Analysis of Gene Therapy Landscape

Gene Therapy for Neuromuscular Diseases

The Client

• US-based biotech with single asset gene therapy in late-stage development for treatment of debilitating monogenetic disorder
• Planning follow-on development with same agent in analogous disease subtypes

Challenge

• Seeking to broaden its gene therapy portfolio by bringing additional assets into its pipeline

• Considering acquisition of new gene therapy technologies within the context of existing domain expertise

Approach

• Exhaustive analysis of gene therapy landscape including overview of gene transfer systems and various enabling technologies
• Interaction with key personnel at vector cores, research institutes and biotechs in order to assess licensing or M&A opportunity

Valued Outcome

• Comprehensive searchable database of gene therapy programs in academia and industry
• Facilitate discussion on gene transfer systems, platforms and most tractable indications with client and relevant stakeholders
• Client company acquired for $8.7B
• Enabled client to thoughtfully build gene therapy pipeline to increase optionality and value