Chien-Ju Chen, Ph.D.

Program team lead - Provided end-to-end scientific leadership for gene therapy programs. Accountable for target validation strategy, translatability assessment, and data-driven go/no-go recommendations. Defined hypothesis-driven target validation frameworks integrating human genetics, transcriptomics, and disease biology to prioritize targets with clinical relevance and mechanistic clarity. - Led external collaborations by defining preclinical research plans, lead identification strategies, and data interpretation frameworks; moderated scientific decision-making in joint research committee meetings. - Presented program strategy, risks, and progress to internal leadership and governance boards, translating complex datasets into actionable development decisions. Assessed program risk across efficacy, safety, and feasibility dimensions, balancing biological rationale, model validity, and therapeutic modality constraints to guide program strategy. - Led cross-functional project teams spanning in vivo pharmacology, stem cell biology, translational medicine, and computational biology to deliver proof-of-concept and target engagement data. Owned project-level resourcing and budget planning. - Established functional assays and defined translatable efficacy endpoints and biomarker strategies across in vivo and in vitro disease models. Directed transcriptomic analyses from disease tissues and cellular models to define disease signatures, stratify phenotypes, and inform candidate advancement. - Mentored direct reports and junior scientists, aligning individual development with program objectives and organizational priorities. - Served as subject matter expert in neurodevelopmental rare diseases, shaping portfolio-level target prioritization and investment decisions through integrated assessment of biological rationale, translational feasibility, and development risk. contributing to asset evaluation and portfolio strategy teams and shaping target selection.

Lab of Dr. Mauro Costa-Mattioli Investigated the involvement of different mTOR complexes in cognition, ASD, and epilepsy using transgenic mouse models. Identified mTORC2 as the key regulator for the pathogenesis of epilepsy and ASD in the Pten-deficient mTORopathy (Cowden syndrome). - Utilized metabolomics, in vitro functional assays (Seahorse), and biochemistry to uncover that increased glycolysis regulated by mTORC2-AKT signaling as the cellular mechanism underlies epilepsy and ASD-like behavior. Developed antisense oligonucleotide (ASO) treatment targeting mTORC2 in mouse autism and epilepsy disease model lacking Pten. Key achievements: A first-author publication in Nature Medicine, two conference presentations, one poster award, one travel award. - Performed behavioral, biochemistry, and IHC assays to elucidate how different mTOR complexes regulate the formation of LTD in hippocampus. Key achievement: One co-first author publication in Nature Neuroscience. - Piloted in vivo seizure measurements for mouse models in the lab. Identified mTORC2 as a potential new antiepileptic drug target by demonstrating ameliorated seizure in mTORC2 knock-out mice. Performed EEG and MEA recording to access the ability of mTORC2-inhibiting ASO to prevent or suppress neuron hyperexcitability and seizures. Key achievements: One HIN R01 grant. One co-author manuscript under review. - Established patient-derived Down Syndrome neuron culture utilizing single-step induction method to convert hiPSC into excitatory cortical neurons. Characterized Down Syndrome hiPSC-derived neurons using immunofluorescence and western-blot.

Supervisor: Mei-Shang Ho Supported an external collaboration drug discovery project with the goal to screen naturally-occurring compounds from Curcuma Longa extracts to identify anti-diabetic and neural-protective drug candidates. - Utilized ELISA and luciferase reporter assay to screen for GLP-1 receptor activating components from plant extracts. - Performed and optimized behavior tests for memory impairments in diet-induced diabetic mice. - Examined neural death and neural proliferation in diet-induced diabetic mice. - Led a team of in vivo scientists to establish pilot efficacy study workflows including physiological (blood glucose, blood insulin, and OGTT) and neurological examinations to validate the effect of lead compounds in the mouse model.

Supervisor: Dr. Chih-Cheng Chen Molecular Pain Lab, focusing on the molecular mechanism of chronic muscle pain - Performed behavior test to measure pain level in transgenic mice and fibromyalgia disease model to uncover the anti-nociceptive effect of substance P which let to a publication in PNAS • Analyzed muscle histology samples to identify an anti-inflammatory role of ASIC3 to support a publication in Molecular Pain • Utilized multidisciplinary approach including IHC, qPCR, behavioral tests, and ELISA to investigate the neural circuit and molecular mechanism involved in chronic muscle pain-induced depression • Demonstrated the physical stiffness of culture surface had an effect on the morphology and cytoskeleton distribution in primary mouse DRG DRG neurons

Studied cellular and molecular neuroscience with a focus on the role of mTOR pathway in cognitive function, ASD, and Epilepsy.

Dissertation: Role of ASIC3 in spinal cord neuron central sensitization in chronic muscle pain and related emotional disorder