Clinical Validation, TPP Refinement and Value Assessment of iPSC derived Cell Therapies

The Client

• Venture-backed preclinical biotech with a universal allogenic cell platform focused on non-oncology.

• Client was seeking to understand the market opportunity and value proposition for their regenerative cell therapy in two highly prevalent, yet very different, indications of Heart Failure (HF) and Parkinson’s Disease (PD).

Challenge

• Addressing development risk for RegMed approaches to HF. It was crucial to elucidate the critical differences in our client’s platform from prior approaches and incorporate lessons learned from trial failures.
• Mapping how client’s PD program could compete in future landscape potentially impacted by gene therapy.

Approach

• KOL engagement helped to clarify how PD and HF patients are presently segmented and staged for treatment. Additional thought leader insight was gathered regarding present and future unmet medical needs and where regenerative advanced therapies would likely be positioned to address those clinical shortcomings.

Valued Outcome

• Clear understanding of how their cell therapy platform is differentiated from competing programs, identified which target patient segments are most likely to realize a clinical benefit, and developed well-supported estimations of respective market sizes.
• Company acquired for $2B by a big pharma company.