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Validation strategies for novel CNS targets: From molecular mechanism to therapeutic candidate

04 May 2026
Cell Therapy Manufacturing
  • What experimental designs are required to transform novel findings into robust preclinical datasets that withstand rigorous scrutiny regarding clinical translatability?
  • How can research programs simultaneously advance novel mechanistic hypotheses while satisfying the rigorous demands for independent validation and toxicological safety?
  • What are the prevalent statistical or experimental shortcomings in early-stage data packages that typically lead to failure during external scientific diligence?
  • How can preclinical studies be designed to demonstrate that a novel target offers a distinct biological impact or improved efficacy profile relative to established therapeutic modalities?
Industry Expert
Anka G. Ehrhardt, Director, Cell-Based Assays | Analytical Research & Development - Merck