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Acquisition Guidance for a Novel Gene Therapy

CBPartners conducted a business development assessment to understand the pricing and market access opportunity for a spinal muscular atrophy gene therapy.


To provide Business Development and Licensing (BD&L) support by assessing the market landscape, product value, and resulting pricing and access potential for PRODUCT X gene therapy launch scenarios in spinal muscular atrophy (SMA).



Conduct payer and KOL in-depth interviews (IDIs) to understand clinical value perceptions of PRODUCT X and its associated Pricing and Market Access (P&MA) opportunity across key European markets, and evaluate whether PRODUCT X represents a good investment for the client seeking to expand their cell and gene therapy portfolio.


  • Moderator guides and research stimuli to support primary research interviews across a range of SMA expert types
  • Tailored recommendations on PRODUCT X’s optimal pricing and access outcomes across EU-3 markets to support go / no-go decision-making process

Illustrative Outputs


CBP provided acquisition guidance for a novel SMA gene therapy based on pricing and market access insights in a high unmet need, pediatric, orphan space.