Richie Khanna, Ph.D

Bringing 25 years of executive leadership experience to non-clinical drug development and helping clients achieve their IND, NDA, and BLA goals. Worked extensively on small molecules, biologics, and gene therapy modalities in neurodegenerative, neuromuscular, ophthalmic, cardiovascular, and lysosomal storage disorders space, and providing the following expertise: # Nonclinical gap analysis with risk assessments and building an IND roadmap for nonclinical regulatory strategy # Authoring of nonclinical pharmacology summaries required for Module 2 for IND submission as well as IND-enabling preclinical research study reports (non-GLP and GLP) required for Module 4 for IND submission #Authoring of non-clinical sections for IBs, DSURs, Type C and B briefing books, ODD, PRIME, PIP, NDA, and BLA # Providing strategic leadership and project management role for the non-clinical drug development program # Supporting design of IND-enabling studies in compliance with FDA regulations (expertise in designing and conducting pharmacokinetics, pharmacodynamics, efficacy, and GLP toxicology studies) # Identifying, selecting, and managing CROs for in-life of IND-enabling studies (non GLP and GLP) # Identifying, selecting, and managing CROs for analysis of animal samples from IND studies including development, qualification and validation of assays (expertise in managing qPCR, ddPCR, Westerns, ELISA, MSDs, LCMS, immunohistochemistry, immunofluorescence assays etc.) # Translating nonclinical to clinical pharmacology and bioanalytical science including the strategic design for phase-appropriate endpoints, selection of doses, bioanalytical assays, biomarkers etc. for clinical studies.

Coordinated and managed strategic activities/initiatives for CMO’s Clinical Development Organization by work closely with CMO and SVPs/VPs and conducted leadership and Sr. staff meetings geared towards discussing high priority activities/issues/risks etc. to facilitate strategic decisions. Supported review of key clinical documents for critical scientific/analytical input in support of Regulatory submissions. Chaired non-clinical Pharmacology/Toxicology sub-matrix team meetings and worked cross-functionally with Science, Toxicology, Translational, CMC, and Regulatory Departments to brainstorm strategic discussions on non-clinical studies required for early clinical development of gene therapy programs.

Built and managed In Vivo Pharmacology Department of 10-12 scientists at varying levels of Bachelors, Masters, and PhD degrees, running comprehensive drug development from in-life to bio-analysis, including biochemical, histological and functional assays, in disease-relevant rodent models. Led design, implementation, coordination, and analysis of all non-clinical studies in support of Pharmacokinetic, Pharmacodynamic, and efficacy evaluation of lead candidates geared towards mechanism of action and proof of concept.

• Director, Amicus Therapeutics, Cranbury, NJ (2014-2018) • Principal Investigator, Amicus Therapeutics, Cranbury, NJ (2010-2014) • Sr. Research Investigator, Amicus Therapeutics, Cranbury, NJ (2006-2010) • Research Scientist, Amicus Therapeutics, New Brunswick, NJ (2005-2006) • In vivo Biologist, temp, Amicus Therapeutics, New Brunswick, NJ (2004-2005)

Post Doctoral research involved in identification and characterization of role of Poly(A) binding protein and human decapping enzyme (hDcp2) in regulation of mammalian gene expression.

Post Doctoral research involved in development of RBC membrane protein "dematin" and "calpain" (cysteine Protease) knockout mice and identification of physiological significance of these proteins in stability of cytoskeleton.