Certara was approached by a private gene therapy company specializing in the development of RNA carriers. Their therapeutic areas of interest are antivirals, cancer immunotherapies, and gene & cell therapies. The client had licensed a highly safe and easy-to-use lentiviral vector technology that can efficiently deliver RNAs in vitro and in vivo. However, the company had not yet identified therapeutic indications to target.
Given the complexity and breath of the project, we suggested a multi-step secondary research approach. Our teams compiled a list of over 200 indications in which transient RNA expression can lead to a therapeutic effect; each indication was then assessed on eligible population, severity and unmet need, and feasibility of clinical proof of concept. We selected the top 25 “gateway” and “value” indications and then further evaluated for feasibility using a set of 9 sub-criteria and competitive intensity. A Target Product Profile (TPP) was created for each final selected indication, comparing the lentiviral product to the most relevant comparator, including development of the most impact value proposition in each instance.
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