Strategic and Regulatory Consulting for Cell and Gene Therapy.Our mission is to accelerate the development of cell and gene therapies from preclinical stage to FDA-approved products and disease cures. Our team of experts provides preclinical, clinical, manufacturing, and regulatory support to biomedical companies and academic institutions to help you bring your ideas to IND.

• Support preclinical and clinical project management for the Center for Gene Therapy of the Hematologic Malignancies and Stem Cell Transplantation Institute at City of Hope.• Coordinate clinical trial design, writing and submission of clinical protocols, informed consent forms and Investigational New Drug (IND) for the treatment of brain cancer, HIV/AIDS or sickle cell disease using gene or stem cell therapies (e.g. CAR T cells or hematopoietic stem progenitor cells).• Collaborate with laboratory and clinical researchers to organize manuscript writing, data presentation and article submission. • Wrote two grant applications that were awarded over $7M by the California Institute for Regenerative Medicine (CIRM).• Assist with identification of funding opportunities, grant writing and submission.

The SMA Foundation is a non-profit biomedical research foundation focused on advancing the development of therapeutics for Spinal Muscular Atrophy. The Foundation is the second largest funder of SMA research worldwide (after the NIH) and has invested over $100 million in basic, translational, and clinical research.As a Scientist at the SMA Foundation, I:• Identified and evaluated new scientific and drug development opportunities with a special emphasis on muscle-enhancing therapies (anti-myostatin antibodies, SARMs, mitochondria biogenesis stimulators), medical devices, and SMA biomarkers.• Prepared materials on the therapeutic and commercial potential of SMA to convince companies developing therapies for muscle wasting disorders (Duchenne muscular dystrophy, cancer cachexia, sarcopenia, COPD or hip fracture) to repurpose their drugs.• Supported project teams by coordinating research activities both internally and externally with major pharmaceutical or biotech companies, academic institutions and CROs to move therapeutic candidates through preclinical development and potentially into clinical trial. - Designed PK/PD and efficacy studies. Performed quantitative data analysis. - Served as lead contact for collaborators including senior management and KOLs, communicating study results and providing technical expertise on preclinical studies. • Authored and reviewed MTAs, CDAs, budgets, executive summaries of project or Foundation activities used for strategy decisions. • Performed site visits and represented the Foundation at international scientific meetings.• Educated parents of children with SMA and new industry partners on the ongoing SMA clinical trials.

• Successfully developed the first fully humanized model of sporadic ALS • Discovered a necroptotic pathways that is triggered in motor neurons by sporadic and familial ALS astrocytes • Identified potential toxic factor released by toxic ALS astrocytes • Forged partnership with industry to develop AAV mediated gene therapy technology in the Motor Neuron Center,the Target ALS research initiative, and Biogen Idec• Designed and executed preclinical studies testing AAV-shRNA mediated therapy in ALS animal models• Developed behavioral paradigms (loaded grid and inverted screen tests) for assessing grip strength in ALS animal models for the Motor Neuron Center and affiliated scientists• Reviewed IACUC protocols• Scientific reviewer for “French Muscular Dystrophy Association/Telethon” research grants• Produced publications in Neuron and Medecine/Science and Cell Reports.Awards and honors: Selected to present research work at the 24th International Symposium on ALS/Motor Neuron Disease Conference in Milan (2013) and the quarterly research update at the Neurology Department Grand Rounds (2014).

• Executed in vitro drug screening to characterize the non-cell autonomous toxic mechanisms in ALS: identified key elements of the death cascade activated in motor neurons upon exposure to familial ALS astrocytes• Successfully tested the cytotoxic properties of mutant glial restricted precursor graft in the spinal cord of healthy rat (collaboration with Johns Hopkins ALS research team)• Designed and executed two preclinical studies to test the neuroprotective properties of small molecules in ALS mouse model (collaboration with the Scripps Research Institute, Florida)• Editorial reviewer for Gene, Neuropharmacology and Journal of Neurochemistry• Awarded a R21 grant as co-investigator• Supervised undergraduate students (2 years)• Produced publications in Neuron, Journal of Receptor Ligand and Channel Research and Neurobiology of DiseaseAwards and honors: Recipient of the Philippe Foundation grant for exchange programs between France and the United States (2009) and Recipient of the Keystone Symposia Scholarship for the conference Cell Death Pathways: Beyond Apoptosis in Banff, Alberta (2012). Selected to present research work at the Society for Neuroscience meeting in New Orleans (2012).

• Fully characterized the expression profile of somatostatin receptor sst2A in the developing rat and human brain and identified its role in neuronal migration.• Developed expertise in the normal and pathological brain development.• Imported cell migration assays and video microscopic technic in the laboratory.• Selected for two years as teaching fellow in Neurophysiology for Psychology Bachelor students at the Psychology Institute and was fully responsible for the content of the class being taught.• Produced four publications in PloS One, Stem Cells and Development, Developmental Neurosciences and Journal of Neuroscience and one book chapter in Cambridge University PressAwards and honors: Summa Cum Laude, Research and Merit based Fellowships from the French Ministry of Research and Education