Challenge:
An early-stage biotechnology startup company reached out to Alacrita for critical support of its two lead AAV-delivered gene therapy programs. As a virtual company, the client needed expert analysis of ongoing experimental research and development planning of future preclinical PoC studies conducted by CROs and academic collaborators. The client also requested support for planning CMC and regulatory activities needed to progress these programs to IND.
Solution:
An Alacrita consultant worked closely with gene therapy, regulatory, and CMC consultants from Alacrita’s extensive expert network. We provided the client with ongoing technical data review, analysis, and preclinical development recommendations for both gene therapy programs including identification, timing, and design of preclinical animal model studies (mice, NHPs, etc); efficacy, biodistribution, safety, and toxicology studies; identification of risk factors and mitigation strategies; research timelines with go/no-go decision points; coordination of studies with CROs and academic collaborators to drive completion of translational studies; and presentations of scientific data to potential investors. Alacrita CMC and regulatory experts also assembled a CMC and regulatory roadmap for the client’s two gene therapy programs through preclinical development and into clinical development. The roadmaps highlighted exactly what needed to be done for each program, in what sequence, to allow success at key milestones.
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