Webinar Bios: Public-Private Partnerships (PPPs) for Drug Discovery


Bios for CDD Town Hall: Public-Private Partnerships (PPPs) for Drug Discovery

Ronald N. Margolis, Ph.D. NIDDK
Program Director (Contractor), Division of Diabetes, Endocrinology, and Metabolic Diseases


Ron is the program director of the Endocrinology and Hormone Signaling Program– responsible for scientific oversight and management of approximately 100 grants relating to the nuclear hormone receptor superfamily, a unique class of proteins that helps regulate an array of molecular functions, such as homeostasis, reproduction, development, and metabolism, through the interaction and control of gene expression.

Dr. Margolis’ interests include supporting research that advances control over these major diseases through manipulation of gene expression and transcription factors using drug therapy. Some research topics covered under in Ron’s portfolio include signal transduction and regulation of gene expression, structure and function of the steroid hormones, receptor structure, interaction with cytoplasmic chaperones and ligands, nuclear translocation, and mediator proteins.

Ron serves as project scientist for the Nuclear Receptor Signaling Atlas (NURSA) initiative. The NURSA initiative is a web-accessible bioinformatics resource that provides current and emerging data on nuclear receptor structure, function, and role in disease that is organized into user-mineable forms.

As the NIDDK liaison to the BD2K Executive Committee – he is a Co-lead for the Data Discovery Index to develop the means for finding, accessing, interoperating, and re-using biomedical big data.

Co-lead for the Common Fund project to “Illuminate the Druggable Genome” designed to accrue and develop information about the unannotated genes within the druggable genome (http://commonfund.nih.gov/idg/index).

Various Committee Memberships include:

  • NIH Common Fund Big Data Working Group Member
  • NIH Common Fund Project Team, Illuminating the Druggable Genome Chair
  • NIH Common Fund Project Team, LINCS Consortium Member
  • BD2K Executive Committee Member
  • NIH Common Fund Project Team and Steering Committee, Molecular Libraries Screening Centers Member
  • BD2K Data Discovery Index Working Group Member
  • Nuclear Receptor Signaling Atlas Cooperative Agreement Consortium Project Scientist
  • Steering Committee, Nuclear Receptor Signaling Atlas Member
  • Federal Working Group on Bone Diseases Member

James A. Hendrix, PH.D.
Director, Global Science Initiatives
Alzheimer’s Association


James A. Hendrix, Ph.D., is director of global science initiatives at the Alzheimer’s Association.

As a member of the Medical and Scientific Relations Division, Jim provides leadership on specific domestic and international efforts focused on advancing the division’s science agenda.

A critical element of his role is to manage industry consortia such as the Alzheimer’s Association Research Roundtable (AARR); lead the Global Biomarker Standardization Consortium; manage and direct future meeting efforts and output; and assist with the coordination of the $100 million dollar Imaging Dementia—Evidence for Amyloid Scanning (IDEAS) Study on the clinical usefulness of amyloid PET imaging.

Dr. Hendrix received his Ph.D. and a postdoctoral fellowship from Colorado State University. Before joining the Alzheimer’s Association, Dr. Hendrix was a pharmaceutical scientist with a focus on drug discovery for CNS diseases. Dr. Hendrix spent 18 years working at Sanofi-Aventis and predecessor companies, where he rose to level of senior director, U.S. site head for CNS research. He also spent two years working in the biotech industry with various companies, including Oligomerix, which is focused on tau for the treatment of Alzheimer’s disease.

The Alzheimer’s Association is the leading voluntary health organization in Alzheimer’s care, support and research. Our mission is to eliminate Alzheimer’s disease through the advancement of research, to provide and enhance care and support for all affected, and to reduce the risk of dementia through the promotion of brain health. Our vision is a world without Alzheimer’s.

Jair Lage de Siqueira-Neto, Ph.D.
Skaggs School of Pharmacy and Pharmaceutical Sciences


Research Summary: Drug Discovery, High- Throughput, High-Content Screening
Dr. Siqueira-Neto’s research has focused on development and implementation of screening assays to identify active molecules and natural products against parasites causing neglected tropical diseases.

Academic Achievements
Education: B.S. in Biological Sciences (2003) University of Campinas, SP – Brazil; Ph.D. in Molecular Genetics (2007) University of Campinas, SP – Brazil; Postdoctoral Fellow (2007-2009) Institut Pasteur Korea – South Korea.

Awards and Honors: CAPES Doctorate International Internship Scholarship (2005); DNDi Partnership of the Year, New Delhi, India (2010).

Leadership Experience: Team Leader and Drug Discovery Program Coordinator at Institut Pasteur Korea (2009-2012); Kinetoplastida Core Director from the Center for Discovery and Innovation in Parasitic Diseases, UCSF & UCSD (2013-present); Assistant Adjunct Professor at Skaggs School of Pharmacy and Pharmaceutical Sciences, UCSD (2014-present).

Key Contributions to Pharmaceutical Sciences:

  • Development of the first High-Content Screening for Leishmania and Trypanosoma cruzi

Among Neto’s recent publications:

  • Dossin et al. (2008) Automated nuclear analysis of Leishmania major telomeric clusters reveals changes in their organization during the parasite’s life cycle. PLoS One. 3(6):e2313.
  • Siqueira-Neto et al. (2010) Antileishmanial high- throughput drug screening reveals drug candidates with new scaffolds. PLoS Negl Trop Dis. 4(5):e675.
  • Freitas-Junior et al. (2012) Visceral leishmaniasis treatment: What do we have, what do we need and how to deliver it?International Journal for Parasitology: Drugs and Drug Resistance. 2:11–19.
  • Siqueira-Neto et al. (2012) An Image-based High- Content Screening Assay for Compounds Targeting Intracellular Leishmania donovani Amastigotes in Human Macrophages. PLoS Negl Trop Dis. (6):e1671.
  • Moon et al. (2014) An Image-Based Algorithm for Precise and Accurate High Throughput Assessment of Drug Activity against the Human Parasite Trypanosoma cruzi. PLoS One. 9(2):e87188.
  • Calvet et al. (2014) 4-aminopyridyl-based CYP51 inhibitors as anti-Trypanosoma cruzi drug leads with improved pharmacokinetic profile and in vivo potency. J Med Chem. 2014 Aug 7. [Epub ahead of print]

Potential Collaborative Programs with the Pharmaceutical Industry

  • Drug discovery for neglected tropical diseases
  • High-Throughput, High-Content Screening assay development