A biopharmaceutical company developing novel immunomodulatory technology was exploring the potential clinical use of its lead candidate in combination with AAV based gene therapies to treat rare diseases. Alacrita was commissioned to provide an overview of the opportunity in minimising host-rejection of AAV-based gene therapies in development for 13 rare diseases. 


Alacrita conducted a deep dive into the AAV gene therapy landscape for each target indication, evaluating the technical and commercial positioning of all pipeline AAV-based gene therapy programs. We performed secondary research to understand the proportion of patients expected to generate neutralizing antibodies against the AAV vector serotypes being used in each therapy, the prospective impact of non-viral-vector based gene therapy approaches, as well as any other stand-out non gene-therapy modalities that could mitigate a clinical need for manipulating immunogenicity of the AAV vectors with immunomodulatory enzymes.

Our consultants then triaged each indication using a semi-quantitative score system for each pipeline AAV-based gene therapy program. Scores were generated based on both technical and commercial risk parameters and each program was then ranked relative to others. Using this approach, we were able to highlight programs/indications that presented the biggest unmet need for an immunomodulatory enzyme to support gene therapy, and thus we were able to select prospective partners for our client to initiate discussions with. 


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