An early-stage, venture-backed, private biotechnology company developing a novel multi-gene therapy approach to addressing chronic, age-related diseases requested an opportunity mapping assessment and target indication prioritization to identify the optimal indication strategy for development and commercialization of the therapy. Given the disruptive nature of this potential therapy, especially from a cost of care standpoint, the prioritization needed to take into account aspects like timing of therapy, burden of proof and budget impact, in additional to traditional considerations of feasibility and opportunity.


Alacrita first assessed ~50 potential target indications that could benefit from the gene therapy based upon biological rationale and US patient prevalence as scoring metrics. Indications were sorted into biological families depending on the key drivers of disease (cardiovascular, metabolic, obesity, renal/liver). Due to the innovative and potentially disruptive nature of the client’s approach, it was recognized that the indication strategy should involve a development journey from an initial “gateway” indication (high scientific rationale, low disease prevalence) through to larger biologically-related larger patient populations once POC had been demonstrated. 

Thus, we proposed a shortlist of potential ‘indication journeys’ for client consideration, in each case profiling the severity and progression of disease, the optimal stage of disease for treatment with the “preventative” gene therapy, preclinical models and likely costs to IND, current standards of care, clinical benchmarks, the estimated cost and requirements for clinical studies to POC and regulatory submission. Finally, we provided a recommendation on the best initial indication to prioritize and its associated downstream journey indications.

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