CASE STUDIES

Roadmaps / Gap Analysis

For an academic research group developing an AAV-delivered gene therapy product for a cardiology indication, we provided a preclinical gap analysis, including CMC, regulatory, and financing strategic roadmaps, with the ultimate goal of a NewCo spinout.

Gap Analysis / Preclinical Strategy and Execution

An academic researcher developing a class of small molecule antivirals was interested in external input to advise upon the degree of readiness of the project for partnering and/or progression to IND. We conducted a thorough gap analysis of the existing in vitro data, identified the most significant gaps, and suggested critical experiments designed to de-risk the program for investment, with defined go/no-go decision points.

Gap Analysis / Landscaping

For an Asian drug discovery research center, we provided a gap analysis and technical assessment of its internal AAV biomanufacturing capabilities, a global competitive AAV manufacturing market landscape analysis, and a strategic review of the challenges or bottlenecks facing the AAV manufacturing industry, including a summary of novel technologies designed to mitigate individual bottlenecks.

Search and Evaluation

For a well-capitalized, private mid-size pharma company, we supplemented its North America scouting efforts by identifying early-stage, first-in-class molecules (large or small) with strong biology underpinning the target and clear opportunity to stratify patients based on target expression.

Search and Evaluation / In-Licensing

We supported the global asset scouting efforts of a venture-backed, private biotech company seeking in-licensing opportunities in inflammatory disease and neurodegenerative disorders by identifying early-stage opportunities for in-licensing, co-development, or acquisition, with several moving into further due diligence, and exchange of term sheets for one asset.

Due Diligence

We supported a detailed due diligence of a live, attenuated COVID-19 vaccine asset in development by one of a venture capital firm's portfolio companies interested in partnering the asset. The diligence included all preclinical, CMC and regulatory development plans and study documents. An overview of the competitive landscape was also provided to highlight differentiation of the asset. Potential risks and mitigation strategies were provided to improve the marketability of the asset.

Partnering / Asset Valuation

A Venture fund client had identified a novel Phase 1-ready asset for treating a rare CNS infectious disease and was interested in licensing the asset and founding a new company to further its development. We led a triage due diligence (DD) to determine if a full DD was warranted, and potentially a subsequent full DD effort to identify any significant risks, to provide strategies for mitigating those risks, and to provide a recommendation on whether to pursue further licensing discussions. Based on the outcome of the due diligence, we provided an rNPV valuation of the asset and license negotiation support.

Indication Opportunity Mapping / Prioritization

For a private venture capital firm seeking a strategic development plan for a new portfolio company, we characterized the technical feasibility and commercial attractiveness for the NewCo’s oncology small molecule asset, and provided a therapeutic area prioritization.

Indication Opportunity Mapping

BioHorizon provided several new indication opportunity assessments to guide the selection of a new therapeutic area by the executive team of a public, mid-sized pharmaceutical company developing small molecule therapies in the infectious disease space. The assessment included an analysis of the competitive intensity, regulatory path, commercial attractiveness, technical feasibility, and market opportunity for each indication.

Indication Opportunity Mapping / Prioritization

For an early-stage, venture-backed, private biotechnology company developing a novel multi-gene therapy approach to addressing chronic, age-related diseases, we provided an indication opportunity mapping assessment and target indication prioritization to identify the optimal strategy for development and commercialization of therapies for primary and follow-on indications.