Focus Area Advanced Therapeutics

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


  • 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


  • 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