West Coast CNS Therapeutics Xchange
San Francisco
29 September, 2022
Welcome to hubXchange’s West Coast CNS Therapeutics Xchange 2022, bringing together executives from pharma and biotech to address and find solutions to the key issues faced in developing CNS therapies.
Discussion topics will cover Novel Targets, Preclinical, Clinical Studies, Advanced Therapies and Drug Delivery.
Take advantage of this unique highly interactive meeting format designed for maximum engagement, collaboration and networking with your peers.
NOTE: This will be an In-Person event
VENUE DETAILS: DoubleTree by Hilton San Francisco Airport Hotel, 835 Airport Blvd., Burlingame CA 94010-9949
Novel Targets
Operant conditioning procedures have played an important role in psychopharmacology research both in academic and applied drug discovery settings. The purpose of this presentation is to describe three examples from a rodent based operant platform separately established both at IVS and TPH, and now combined into a single unit within the merged company, has contributed to recent CNS drug discovery efforts.
- The VMAT2 inhibitor tetrabenazine as a translational approach to discover new treatments for depression.
- Operant procedures designed to investigate both “micro” and “macro” dose psychedelics.
- Development of an operant platform approach to discover new treatments for ADHD
Chief Scientific Officer, Transpharmation
Guy Higgins has 30 years international experience in CNS R&D with major pharmaceutical companies (Glaxo-Wellcome,
Hoffmann-La Roche, Schering-Plough, NPS Pharmaceuticals). He has success in developing translational models and bringing multiple NCEs to clinical development. Guy is also the Adjunct Professor of Pharmacology, U. Toronto, with 100+ peer-reviewed papers in Neuroscience Drug Discovery.
9:05 – 10:05am
Novel Targets in Neurodegeneration Therapeutics
- Why are we not trying to do more with focus on the lives of patient in such dire symptomatic and QOL need right now, rather than placing so many bets and so much investment (human and financial) on ‘pie in the sky’ wishful thinking and false promises of disease modification
- Is the complex and heterocellular nature of cerebral function, and accordingly neurodegenerative pathobiology, too challenging for focuses on reductionist monogenic or single cell-type approaches to novel therapy?
- In the face of such dismal failure in targeting protein pathobiological byproducts of neurodegeneration for therapeutics, have we gone remotely close enough to understand the earliest changes occurring in the brains of the most prevalent, sporadic neurodegenerative diseases to identify truly transformational approaches to disease
- How can we continue to focus so heavily on ‘novel’ disease modifying targets in degeneration when there is so little translational evidence for their role in causing the underlying loss of function, and so few markers to reliably predict potential for offering benefit?
- Are there any common mechanistic threads that can connect therapeutic approaches more broadly, for greater potential to achieve greatest impact in neurodegeneration (lumping rather than splitting)?
President & CEO, Curasen Therapeutics Inc.
Anthony Ford has over 30 years of neuroscience discovery & development experience from leading biopharma companies, and is CEO of CuraSen Therapeutics, which he co-founded as venture partner for NLV Partners in 2018. Prior to CuraSen, he was founder and CSO for Afferent Pharmaceuticals, and led development of first-in-class P2X3 antagonists for sensory conditions until the company’s acquisition by Merck in 2016. Previously, he held positions at Roche Pharmaceuticals (Palo Alto) over 20 years, including head of neuroscience, initiating and driving innovative programs in neurobiology. Dr. Ford trained in pharmacology / neuroscience at Universities of Bradford and Nottingham, UK and has published extensively with focus on therapeutics for poorly addressed neuroscience diseases.
Spotlight Presentation: Enhanced Delivery of Biotherapeutics in Nonhuman Primates
- Altasciences Preclinical Services
- Specialized delivery methods in nonhuman primates
- Case studies: Gene Therapy & Stem Cell Transplantation
Vice President Toxicology, Altasciences
Norbert Makori is Vice President of Toxicology, Altasciences. He has over 20 years of industry experience as a toxicologist (general and reproductive toxicology) and immunotoxicology, including study director role and management. Prior to joining Altasciences, Norbert was Director of the General Toxicology group at Charles River Laboratories in Ashland, Ohio. Prior to that, he held the position of Director of Toxicology and Immunotoxicology at WIL Research Laboratories. Norbert is a Diplomate of the American Board of Toxicology (DABT), trained as a veterinarian and has a PhD in Comparative Pathology from the University of California in Davis.
1:55 – 2:55pm
Utility or Futility of target-based screening for identifying disease-modifying therapies for CNS disorders
- Has the deconvolution of human disease biology and human genetics led to the identification of promising drug targets for CNS disease?
- Will target-based virtual database screening and in silico molecular design live up to the promise of dramatically shortening drug development timelines and reducing R&D expenses?
- Will AlphaFold live up the hype and revolutionize drug discovery?
- Because preclinical animal models are poor predictors of human efficacy in the clinic is there a compelling need to develop in vitro human disease models?
- What is the status of iPSC-based disease modeling and drug discovery for CNS disorders?
- Can functional phenotypic screening of patient-derived brain organoids identify promising disease-modifying targets and therapeutic candidates for CNS disorders?
Chief Scientific Officer, Vyant Bio
Robert Fremeau is an experienced R&D leader with over two decades of drug discovery experience in academia and industry advancing next-gen drug development for severe neurological disorders. He is an accomplished scientist and biotech entrepreneur with an established history of scientific innovation and program leadership at the intersection of target discovery and validation, translation, and clinical development. As a Scientific Director at Amgen Inc., he led and contributed to multiple teams that advanced small molecules into clinical trials against innovative targets across neurological indications. Throughout his academic career at Duke University and UCSF, Dr. Fremeau made seminal contributions to the first molecular and functional characterization of the receptors and transporters for the biogenic amine and amino acid neurotransmitters. Dr. Fremeau holds numerous patents and has offered over 65 peer-reviewed publications in high impact scientific journals.
Preclinical Topic: Brain-Wide Cellular Resolution Measurement of Therapeutic Distribution, Target Engagement and Efficacy Using Tissue Clearing and Machine Learning-Enabled Software
- Recent advances in optical clearing and light sheet imaging have opened an exciting new avenue for neuroscientists, providing access to the intricate anatomy of intact brain
- The Translucence Biosystems pipeline provides unbiased and complete cellular resolution measurements of brain-wide therapeutic distribution and efficacy in pre-clinical rodent brains
- We will show examples of our methods helping CNS drug discovery efforts
visualizing Ab therapeutics crossing the BBB, measuring β-Amyloid plaques in AD model mice and examining brain-wide effects of small molecules on neuronal activity - The discussion will focus on the needs in CNS Drug Discovery for better pre-
clinical measures of therapeutics in the brain and cases where tissue clearing
and light sheet imaging may or may not be appropriate
CEO, Translucence Biosystems
Damian Wheeler is the co-Founder and CEO of Translucence Biosystems. As a
neuroscientist with more than 25 years of research experience, both in academia and in
drug discovery, Damian’s focus is on developing and commercializing the next-
generation technologies to advance neuroscience research and accelerate CNS drug
discovery. During his Ph.D. studies at McGill University and Post-Doc at Stanford,
Damian became a leading expert in the signaling mechanisms by which neuronal
activity activates gene transcription. He then spent 9 years at Dart NeuroScience
developing cognition-enhancing drugs that engage these mechanisms. In mid-2018,
Damian co-founded Translucence Biosystems Sunil Gandhi and Ricardo Azevedo.
4:45 – 5:45pm
Emerging non-Amyloid targets for Alzheimer’s disease
- Given the continuing reports of negative studies, is the amyloid hypothesis dead?
- How can we advance our understanding of Alzheimer’s biology beyond amyloid? AI/ML? Novel biomarkers?
- How can small or emerging biotechs advance their innovative hypotheses?
Vice President Clinical Development, Neuroscience & Rare Disease, Recursion
Glenn Morrison is a neuroscientist by training and has over 20 years experience in clinical development. He is currently VP Clinical Development, Neuroscience and Rare Diseases at Recursion. Before joining Recursion, Glenn was leading clinical development at both small and large bio-pharma companies. Most notably, as VP of Global Clinical Development at Zogenix, he led clinical development efforts to deliver FDA and EMA approvals of fenfluramine for Dravet syndrome, and led development efforts to support FDA and EMA submission and future approval for Lennox Gastaut Syndrome. He spent six years at Roche/Genentech where he led the Phase 3 global implementation for two anti-amyloid antibodies in Alzheimer’s disease. Other relevant therapeutic area experience includes Down syndrome, Autism, Fragile X, MS and spinal cord related spasticity, and ALS. Glenn earned his BSc, and MSc and PhD in neurobiology all from the University of Toronto, and did post-doctoral research at UCLA.
Preclinical
Operant conditioning procedures have played an important role in psychopharmacology research both in academic and applied drug discovery settings. The purpose of this presentation is to describe three examples from a rodent based operant platform separately established both at IVS and TPH, and now combined into a single unit within the merged company, has contributed to recent CNS drug discovery efforts.
- The VMAT2 inhibitor tetrabenazine as a translational approach to discover new treatments for depression.
- Operant procedures designed to investigate both “micro” and “macro” dose psychedelics.
- Development of an operant platform approach to discover new treatments for ADHD.
Chief Scientific Officer, Transpharmation
Guy Higgins has 30 years international experience in CNS R&D with major pharmaceutical companies (Glaxo-Wellcome,
Hoffmann-La Roche, Schering-Plough, NPS Pharmaceuticals). He has success in developing translational models and bringing multiple NCEs to clinical development. Guy is also the Adjunct Professor of Pharmacology, U. Toronto, with 100+ peer-reviewed papers in Neuroscience Drug Discovery.
Is disease modification truly attainable for complex degenerative diseases like AD?
- Does the approval of multiple therapeutics for multiple sclerosis provide lessons for developing
disease modifying drugs for other neurodegenerative disorders? - How do past failures and a controversial approval (Aducanumab) inform targets based on the
amyloid hypothesis? Are we targeting the right form(s) of amyloid (e.g. Ab processing, soluble
oligomers, Ab plaques)? - Does protein misfolding offer shared targets/strategies across multiple neurodegenerative
diseases? - Does modification of processes such as inflammation, chaperoning, and phagocytosis offer
alternative/complementary approaches to directly targeting aberrant proteins? - How will imaging agents and earlier assays to detect disease pathology affect the design of
disease modifying drugs for complex degenerative diseases?
Principal Consultant, Vector BioSolutions
Dr. Edward (Ted) Spack is a cellular immunologist focused on neuroinflammation with over 25 years of SF Bay area biotech experience, including preclinical development of drug candidates for AD and MS. He has previously served as Managing Director of a funding initiative between the National MS Society and EMD Serono and co-founded Therini Bio Inc., a biotech spinout from the Gladstone Institute developing a lead monoclonal antibody for neurodegenerative disorders. He is currently an independent preclinical consultant and chairs an NIH U01 committee for AD drug development. He received his academic training at The Johns Hopkins University and Stanford University.
The relevance of EEG biomarkers to address CNS drug discovery challenges
- Can EEG biomarkers participate to the phenotyping of a model
- Beyond the trace: explore different kinds of applications for EEG Phenotyping
- What type of therapeutic areas are still out of reach for the EEG technology?
Scientific Sales Engineer USA & LATAM, SynapCell
Hugo holds a MSc in Neuroscience and a Master’s degree in international business in science. He joined SynapCell’s business development team in 2020 and quickly became the manager of the USA and LATAM zone. His background allows him to possess skills in both neuroscience and sales. Thus, he can understand the current needs of the biopharma industry towards drug discovery for CNS disorders, and how to address them. Thanks to his experience at SynapCell, Hugo shares his insights for the development of new offers and the establishment of new business strategies.
Head of Technology and Principal Scientist, SynapCell
Julien hold a PhD in Neuroscience from the University of Lyon, France. He then did a post-doc at the Center for Addiction and Mental Health at Toronto, Canada, followed by another post-doc at the University of Toronto. Julien has been working at SynapCell since 2017 when he first started as a Research and Innovation Scientist to finally be appointed in 2021 as Head of technology and Principal Scientist. He use his extensive knowledge of neuroscience and physiology to manage R&D Preclinical projects and to develop new innovative solutions for our sponsors. With over 12 years of experience in the field, he is well-versed in the emerging technologies and industry trends.
- Altasciences Preclinical Services
- Specialized delivery methods in nonhuman primates
- Case studies: Gene Therapy & Stem Cell Transplantation
Vice President Toxicology, Altasciences
Norbert Makori is Vice President of Toxicology, Altasciences. He has over 20 years of industry experience as a toxicologist (general and reproductive toxicology) and immunotoxicology, including study director role and management. Prior to joining Altasciences, Norbert was Director of the General Toxicology group at Charles River Laboratories in Ashland, Ohio. Prior to that, he held the position of Director of Toxicology and Immunotoxicology at WIL Research Laboratories. Norbert is a Diplomate of the American Board of Toxicology (DABT), trained as a veterinarian and has a PhD in Comparative Pathology from the University of California in Davis.
- What are temporal biomarkers?
- Why reverse translate?
- How to work smarter?
CEO and Founder, Gryphon Bio
William E. Haskins (Co-Founder and CEO, GRYPHON BIO, Inc.), together with Co-Founder Kevin Wang, has discovered novel temporal biomarkers and therapeutic targets from unexpected waves of brain molecules in the blood of subjects with CNS disorders, including traumatic brain injury (TBI). He previously led R&D teams for biologics at small and large biotechnology companies, including Genentech. He is highly experienced with bio/analytical chemistry, ultrahigh sensitivity and -omics technologies, bioinformatics, biomarkers, and CNS biology. He has co-authored numerous peer-reviewed publications and patents, and he is the PI for several grant awards and clinical trials.
Poster Session: Advancing preclinical (neuro)inflammation models to accelerate CNS drug discovery
- Are existing inflammation models fit for purpose?
- Is there a role for peripheral immune cells in CNS drug discovery?
- How can we back-translate clinical findings to the laboratory?
Head of Transpharmation Ireland, Transpharmation
Jack has more than 10 years experience in CNS research. He is currently Head of Transpharmation’s Irish site, based at Trinity College Dublin, where he is also an Adjunct Assistant Professor in Physiology at the Trinity College School of Medicine. Jack’s experience spans psychiatric, neurodegenerative and inflammatory disorders and his research implements a translational focus ranging from preclinical models of disease to clinical biomarker discovery. After completing a PhD in neuropharmacology at Trinity College Dublin in Ireland focusing on the behavioural and molecular effects of cannabinoids, Jack spent time as a researcher at the University of Michigan in the USA. Following that, he returned to Ireland taking up a post at the APC Microbiome Institute at University College Cork leading an industry-academia partnership aimed at developing novel therapeutic interventions for inflamm-ageing. Since joining Transpharmation in 2014, Jack has been instrumental in the foundation and development of the company’s R&D-focused Irish site, where he steers scientific development and manages external partnerships. Jack sits on the board of Ireland’s national neuroscience society, Neuroscience Ireland, as the industry representative.
4:45 – 5:45pm
Methods to increase the success rates of drugs entering clinical phases
- What is the biggest hurdle so far to increase the success rate of drugs entering clinical trials?
- What are we doing to overcome the hurdles right now?
- What lesson have we learned from working on tackling the hurdles?
- What can we do in the next 3-5, or 10 years to increase the success rate?
- Any new methods, approaches, or technologies you want to share that can improve the success rate?
President and CEO, NeuCyte
Tao Huang is President/CEO of NeuCyte, a biotechnology company focused on CNS drug
discovery. NeuCyte has developed a highly functional in vitro platform using hiPSC-derived neurons for target identification and validation, efficacy testing, neurotoxicity assessment, and disease modelling. Tao has been an investor and patent attorney prior to co-founding NeuCyte. He obtained his Ph.D. in Biochemistry from Peking Union Medical College, where he studied the function of fragile X syndrome gene during development. He received his post-doctoral trainings from the University of
Colorado School of Medicine and Princeton University, and obtained his J.D. from the University of Michigan Law School.
Clinical Studies
Registration
Operant conditioning procedures have played an important role in psychopharmacology research both in academic and applied drug discovery settings. The purpose of this presentation is to describe three examples from a rodent based operant platform separately established both at IVS and TPH, and now combined into a single unit within the merged company, has contributed to recent CNS drug discovery efforts.
- The VMAT2 inhibitor tetrabenazine as a translational approach to discover new treatments for depression.
- Operant procedures designed to investigate both “micro” and “macro” dose psychedelics.
- Development of an operant platform approach to discover new treatments for ADHD
Chief Scientific Officer, Transpharmation
Guy Higgins has 30 years international experience in CNS R&D with major pharmaceutical companies (Glaxo-Wellcome,
Hoffmann-La Roche, Schering-Plough, NPS Pharmaceuticals). He has success in developing translational models and bringing multiple NCEs to clinical development. Guy is also the Adjunct Professor of Pharmacology, U. Toronto, with 100+ peer-reviewed papers in Neuroscience Drug Discovery.
Utilizing clinical biomarkers to enhance drug development in neurodegenerative diseases
What are the advantages and disadvantages of currently used biomarkers of neurodegeneration
How important are target engagement biomarkers in early clinical development
What are some emerging biomarkers that can be used effectively for patient enrichment?
What data are necessary to qualify response biomarkers as surrogate endpoints?
- In what areas is the need greatest for the development of novel biomarkers?
Senior Vice President Translational Medicine and Clinical Strategy, Asceneuron SA
Ryan Schubert is the Senior Director of Clinical Development and Head of Translational Medicine at Asceneuron. He is responsible for clinical strategy, medical oversight, operational support, and biomarkers for Asceneuron’s pipeline of small molecule drugs.
Ryan received his M.D. from the Stanford University School of Medicine and completed an Internal Medicine Internship, Neurology Residency, and Post-Doctoral Fellowship as a physician-scientist at the University of California, San Francisco. Ryan is passionate about translating cutting-edge scientific discoveries into new medicines for patients with high unmet medical needs. He is a patent inventor, the author of over ten peer-reviewed publications, and holds a B.S. in Biology from the University of Southern California.
Bruce Morimoto has over 25 years of industry experience in leading project teams in the development of innovative medicines, providing guidance in the design and execution of preclinical, clinical and regulatory strategies with a therapeutic focus in Parkinson’s, Alzheimer’s and frontotemporal dementias. Previously, Bruce held leadership roles at Alkahest, Celerion, Cerecin and Allon Therapeutics, and works closely with the Michael J Fox Foundation, chairing one of their scientific review panels. He is an advisor to several biotech companies helping to move their programs through clinical development and
drug registration. Bruce started his career on the faculty in the Chemistry Department at Purdue University where his independent research focused on neuronal signal transduction.
Bruce earned his doctorate in biochemistry from UCLA and completed a postdoctoral
fellowship at the University of California Berkeley.
- How to overcome the complex, and therefore risky, treatment needs associated with older adults to include them in a trial
- How to align the population under investigation and the intended consumer without compromising the external validity of benefit-risk assessments made for launched drugs?
- Is the recently released FDA guidance on the Inclusion of Older Adults in Cancer Clinical Trials feasible?
- What are the key considerations when including older adults in clinical trials: with a focus on accurately monitoring cognition?
Director of Clinical Affairs, Cambridge Cognition
Kenton has over 25 years of biotechnology and pharmaceutical experience with involvement in all aspects of the industry from drug discovery and clinical development through to pre-commercialization. He currently serves as Director of Clinical Affairs at Cambridge Cognition, a leading neuroscience technology company optimizing the assessment of cognition for better brain health research. Prior to this, Kenton served as Chief Scientific Officer for New Mexico-based biotech Zocere, Inc. where he worked on developing a neuroprotectant drug designed to combat brain injury resulting from stroke. From 1998 to 2012, Kenton worked at Myriad Pharmaceuticals, Inc. in Salt Lake City and served in a variety of roles including Senior Director of Scientific and Clinical Affairs, Chief Scientist of the tarenflurbil clinical development program (an investigational drug for the treatment of Alzheimer’s disease, developed through Phase 3), Director of Neurodegeneration Therapeutics Discovery and Director of Strategic In-licensing and Scientific Evaluation.
Clinical Scientist, Cambridge Cognition
John is a Boston-based licensed clinical psychologist and neuropsychologist, receiving his doctorate from Louisiana State University. He has a background conducting neuropsychological evaluations in hospitals and healthcare settings and has published extensively in the development and implementation of digital tools and devices used to measure cognitive and daily functioning in a range of clinical populations.
Spotlight Presentation: Enhanced Delivery of Biotherapeutics in Nonhuman Primates
- Altasciences Preclinical Services
- Specialized delivery methods in nonhuman primates
- Case studies: Gene Therapy & Stem Cell Transplantation
Vice President Toxicology, Altasciences
Norbert Makori is Vice President of Toxicology, Altasciences. He has over 20 years of industry experience as a toxicologist (general and reproductive toxicology) and immunotoxicology, including study director role and management. Prior to joining Altasciences, Norbert was Director of the General Toxicology group at Charles River Laboratories in Ashland, Ohio. Prior to that, he held the position of Director of Toxicology and Immunotoxicology at WIL Research Laboratories. Norbert is a Diplomate of the American Board of Toxicology (DABT), trained as a veterinarian and has a PhD in Comparative Pathology from the University of California in Davis.
Are circulating neurodegeneration biomarkers ready for prime time?
- Challenge of biomarker development for neurodegeneration due to matrix limitations
- Imaging biomarkers – utility in clinical practice vs clinical trials
- Circulating neurodegeneration markers – utility in individual treatment vs study monitoring
Head, GI and Neurology Disease Teams, Bristol-Myers Squibb
Sarah Harris received her Ph.D. in Immunology from University of Rochester School of Medicine and Dentistry and has experience in startup, biotech, big pharma, CRO and non-profit foundations leading translational medicine and bioanalytical activities for compounds in oncology, vaccines, immunology and neurology. She has supported compounds from IND through post-marketing stages including Rituxan, Zevalin, Tysabri and Zeposia. Sarah currently serves as a scientific advisor for multiple non-profit family foundations with focus on neurodegenerative disorders in children and leads the Neurology, GI and Atopic Disorders Translational Medicine disease teams at BMS.
1-2-1 Meetings / Networking Break
1-2-1 Meetings / Networking Break
Afternoon Refreshments
Advanced Therapies
Operant conditioning procedures have played an important role in psychopharmacology research both in academic and applied drug discovery settings. The purpose of this presentation is to describe three examples from a rodent based operant platform separately established both at IVS and TPH, and now combined into a single unit within the merged company, has contributed to recent CNS drug discovery efforts.
- The VMAT2 inhibitor tetrabenazine as a translational approach to discover new treatments for depression.
- Operant procedures designed to investigate both “micro” and “macro” dose psychedelics.
- Development of an operant platform approach to discover new treatments for ADHD
Chief Scientific Officer, Transpharmation
Guy Higgins has 30 years international experience in CNS R&D with major pharmaceutical companies (Glaxo-Wellcome,
Hoffmann-La Roche, Schering-Plough, NPS Pharmaceuticals). He has success in developing translational models and bringing multiple NCEs to clinical development. Guy is also the Adjunct Professor of Pharmacology, U. Toronto, with 100+ peer-reviewed papers in Neuroscience Drug Discovery.
Delivering biotherapeutics to a large volume of brain parenchyma: routes of administration, efficiencies, and other considerations
- What are the major physiological and anatomical factors governing CNS biodistribution across species?
- How are currently approved CNS biotherapeutics administered and where are their target sites?
- What are the pros and cons of existing and emerging approaches for CNS delivery of biotherapeutics?
- What additional drug development considerations are important for CNS drug delivery approaches (acute -vs- chronic indications, immunogenicity, etc.)?
Denali Fellow, Adjunct Associate Professor at the University of Minnesota, & IBBS Vice President, Denali Therapeutics
Robert Thorne joined Denali Therapeutics as a Denali Fellow in 2018 after over 10 years on the faculty at New York University School of Medicine and the University of Wisconsin-Madison. Robert’s research interests include the biology of CNS barriers, routes of administration bypassing the blood-brain barrier (BBB), CNS biodistribution, and receptor-mediated transcytosis across the BBB. He is currently the Vice President of the International Brain Barriers Society. Robert has served as chair for the 2016 ‘Barriers of the CNS’ Gordon Research Conference, 2019 AAPS-IBBS CNS delivery workshop, and an upcoming 2023 ‘Drug Delivery to the Brain’ Keystone Conference.
Spotlight Presentation: Enhanced Delivery of Biotherapeutics in Nonhuman Primates
- Altasciences Preclinical Services
- Specialized delivery methods in nonhuman primates
- Case studies: Gene Therapy & Stem Cell Transplantation
Vice President Toxicology, Altasciences
Norbert Makori is Vice President of Toxicology, Altasciences. He has over 20 years of industry experience as a toxicologist (general and reproductive toxicology) and immunotoxicology, including study director role and management. Prior to joining Altasciences, Norbert was Director of the General Toxicology group at Charles River Laboratories in Ashland, Ohio. Prior to that, he held the position of Director of Toxicology and Immunotoxicology at WIL Research Laboratories. Norbert is a Diplomate of the American Board of Toxicology (DABT), trained as a veterinarian and has a PhD in Comparative Pathology from the University of California in Davis.
1:55 – 2:55pm
- Can novel biomarkers replace traditional clinical outcome measures? Eg easy to deploy technologies to detect cognitive, fine motor deficits, or bulbar symptoms?
- Can novel biomarkers replace traditional fluid or imaging biomarkers? Eg easy to deploy technologies to predict amyloid positivity for AD etc.
- Can active or passive monitoring through wearables or smartphones replace the in person clinic assessments?
- Are these technologies ready for prime time? What is the path to validate (technical and clinical) these novel technologies?
VP Translational Sciencies, Tranquis Therapeutics Inc
Enchi Liu, Ph.D., has more than 20 years of experience in drug development, spanning translational, early, and late development. Her research spans CNS (mood, pain, sleep), neurodegenerative disorders (AD, HD, ALS, PD), and rare diseases (AL and ATTR amyloidosis, LSDs). She is expert in integration of biomarkers into preclinical and development programs. Prior to joining Tranquis, she was PTL for Neurology at Annexon, lead Clinical and Translational Sciences at Escape Bio, PTL at Prothena, and Clinical Biomarker Leader at Janssen for multiple CNS programs. She did postdoctoral research at the University of Arizona and received her Ph.D. from Cornell University.
Drug Delivery
Operant conditioning procedures have played an important role in psychopharmacology research both in academic and applied drug discovery settings. The purpose of this presentation is to describe three examples from a rodent based operant platform separately established both at IVS and TPH, and now combined into a single unit within the merged company, has contributed to recent CNS drug discovery efforts.
- The VMAT2 inhibitor tetrabenazine as a translational approach to discover new treatments for depression.
- Operant procedures designed to investigate both “micro” and “macro” dose psychedelics.
- Development of an operant platform approach to discover new treatments for ADHD
Chief Scientific Officer, Transpharmation
Guy Higgins has 30 years international experience in CNS R&D with major pharmaceutical companies (Glaxo-Wellcome,
Hoffmann-La Roche, Schering-Plough, NPS Pharmaceuticals). He has success in developing translational models and bringing multiple NCEs to clinical development. Guy is also the Adjunct Professor of Pharmacology, U. Toronto, with 100+ peer-reviewed papers in Neuroscience Drug Discovery.
- Are there advantages in complementing in vitro electrophysiological studies
performed in rodent tissues with data obtained from human neurons in R&D and - preclinical studies?
What progress have been achieved by using human iPSCs-derived neuronal cultures
for in vitro electrophysiological studies? - Can 3D brain organoids be a useful tool and present significant advantages to study neurodevelopment using electrophysiology?
- Could surgical wastes be valorized for in vitro electrophysiological studies in human
tissues?
Chief Executive Officer, Ephyx Neuroscience
Txomin has over 8 years of experience planning and performing in vitro electrophysiology studies for R&D and preclinical projects. He holds a PhD in neurobiology from McGill University (Montréal, QC, Canada), during which he obtained twice the Excellence award from the Integrated Program in Neuroscience. After postdoctoral experiences specialized in electrophysiology, he co-founded EphyX Neuroscience with the goal to apply his skills and expertise to boost neuroscience-related preclinical development by providing highly functional electrophysiological data from rodent and human neurons.
Spotlight Presentation: Enhanced Delivery of Biotherapeutics in Nonhuman Primates
- Altasciences Preclinical Services
- Specialized delivery methods in nonhuman primates
- Case studies: Gene Therapy & Stem Cell Transplantation
Vice President Toxicology, Altasciences
Norbert Makori is Vice President of Toxicology, Altasciences. He has over 20 years of industry experience as a toxicologist (general and reproductive toxicology) and immunotoxicology, including study director role and management. Prior to joining Altasciences, Norbert was Director of the General Toxicology group at Charles River Laboratories in Ashland, Ohio. Prior to that, he held the position of Director of Toxicology and Immunotoxicology at WIL Research Laboratories. Norbert is a Diplomate of the American Board of Toxicology (DABT), trained as a veterinarian and has a PhD in Comparative Pathology from the University of California in Davis.
Poster Session: MRI-guided, convection enhanced delivery of cell and gene therapies to the central nervous system of nonhuman primates
- Cell and gene therapies hold the promise to provide curative treatments for previously untreatable diseases, including those impacting the central nervous system (CNS). Unlike traditional drugs, where systemic delivery proves efficacious, many cell and gene therapies require precise administration routes to access specific cell populations affected by disease processes, minimize adverse effects, and reduce dose levels.
- In preclinical studies of cell and gene therapies, sponsors are exploring various routes of administration and this poster will focus on state-of-the-art surgical delivery techniques in nonhuman primates for safety assessment of therapies targeting the CNS.
- Data presented herein will cover workflow considerations, key benefits, and limitations of MRI-guided convection-enhanced delivery in preclinical studies.
Senior Director, Veterinary Medicine & Translational Science, ClearPoint Neuro, Inc.
Geary Smith currently serves as the Senior Director of Veterinary Medicine and Translational Science at ClearPoint Neuro, Inc. Previously, he directed a facility at the Children’s Hospital of Philadelphia Research Institute that partnered with faculty and external sponsors to complete proof-of-concept and GLP-compliant, nonclinical studies for novel therapeutics and medical devices. His work, utilizing a variety of nonhuman primate species, focuses on neurological disease. He graduated from the University of Georgia School of Veterinary Medicine, completed a residency in Laboratory Animal Medicine at Emory University, and completed a fellowship in nonhuman primate medicine at the Yerkes National Primate Research Center.
Non-invasive methods to increase brain drug uptake
- What are some novel molecular targeting approaches for optimizing delivery of potential therapeutics across the BBB in order to improve brain drug uptake?
- Which experimental methods can we use to accurately measure and compare the biodistribution of novel therapeutics in the brain?
- Understanding the biodistribution of drug candidates and their targets is critical to determining potential therapeutic efficacy. What are the optimal biodistribution profiles (at the levels of both cellular and brain-region specificity) associated with different CNS disease indications?
Principal Scientist/Lab Leader- Microscopy & Image Analysis, Denali Therapeutics
Meredith Calvert has 15 years+ experience building and leading bioimaging platforms. She develops quantitative imaging tools for exploring cellular mechanisms and leveraging image data in biological research. Meredith’s team at Denali uses advanced imaging to explore the biodistribution of experimental therapeutics and biomarkers, furthering drug discovery efforts toward defeating neurodegeneration.
Meredith received a Ph.D in Microbiology from the University of Virginia. She has directed Imaging and Biocomputing facilities in Singapore and at the Gladstone Institutes, San Francisco, taught and directed graduate courses and imaging workshops, and served in leadership roles with the Association of Biomolecular Resource Facilities, the Western Association of Core Directors, and is a founding board member of BioImaging North America.