Program

 

Tuesday, May 4, 2021 – all times in US Eastern Time

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Session/Lecture

11:00 AM ET
Opening Remarks and ADDF Funding Opportunities
11:15
KEYNOTE: Delivery of Protein Therapeutics to the CNS
11:55
SESSION 1: EMBARKING ON A DRUG DISCOVERY CAMPAIGN
11:55
Session Overview
12:00 PM
What Makes a Good Translational Drug Target?
12:25
CROs to Outsource Pharmaceutical Research Services
12:50
Assembling an Interdisciplinary Team
1:15
PANEL DISCUSSION
1:35
Break, Short Presentations and NIA Funding Opportunities
2:00
SESSION 2: REPURPOSING AND REPOSITIONING
2:00
Session Overview
2:05
Challenges to Create a Drug Repurposing Library and Its Application to the Treatment of AD
2:30
Disadvantages (and Advantages) of Drug Repurposing
2:55
From Reformulation to Toxicology
3:20
IP Strategy and Commercial Consideration in Repurposing and Repositioning Programs
3:45
Panel Discussion
4:05
Short Presentations and Networking
5:00
End of Day 1

Chair/Speaker

11:00 AM ET
Howard Fillit, MD - Alzheimer’s Drug Discovery Foundation Read more
11:15
Matthew Troyer, MD - Denali Read more
11:55
SESSION 1: EMBARKING ON A DRUG DISCOVERY CAMPAIGN
11:55
CHAIR: D. Martin Watterson, PhD - Northwestern University Read more
12:00 PM
Rouba Kozak, PhD - Takeda Read more
12:25
Tilmann Brotz, PhD - PharmaDirections Read more
12:50
Susan Catalano, PhD - Cognition Therapeutics Read more
1:15
Chair and Presenters
1:35
Break, Short Presentations and NIA Funding Opportunities
2:00
SESSION 2: REPURPOSING AND REPOSITIONING
2:00
CHAIR: Leticia Toledo-Sherman, PhD - Rainwater Charitable Foundation Read more
2:05
Arnab Chatterjee, PhD - Calibr Read more
2:30
Clive Ballard, MD - University of Exeter Read more
2:55
Sharon Rosenzweig-Lipson, PhD - AgeneBio Read more
3:20
Rob Berman, MD - BioHaven Pharmaceuticals Read more
3:45
Chair and Presenters
4:05
Short Presentations and Networking
5:00
End of Day 1

Wednesday, May 5, 2021 – all times in US Eastern Time

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Session/Lecture

11:00 AM ET
KEYNOTE:Epigenetic Treatments for Neurodegenerative Diseases – What Can We Learn from Oncology
11:40
SESSION 3: BIOMARKERS TO ENABLE PRECLINICAL AND CLINICAL PROOF OF CONCEPT STUDIES
11:40
Session Overview
11:45
Target Engagement and Translatability in Preclinical Models of CNS Disease
12:10
Failing Fast: Small Trials to Prove Efficacy
12:35
A Path Toward Precision Medicine in Alzheimer's Disease
1:00
PANEL DISCUSSION
1:20
Break, Short Presentations and NINDS Funding Opportunities
1:45
SESSION 4: CASE STUDIES - CHASING NEW PATHWAYS
1:45
Session Overview
1:50
Design and Optimization of Oligonucleotide Modulators of RNA for the Treatment of Neurodegenerative Diseases
2:15
From Academic Discovery to Commercial Potential: HDI Case Study
2:40
Small Molecules to Modulate Autophagy and Lysosomal Biogenesis in LBD and AD
3:05
Protein Aggregation in LBD, AD and FTD
3:30
PANEL DISCUSSION
3:50
Concluding Remarks
4:00
Short Presentations and Networking
5:00
End of Workshop

Chair/Speaker

11:00 AM ET
Susan Bates, MD - Columbia University Read more
11:40
SESSION 3: BIOMARKERS TO ENABLE PRECLINICAL AND CLINICAL PROOF OF CONCEPT STUDIES
11:40
CHAIR: Edward Spack, PhD - Vector BioSolutions/Therini Read more
11:45
Darrell Henze, PhD - Merck Read more
12:10
Michael Gold, MD - AbbVie Read more
12:35
Philip Scheltens, MD - VUMC Alzheimer’s Center Read more
1:00
Chair and Panelists
1:20
Break, Short Presentations and NINDS Funding Opportunities
1:45
SESSION 4: CASE STUDIES - CHASING NEW PATHWAYS
1:45
CHAIR: Andrew Pieper, MD - Harrington Discovery Institute Read more
1:50
Siew Peng Ho, PhD - Harrington Discovery Institute Read more
2:15
Travis Dunckley, PhD - Arizona State University Read more
2:40
Toren Finkel, MD, PhD - Generian Read more
3:05
Jamie Bilsland, PhD - AstronauTx Read more
3:30
Chair and Panelists
3:50
4:00
Short Presentations and Networking
5:00
End of Workshop

REGISTRATION IS FREE!

SCIENTIFIC ADVISORY COMMITTEE

Howard Fillit, MD—ADDF
Mark Forman, PhD—ADDF
Suzana Petanceska, PhD—NIH/NIA
Andrew Pieper, MD—Harrington Discovery Institute
Edward Spack, PhD—Vector BioSolutions/Therini
Leticia Toledo-Sherman, PhD—Rainwater Charitable Foundation
Alessio Travaglia, PhD—ADDF
D. Martin Watterson, PhD—Northwestern University

CONFERENCE INTRODUCTION

Tuesday, May 4, 2021
11:00 US Eastern Time

Howard Fillit
Alzheimer’s Drug Discovery Foundation

 

Dr. Howard Fillit is ADDF’s Founding Executive Director and Chief Science Officer. His introduction will provide a welcome to our 15th Annual Drug Discovery for Neurodegeneration Workshop, a brief update on ADDF’s recent progress and will highlight the framework for this year’s meeting.

KEYNOTES

Matt Troyer
Denali

Bio

Matt Troyer, MD, leads Early Clinical development at Denali and is responsible for overseeing a team of physician-scientists who execute Denali’s early clinical programs in neurodegenerative disease. The Early Clinical group partners with cross functional teams to design biomarker-driven clinical development strategies and conducts first-in-human studies through investigations in Alzheimer’s disease, Parkinson’s disease, ALS and rare neurodegenerative disorders.

Dr. Troyer is a neurologist with subspecialty training in Parkinson’s disease and Movement Disorders and 15+ years of clinical development experience. Matt received a BA in biology from Illinois Wesleyan University and an MD from Stanford University. He completed neurology
residency training at Harvard Medical School in the Longwood Area Program and completed a fellowship in Parkinson’s disease and Movement disorders at the Institute of Neurology, Queen Square, London. Following his clinical fellowship, Matt did a postdoctoral research fellowship in the laboratory of Robert Edwards at UCSF, focusing on basic mechanisms of presynaptic function and Parkinson’s disease. Matt subsequently joined the Neurology faculty at UCSF with both clinical and research responsibilities. Matt later moved to industry where he led early stage neuroscience programs, both at Merck as Executive Director and Neuroscience Therapeutic Area Lead for Early Clinical Development, and then at Medivation, prior to joining Denali in 2016.

Susan Bates
Columbia University

Bio

Dr. Susan Bates received her MD degree from the University of Arkansas School of Medicine. She completed her clinical training in internal medicine at Georgetown University in Washington, DC, and in medical oncology at the National Cancer Institute (NCI) in Bethesda, MD. Dr. Bates was a Lead Clinical Investigator and Head of the Molecular Therapeutics Section in the Developmental Therapeutics Branch of the Center for Cancer Research before moving to Columbia University in August 2015.

During her years at the NCI, Dr. Bates led a highly successful translational research program focused on mechanisms of multidrug resistance and approaches to evaluate and improve the activity of epigenetic modifying agents. Her laboratory was among the first to clone the multidrug transporter ABCG2, eventually characterizing its function and its role in chemo-resistance and chemo-protection. This effort built upon earlier work elaborating the role of the multi-drug transporter P-glycoprotein that had defined the drug sensitivity profiles of cell lines in vitro, particularly in the NCI-60 cell line panel. The latter observation continues to impact how the NCI-60 cell line panel is used in drug discovery, and helped her identify a novel agent, at that time known as depsipeptide. Dr. Bates brought this drug to the clinic and after completing its phase I testing, served as Principal Investigator of a multi-institutional, international Phase II study of romidepsin (depsipeptide) in cutaneous and peripheral T-cell lymphoma. Working with Gloucester Pharmaceuticals, the data from this study were included in New Drug Applications (NDA) to the U.S. Food & Drug Administration (FDA). This partnership led to approval by the FDA of romidepsin for two indications – initially for cutaneous T-cell lymphoma and later for peripheral T-cell lymphoma.

Dr. Bates’ current interests are both laboratory and clinical in nature. Her laboratory efforts include laboratory and translational studies on drug resistance in T-cell lymphomas and advanced solid tumors including breast, pancreatic, neuroendocrine, renal and lung cancers. Her work is dedicated to new drug development, and finding antineoplastic agents that, alone or in combination with other anticancer agents, improve the options available for difficult to treat cancers. Emanating from the clinical and translational development of romidepsin, a histone deacetylase (HDAC) inhibitor, a current focus is on epigenetic therapies, and the development of combination therapies to use with HDAC inhibitors in refractory advanced cancers, including solid tumors. She also has a special interest in drug delivery and drug distribution and the role of the blood brain barrier in creating a sanctuary site for cancers that metastasize to the brain. Clinically, her goal has always been to translate ideas from the laboratory to clinical trials, an effort that has proven very successful. Clinically she seeks to develop combination therapies with histone deacetylase inhibitors for the therapy of solid tumors; and to develop therapies to treat central nervous system metastases, a complication of cancer that is becoming a greater problem as patients live longer with cancer.

Delivery of Protein Therapeutics to the CNS

Tuesday, May 4, 2021
11:15 US Eastern Time

Current State for Blood Biomarkers in AD

Wednesday, May 5, 2021
11:00 US Eastern Time

SESSION I: EMBARKING ON A DRUG DISCOVERY CAMPAIGN

Tuesday, May 4, 2021
11:55 – 13:35 US Eastern Time

Bio
Dr. Martin Watterson serves in an advisory role to pharmaceutical and biotechnology companies in the areas of process and risk analysis. In addition to industry consulting, Dr. Watterson serves on advisory boards for small business start-ups, biotechnology companies, and non-profit organizations in the area of CNS drug discovery and development. His personal CNS drug development experience includes the discovery and preclinical development of novel small molecule therapeutic candidates that attenuate disease related to synaptic dysfunction, as well as participation in development of protein replacement therapeutics.
 
Dr. Watterson is the G.D. Searle Endowed Chair Professorship at Northwestern University, where he is also Professor of Pharmacology in the Feinberg School of Medicine. Previous relevant activities at Northwestern include the founding of an academic drug discovery research and training program characterized by the generation of multiple CNS drug candidates taken into preclinical and clinical development through the leveraged use of Foundation and NIH funding. He also served in various administrative positions, including Department Chair, University Center Director, and Curriculum Co-Director.
 
Prior to Northwestern, he held faculty positions at The Rockefeller University, where he was an Andrew Mellon Fellow, and at Vanderbilt University Medical Center, where he was Professor of Pharmacology and an Investigator in the Howard Hughes Medical Institute. Dr. Watterson is the recipient of the 2016 Melvin R. Goodes Prize recognizing researchers working in promising areas of drug discovery for Alzheimer’s disease and related dementias.

D. Martin Watterson
Northwestern University

CHAIR
Session Overview

Bio

Rouba Kozak obtained her PhD in neuroscience and eating behavior from Henri Poincare University in France, and has an extensive background in cognitive neuroscience with focus on translational research. Specifically, she has focused on bridging the gap between mechanistic studies in preclinical animal models, which provide exquisite detail on how brain circuits control discrete behaviors, and the measurement of functional impact of new therapies in clinical settings (e.g. neuro-degenerative and neuro-psychiatric patient populations).

Dr. Kozak’s scientific training ranged from studies on the mechanisms of obesity to cognition, employing state-of-the- art behavioral and neurochemical sampling techniques; providing her with a unique ability to conceptualize and design experiments in support of programs spanning the wide-ranging disease domains covered in the Takeda portfolio. She has utilized complex behavioral and neurochemical methods to test hypotheses regarding etiologies of neuropsychiatric disorders, as well as to develop translational models to test the efficacy of novel pharmacotherapeutics for the enhancement of cognitive function.

Rouba Kozak
Takeda

What Makes a Good Translational Drug Target?
Bio
Dr. Tilmann Brotz is a senior Development Sciences Consultant with over 15 years of experience in the biopharmaceutical industry leading toxicology, DMPK, in-vivo pharmacology, clinical pharmacology and diagnostic groups. 
 
Most recently Dr. Brotz was Sr. Director, Development Sciences at Achaogen where he contributed to multiple antibiotics programs from lead optimization through Phase 3. Prior to Achaogen, Dr. Brotz worked as head of preclinical development and as a consultant for numerous emerging companies, including VIA Pharmaceuticals where he led the non-clinical and clinical efficacy for a clinical Phase 2 5lipoxygenase inhibitor. 
 
He is an advisor for the CLSI FAST Program and a member of the Scientific Review Board of the Alzheimer’s Drug Discovery Foundation.

Tilmann Brotz
PharmaDirections

CROs to Outsource Pharmaceutical Research Services
Bio
Dr. Susan Catalano is the founder of Cognition Therapeutics and architect of its proprietary and unique biological discovery platform that is based on unbiased phenotypic screens in the target cell population of mature primary neurons. Using her 15 years of industry experience, she and her team discovered and developed the company’s drug candidate CT1812 (Elayta), currently in clinical testing for the treatment of patients with mild-to-moderate Alzheimer’s disease.
 
Prior to founding Cognition Therapeutics, Dr. Catalano was director of discovery biology for Acumen Pharmaceuticals, leading the team that discovered Acumen’s lead candidates targeting Aβ oligomers. Earlier at Rigel, she led the team that pioneered the use of high content phenotypic screening to discover the Aurora kinase inhibitor R763. In scientific leadership roles within the neurophysiology and neuroimaging groups at Roche Palo Alto she led exploratory programs against targets involved in anxiety, depression and schizophrenia.
 
Dr. Catalano received her Ph.D. from U.C. Irvine and postdoctoral training at U.C. Berkeley with Dr. Carla Shatz and at Caltech with Drs. Mary Kennedy and Scott Fraser studying the neurobiology of synaptic plasticity.

Susan Catalano
Cognition Therapeutics

Assembling an Interdisciplinary Team

SESSION II: REPURPOSING AND REPOSITIONING

Tuesday, May 4, 2021
14:00 – 16:05 US Eastern Time

Bio
Dr. Leticia Toledo-Sherman is Senior Director of Drug Discovery for the Tau Consortium and Adjunct Assistant Professor of Neurology at UCLA. In her role at the Foundation, she provides strategic direction and oversight of the drug discovery portfolio. Dr. Toledo-Sherman brings critical expertise and best practices in neurodegenerative disease drug discovery to accelerate the development of therapies for Tauopathies.
 
Previously, she was Director of Medicinal Chemistry and Computer-Aided Drug Design at the CHDI Foundation, leading drug discovery programs for therapeutic development in Huntington’s Disease (HD). Prior to joining CHDI, Dr. Toledo-Sherman was Executive Director of Chemistry at Lymphosign (Pharmascience Inc), leading the development of therapeutics for blood cancers. From 2000 to 2004, she led a multi-site, multidisciplinary team using chemical proteomics and bioinformatics to discover therapeutic targets and to investigate the mechanism of action of drugs. 
 
Dr. Toledo-Sherman started her professional career as a leading scientist at Kinetix Pharmaceuticals (acquired by Amgen), where she implemented an in silico platform aimed at rational drug design of kinase inhibitors targeting multiple therapeutic areas. She has served on the Science Advisory Board of the SGC-UNC Kinase Unit and is currently on the Science Advisory Board of The Chemical Probes Portal.
 
Dr. Toledo-Sherman is an advocate and supporter of open science at the pre-competitive drug discovery stage. She received a PhD in Organic Chemistry from The State University of New York at Stony Brook and did postdoctoral research at the Massachusetts Institute of Technology and The Skaggs Institute for Chemical Biology at The Scripps Research Institute in La Jolla, California.

Leticia Toledo-Sherman
Rainwater Charitable Foundation

CHAIR
Session Overview

Bio
Professor Clive Ballard is Pro-Vice-Chancellor and Executive Dean at the College of Medicine and Health, University of Exeter. 
 
Dr. Ballard has led or co-authored well over 600 research papers. He received the prestigious Weston Brain Institute International Outstanding Achievement Award 2019. His main research specialisms are drug discovery, dementia prevention, improving care for people with dementia in residential homes. He is also an expert in Lewy Body Dementia and Parkinson’s Disease Dementia, and is an advocate of better prescribing and treatment pathways to improve outcomes for individuals. 
 
Before joining Exeter, Dr. Ballard held directorship roles at King’s College London Institute of Psychiatry and at the Alzheimer’s Society. He was pivotal in the successful campaign to overturn a NICE decision and make anti-dementia drugs available for people with dementia.
 
He is part of a growing team of outstanding researchers at the College of Medicine and Health, which is recruiting high-caliber academics and significantly expanding educational programs and student numbers. In the last year, the College has reached the Leiden ranking’s world top 20 for the quality of its Health and Biomedical Science research, attracted a £10 million donation from the Dennis and Mireille Gillings Foundation, launched a new Academy of Nursing and received permission to expand Medicine student numbers.

Clive Ballard
University of Exeter

Disadvantages (and Advantages) of Drug Repurposing
Bio

Arnab K. Chatterjee completed a Bachelor of Arts in Chemistry and minor in Business from Northwestern University in 1997 and a brief period as an IT consultant, he proceeded on to the Caltech to conduct his chemistry doctoral thesis research in the laboratory of Professor Robert H. Grubbs in from 1999-2002 (Professor Grubbs was awarded the Nobel Prize in Chemistry in 2005 for his work in the area olefin metathesis). Arnab joined the Genomics Institute of the Novartis Research Foundation (GNF) as a Principal Investigator in the chemistry department. His work over of 9+ years has been focused on hit-to-lead and lead optimization in several medicinal chemistry projects ranging a variety of therapeutic areas (neuroscience, oncology, respiratory disease and infectious diseases).  The project teams he has worked with have produced 8 preclinical candidates optimized for both inhaled and oral formulations with three novel first-in-class compounds in clinical trials including KAF156 currently in Phase 2b a first-in-class antimalarial.

Since May 2012 he has been responsible for setting up and leading the chemistry group at the Calibr in La Jolla, CA working across a wide-variety of disease areas including three compounds in clinical trials and three in IND-enabling studies in the areas of malaria, HIV, regenerative medicine and most recently for SARS-2. His research interests include application of novel synthetic methods to expedite the structural diversification in medicinal chemistry, cell-based lead optimization, advanced formulation methods for delivery of small molecules and drug repurposing.

Arnab Chatterjee
Calibr

Challenges to Create a Drug Repurposing Library and Its Application to the Treatment of AD
Bio
Dr. Sharon Rosenzweig-Lipson is Vice President of Research and Development for AgeneBio. Dr. Rosenzweig-Lipson offers AgeneBio expertise in screening strategies, in vivo models, translation and clinical development strategy with more than 25 years of experience developing compounds for psychiatric and neurologic indications in the pharmaceutical industry. Her career in big pharma includes American Cyanamid, American Home Products, Wyeth and Pfizer. Sharon has successfully led teams from the exploratory phase through to a successful Phase 2 POC in schizophrenia and the current AgeneBio Phase 3 trial in MCI due to AD.
 
She earned her PhD in Behavioral Neuroscience from Harvard University. Sharon’s long career in the pharmaceutical industry running neurology and psychiatry programs from the exploratory phase up through clinical Phase 2 proof of concept studies provides her with an in depth understanding of the drug discovery and development process. In her role as Vice President of Research &Development for AgeneBio, Sharon has led the team in all aspects of the AGB101 drug development program (HOPE4MCI trial) and the GABA-A a5 PAM Discovery program.

Sharon Rosenzweig-Lipson
AgeneBio

From Reformulation to Toxicology
Bio
Rob Berman, MD, is a board member and advisor to multiple biotechnology companies that are focused on developing drugs for central nervous system disorders.  His research career started in academia at Yale, where he studied the mechanism of action of antidepressants and novel treatments for psychiatric disorders including exploiting the value of already-approved agents such as ketamine and pindolol.  For over 20 years he has worked within the industry, first at Pfizer and then Bristol-Myers Squibb in clinical development roles, including leading the clinical program for avagacestat (a gamma secretase inhibitor for the treatment of Alzheimer’s Disease). 
 
In 2013, he co-founded Biohaven Pharmaceuticals (NYSE:BHVN) and was Chief Medical Officer until 2019. Biohaven is assessing the efficacy of troriluzole (a prodrug of riluzole) in the treatment of Alzheimer’s disease.  He completed his undergraduate degree at Yale University in Molecular Biophysics and Biochemistry, where he oversaw the development of classmate Mark Forman MD PhD, and yet somehow was allowed to get his medical degree from the Mt Sinai School of Medicine.  
 
He is currently an Adjunct Professor of Psychiatry at the Yale University School of Medicine and has over 70 peer-reviewed publications. In addition, he is a strategic advisor to Biohaven Pharmaceuticals and BioXcel Therapeutics, as well as a board member of Gilgamesh Pharmaceuticals and Osmol Therapeutics, the latter of which is pursuing the development of a repurposed drug for treatment of neuropathy.

Rob Berman, MD
BioHaven Pharmaceuticals

IP Strategy and Commercial Consideration in Repurposing and Repositioning Programs

SESSION III: BIOMARKERS TO ENABLE PRECLINICAL AND CLINICAL PROOF OF CONCEPT STUDIES

Wednesday, May 5, 2021
11:40 – 13:20 US Eastern Time

Bio

Dr. Edward (Ted) Spack has over 25 years of biotech translational experience, including preclinical development of drug candidates for multiple sclerosis, nosocomial infection, and biodefense. At SRI International, Dr. Spack directed the PharmaSTART program (a consortium of SRI, Stanford, UC Berkeley, UC San Diego, and UC San Francisco), drafting preclinical development blueprints that led to several major grants and new biotech companies. He has consulted with the NIH translational core services committee and several NIH institutes on preclinical development and serves on several study sections, including the NIA Alzheimer’s Disease Drug Development review panel, the NIH Small Business Review on Drug Discovery for Aging, Neuropsychiatric and Neurological Disorders, and the Falk Trust Catalyst and Transformational Award programs.

As Managing Director of an innovative partnership between the National Multiple Sclerosis Society and EMD Serono he supported innovative early stage MS drug discovery and development projects in academic labs and biotech companies. Through the California Life Sciences Institute (CLSI) FAST program and the SRI Innovation program he mentors SF Bay area and international academic and industry teams in biotech company formation and pitch decks.

Dr. Spack received his doctoral degree from The Johns Hopkins University and his postdoctoral fellowship in cellular immunology at Stanford University.

Edward (Ted) Spack
Vector BioSolutions/Therini

CHAIR
Session Overview

Bio

Darrell A. Henze, PhD, is the Director of Neuroscience, leading the Sensory Biology and Neuropharmacology group in the Department of Neuroscience at Merck & Co., Inc. Dr. Henze graduated Magna Cum Laude from The University of Rochester and earned his PhD from the University of Pittsburgh in 1998. His PhD work focused on understanding the synaptic properties of the hippocampal Mossy Fiber synapse. He completed a post-doctoral fellowship at Rutgers, The State University of New Jersey working in the laboratory of Dr. Gyorgy Buzsaki focusing on in vivo electrophysiological techniques to study how single cells contribute to network activity.

In 2002, he joined Merck where he worked on discovery programs in the therapeutic areas of schizophrenia, Parkinson’s disease, and Alzheimer’s disease. In 2006, he moved to join the pain and migraine research group providing discovery project leadership. In 2013, he took on the lead role for the Late Discovery Pain and Migraine Department. In 2016, he transitioned to leading an experienced group of in vivo pharmacologists providing  pharmacodynamic and translational biomarker support across the entire neuroscience portfolio, leveraging rodent, dog, and primate models to support the discovery and development of therapeutics for neurodegeneration, cognition, psychiatry, and pain. Most recently, in the fall of 2020, he has resumed responsibility for the discovery portfolio for pain and itch as well as leading in vitro electrophysiology and in vivo pharmacology teams supporting a range of neuroscience programs including neurodegeneration.

Darrell Henze
Merck

Target Engagement and Translatability in Preclinical Models of CNS Disease
Bio

Michael Gold, MS, MD, is currently Vice-President, Development Neurosciences at AbbVie. Before this, Dr. Gold spent time at PPD, UCB Bioscience and Allon Therapeutics. At UCB, he served as vice president of and head of CNS practice, and at Allon Therapeutics, he served as Chief Medical Officer. From 2005-2011, Dr. Gold was with GSK, where he served as medicines development leader for Alzheimer’s disease and vice president of neurosciences, medicines development center. Among his many responsibilities at GSK, he was accountable for all aspects of the execution of the clinical development plans for two late stage assets in the area of Alzheimer’s disease. Earlier in his career, he held positions of increasing responsibility within clinical research at J&J and Bristol-Myers Squibb.

Michael Gold
AbbVie

Failing Fast: Small Trials to Prove Efficacy
Bio

Philip Scheltens, MD, is Professor of Cognitive Neurology and the Director of the Alzheimer Center at the VU University Medical Center in Amsterdam. He is also the initiator and Vice President of Deltaplan Dementia Netherlands. In 2011, Dr. Scheltens was appointed scientific director at the Pearlstring Institute and elected a Member of the Royal Netherlands Academy of Arts and Sciences, where he is now a member of the Board. He is an Honorary Professor of Neurology at University College London.

His main clinical and research interests are Alzheimer’s disease, vascular dementia, frontotemporal dementia, magnetic resonance imaging, PET imaging and biomarkers for disease detection and early diagnosis. He is active in the field of biomarkers and clinical trials and has been the national Primary Investigator for many trials, including phase 1-3 international multicenter clinical trials.

Dr. Scheltens is an active collaborator and contributor to many fields and is currently a member of various research societies including the European Academy of Neurology, the Dutch Society for Neurology, the American Academy of Neurology, the Alzheimer Imaging Consortium, and the ISTAART Consortium. He is also a jury member of the Brain Prize and a member of the organizing committee for the VASCOG conference 2016 in Amsterdam. From 2017 he is (again) a member of the AAIC program committee.

Dr. Scheltens received his MD in 1984 and his PhD in 1993 from the VU University in Amsterdam.

Philip Scheltens
VUMC Alzheimer’s Center

A Path Toward Precision Medicine in Alzheimer’s Disease

SESSION IV: CASE STUDIES – CHASING NEW PATHWAYS

Wednesday, May 5, 2021
13:45 – 15:50 US Eastern Time

Bio

Andrew Pieper, MD, PhD, is a board-certified psychiatrist and neuroscientist in the Department of Psychiatry at Case Western Reserve University and University Hospitals Cleveland Medical Center, where he serves as Morley Mather Chair in Neuropsychiatry. He also serves as Psychiatrist at the Louis Stokes Cleveland VA Medical Center (VAMC), as well as Investigator in the University Hospitals Harrington Discovery Institute, where he is the Director of Neurotherapeutic Discovery.

Dr. Pieper is devoted to patient care and basic science research applied to disease, most keenly focused on neurodegeneration. Clinically, he maintains an active Adult Psychiatry Outpatient Clinic at the VAMC. His goal is to understand and investigate human disorders in order to foster development of new pharmacologic treatments for patients.

Andrew Pieper
Harrington Discovery Institute

CHAIR
Session Overview

Bio

Siew Peng Ho obtained her PhD in synthetic organic chemistry with Donald Cram at UCLA. Her post-doctoral training was with Bill DeGrado on de novo protein design at the Central Research Department at DuPont. Soon after, she began working on nucleic acid therapeutics first at DuPont Pharmaceuticals and later at Bristol-Myers Squibb. She is currently a consultant in nucleic acid therapeutics, largely with Harrington Discovery Institute.

Siew’s comprehensive expertise over 30 years encompasses design, synthesis, cellular and in vivo studies with these molecules. Her group validated over 40 different gene targets in in vivo models for drug discovery programs using antisense and siRNA oligonucleotides in cardiovascular diseases, metabolic diseases, neuroscience and virology. She was also intimately involved in BMS’ therapeutic collaborations with Ionis (drug candidate advanced to Phase 1) and Santaris (now part of Roche).

Siew Peng Ho
Harrington Discovery Institute

Design and Optimization of Oligonucleotide Modulators of RNA for the Treatment of Neurodegenerative Diseases
Bio

Dr. Travis Dunckley obtained his undergraduate degree in molecular and cellular biology and biochemistry from the University of Arizona. His doctoral work was completed at the University of Arizona in the field of molecular and cellular biology. Prior to joining TGen, he studied gene expression at the level of mRNA stability. In 2000, Dr. Dunckley expanded his training to the field of Neurobiology during a fellowship position at Barrow Neurological Institute where he studied structure/function interactions in nicotinic acetylcholine receptors (nAChR) and identified novel genes regulated by nAChR activity. In 2003 he transitioned to the Neurogenomics division at TGen to leverage the rapidly developing genomics field for the identification of relevant therapeutic targets and biomarkers for Parkinson’s disease and Alzheimer’s disease. He has developed efficient single cell isolation and transcriptome profiling methods, high-throughput functional assays directly applicable to relevant phenotypic endpoints in neurodegenerative diseases – including α-synuclein accumulation and tau hyperphosphorylation, and he has performed large scale association studies to identify DNA variants underlying neurodegenerative disease.

Dr. Dunckley has recently moved to the Neurodegenerative Disease Research Center at the Biodesign Institute at Arizona State University where ongoing work involves the role of epigenetics as a potentially modifiable factor contributing to neurodegeneration and the development of novel small molecule inhibitors of the DYRK1A kinase for potential therapeutic applications in Alzheimer’s disease and Down syndrome.

Travis Dunckley
Arizona State University

From Academic Discovery to Commercial Potential: HDI Case Study
Bio

Toren Finkel received his undergraduate degree in Physics and his MD and PhD degree from Harvard Medical School. Following a residency in Internal Medicine at the Massachusetts General Hospital, he completed a fellowship in Cardiology at Johns Hopkins Medical School. In 1992, after completing his clinical training, he came to the NIH as an Investigator within the Intramural Research Program of the National Heart, Lung and Blood Institute (NHLBI). Over the next 25 years at the NIH, he held various positions including Chief of the Cardiology Branch and Chief of the Center for Molecular Medicine within the NHLBI. He has been inducted into the American Society for Clinical Investigation (ASCI) and the Association of American Physicians (AAP). Toren is also a Fellow of the American Association for the Advancement of Science (AAAS) and an elected member of the National Academy of Medicine. He serves on numerous editorial boards including currently serving on the Board of Reviewing Editors for Science.

As of Sept 1, 2017, Dr. Finkel assumed the role of the Director of the Aging Institute, and the G. Nicholas Beckwith III and Dorothy B. Beckwith Endowed Chair of Translational Medicine at the University of Pittsburgh/UPMC. Over the last three decades, his laboratory has made fundamental contributions in our understanding of the role of reactive oxygen species and mitochondrial function in aging and age-related diseases. According to Google Scholar, he currently is one of the highest cited authors in both cardiovascular disease and in aging. Toren also co-founded and is the CSO of Generian Pharmaceuticals. With his colleagues Bill Chen and Yuan Liu he identified a small molecule activator of TFEB which is anticipated to be in Phase I testing within 12 months and will be of potential benefit for a range of age-related disorders including neuro-degenerative diseases.

Toren Finkel
Generian

Small Molecules to Modulate Autophagy and Lysosomal Biogenesis in LBD and AD

Bio

Jamie Bilsland, PhD – AstronauTx

Jamie Bilsland
AstronauTx

Protein Aggregation in LBD, AD and FTD