Commercial Strategy

Gene Therapy Early Development & Launch Strategy

Rare Disease
Clinical Stage
Top 10 Pharma

Business Needs

Following the acquisition of a gene therapy portfolio, our client began immediate preparation for an accelerated launch in two rare disease areas. Facing fierce competition and significant uncertainty for orphan gene therapies, the client retained Acsel to provide a structured approach to address the key business questions and plan strategically.


Acsel collaborated with the client team to co-create the initial strategic plans for two disease areas.

  • Market Definition: We detailed the current and future market landscape, identified where to prioritize efforts within each disease area, and recommended opportunities to provide differential value relative to the competition
  • Competitive Scenario Planning: We scrutinized multiple development plans and strategized against competitors earliest potential approvals
  • Launch Plans: We recommended critical launch activities to ensure speed to market and an effective launch


Acsel’s guidance provided the client with a solid foundational understanding to drive strategic decisions. We evaluated 200 activities across 7 functions to accelerate launch to achieve first-to-market status and to plan for a potential competitive marketing scenario.

In the next phase of our engagement, Acsel will conduct qualitative and quantitative research with selected stakeholders across seven markets to gather insights against remaining key questions, prioritize leverage points to inform strategic focus, and craft stakeholder behavior objectives to outline changes required to succeed in the marketplace.