/inflammation and respiratory diseases, we conducted an appraisal of the preclinical and early clinical path in 14 different target indications against the technical capabilities of the company's gene technology. A report was developed in PowerPoint format and presented to the board of the company.

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