A privately-held biotechnology company developing and exploiting a proprietary technology platform for optimizing and controlling gene therapy expression was looking to evolve its business model, including examining the potential for the company to build its own internal cell and gene therapy pipeline.  The board and main investor had decided to prioritize review of an idea of exploitation of the company’s existing technology in developing a novel gene therapy for a rare cardiovascular condition. Alacrita was asked to develop an outline Target Product Profile (TPP) for a gene therapy for this particular condition.


Alacrita first created a hypothetical TPP based on the technology’s capabilities, which guided commercial, technical and clinical analysis. The TPP addressed factors such as target patient population(s); dosing, format and delivery method; standard of care displaced and unmet needs (disease modification and symptom control); clinical differentiation; prospective pricing; and market access considerations and requirements. Where relevant, we defined criteria for a base case for a minimally viable product and an upside case. This draft TPP was created initially from focussed secondary research, covering published academic literature, competitor disclosures and clinical trial protocols and Alacrita’s internal databases. We then tested, refined and amplified the draft TPP through interviews with KOLs and payers in EU and US. The deliverable was presented to the board.

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