An academic group was interested in developing and commercializing an AAV vector-delivered gene therapy for a cardiac indication through the formation of a NewCo. They required assistance with a technical review of their preclinical program, including development recommendations, a CMC and regulatory strategic roadmap, and recommendations for securing financing for the NewCo.
Alacrita consultants evaluated the client’s existing research program and provided a comprehensive GAP analysis, including risk factors and mitigation strategies. Recommendations were provided for vector optimization to improve efficacy and regulatory compliance. Alacrita conducted a thorough review of AAV capsid serotypes to identify the most appropriate capsids based on tropism and safety. In addition, recommendations were provided for the most appropriate preclinical animal models, including efficacy, biodistribution, safety, and toxicology. Alacrita consultants and outside experts ultimately supplied a preclinical development plan, including general timelines for completion, as well as regulatory, CMC, and financing strategic roadmaps.
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