CNS Therapeutics Xchange
Europe 2021, December 2nd

Welcome to hubXchange’s European CNS Therapeutics Xchange 2021, 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, Gene Therapies and Drug Delivery.
Take advantage of this unique highly interactive meeting format designed for maximum engagement, collaboration and networking with your peers.

Please note this is now a fully VIRTUAL meeting.

Novel Targets

Time

Titles and Bullets

Facilitator

8:00 – 8:30am

Registration

8:30 – 9:00am

Opening Address & Keynote:

Back and Forward Translational Research – How to close the circle?

Massimiliano Bianchi

Founder / President & CEO
Ulysses Neuroscience

Professor Massimiliano (Max) Bianchi launched Ulysses Neuroscience Ltd. in 2019 following over 20 years of international neuropharmacology R&D and executive management experience in industry and CRO including Transpharmation Ireland Ltd (Ireland), MAPREG (France) and GlaxoSmithKline (UK and Italy). A native of Italy, Massimiliano originally studied veterinary medicine in Sassari and Milan before obtaining his PhD in psychopharmacology at the University of Nottingham and embarking on a career in scientific research and the pharmaceutical industry. He has dedicated his scientific career to explore the role of neuronal plasticity and cytoskeletal dynamics in the pathogenesis and treatment of neuropsychiatry and neurodegenerative disorders. Massimiliano is also Adjunct Assistant Professor in the School of Psychology in Trinity College Dublin and Adjunct Associate Professor in the Department of Chemistry at Maynooth University. His company Ulysses Neuroscience Ltd. was founded with the mission to start a New Journey Against Brain Disorders based on Patient-Centricity, Translation Research and Social Responsibility. Ulysses Neuroscience currently works with several pharmaceutical companies around the world to accelerate drug discovery in neuroscience by providing cutting-edge capabilities bridging preclinical and clinical research in neuropsychiatry and neurodevelopmental disorders. Proud of its rapid growth, Ulysses can count on 12 research dedicated staff and two research sites in the Republic of Ireland.

9:05 – 10:05am

Novel Targets in CNS Research- Past, Present and Future?

What are the future exciting drug targets for pain, neurology and psychiatry?
Discuss the current status of our existing “hot” CNS drug targets
Explore the barriers to investigating a truly novel target
Should we re-explore “failed” drug targets of the past

Magnus Walter

Head of Discovery Chemistry, Screening Biology and Operations
AbbVie

Magnus Walter leads Abbvie’s Discovery organisation in Ludwigshafen, Germany focused on the development of disease modifying treatments for neurodegenerative diseases. Drug discovery activities range from target identification to clinical candidate delivery and cover medicinal chemistry, synthetic technologies, assay development, cell culture and high content imaging. Before joining Abbvie in 2018 Magnus had global responsibility for Neurodegeneration Medicinal Chemistry at Eli Lilly and was based in the UK. He has broad experience in medicinal chemistry and his group contributed to drug discovery projects in neuroscience, immunology and metabolic diseases. He made significant contributions to the discovery of several clinical candidates that progressed into late-stage clinical trials.

10:10 – 10:40am

1-2-1 Meetings/Networking Break

10:40 – 11:10am

1-2-1 Meetings / Networking Break

11:10 – 11:20am

Morning Refreshments

11:20am – 12:20pm

Preclinical Models with Predictability and Human Translatability- Can Porcine Models Bridge this Gap?

Differences in sex with regards to therapeutic development
Similarities of porcine skin, innervation and vascularization to humans
Shortcomings of rodent models and the impact on success in CNS drug development
Cost as a factor in choice of porcine models

Sigal Meilin

Chief Scientific Officer
MD Biosciences

Sigal Meilin, PhD, is a graduate of Bar Ilan University in Tel Aviv, Israel. Prior to taking the helm as Chief Scientific Officer of MD Biosciences Innovalora, Ltd. in 2004, Sigal headed the preclinical program at an early-stage company. At MD Biosciences, Sigal established the full preclinical research program with well-known as well as novel rodent models. The research capabilities were expanded with new insight and understanding, eventually encompassing all aspects of in vitro testing, cell-based assays and electrophysiology. In vivo research capabilities were augmented with porcine models which have proven to be a new standard and milestone in translational development. As Chief Scientific Officer, Sigal Meilin has the privilege of seeing many novel therapeutic classes, many different pathways and administration routes, giving a unique perspective on development trends, successes and failures.

12:20 – 1:20pm

Networking Lunch

1:20 – 1:50pm

Spotlight Presentation

Introducing diversity in non-clinical and translational CNS models for better prediction of clinical outcomes

Sigal Meilin

Chief Scientific Officer
MD Biosciences

1:55 – 2:55pm

Triaging Your Drug Targets to Find the Most Relevant Lead

How to triage potential targets based on the most validated human biology and genetics evidence
Explore the different approaches to defining standards for excluding drug targets
Explore how community research experience and confidence in novel targets can de-risk development
Discuss the risks of a research community-based approach to investigating new targets

Jenny Laird

Vice President, Search & Evaluation – Pain & Neurodegeneration
Eli Lilly

Jenny Laird, Ph.D., D.Sc. is Vice-President, Search & Evaluation Pain & Neurodegeneration at Eli Lilly and Company, based at Lilly’s European Headquarters near London, UK. She leads a global Search & Evaluation team complementing Lilly’s internal R&D efforts in Neuroscience by evaluating, in-licensing, or acquiring assets and technologies and by advancing molecules through discovery and development in collaboration with external partners including venture firms. Prior to joining Lilly in 2012, Dr. Laird held roles of increasing responsibility in drug discovery and development at AstraZeneca and Merck, and as an academic PI. Dr. Laird received doctorates from University of Bristol, UK and University of Alicante, Spain. She has served on editorial boards of journals in the field of neuropharmacology, and as an honorary Professor of Pharmacology at McGill University, Canada.

3:00 – 3:30pm

1-2-1 Meetings / Networking Break

3:30 – 4:00pm

1-2-1 Meetings / Networking Break

4:00 – 4:10pm

Afternoon Refreshments

4:10 – 4:40pm

Poster Session

Target discovery – the Euretos approach

Integrating gene-disease annotations and biological networks from multiple sources
Predicting novel gene-disease associations from known genetic associations
Contextualising results with public knowledge in the Euretos platform

Sophie Roerink

Head of translational science
Euretos

Sophie Roerink joined Euretos in 2019. She has a MSc degree in Pharmaceutical Sciences and a PhD in Medicine from Leiden University – studying cancer biology in model organisms. During her postdoctoral work at the Cancer Genome Project in the Sanger Institute and the Princess Maxima Center for pediatric oncology she has worked on understanding cancer biology from cancer genomics data. Within Euretos she heads up the translational science team – translating research questions from customers into data science solutions and/or user-friendly workflows in the Euretos AI Platform

4:45 – 5:45pm

Neuroinflammation (NI) – identifying diagnostic, prognostic and target engagement biomarkers for novel NI drug targets

Cause or effect and when to intervene – are there biomarkers that faithfully recapitulate and track the CNS disease state(s)?
Are current NI biomarkers accurate, reproducible and cost effective or are better biomarkers in development?
Discuss the current developments of drug targets for neuroinflammation and how are they being assessed preclinically?
How to effectively use biomarkers for translation into patients and identify / stratify patients?

Lee Dawson

Vice President, Neuroscience
Cerevance

Lee is Vice President of Neuroscience at Cerevance, in Cambridge, UK. Cerevance is a neuroscience focussed biotech start up using its NETSseq technology to discover and develop truly novel therapeutics for diseases such as Parkinson’s, ALS and dementia. Lee is a neuropharmacologist with over 25 years in the Pharmaceutical industry having held positions across biotech and large Pharma organisations including Wyeth Research (in the UK and US), GlaxoSmithKline, Eisai and Otsuka Pharmaceuticals. During his career Lee has been responsible for global strategic input on all aspects of R&D and has lead projects from early target identification, through drug discovery to phase II proof of concept testing.

5:45 – 6:45pm

Evening Drinks Reception

Preclinical

Time

Titles and Bullets

Facilitator

8:00 – 8:30am

Registration

8:30 – 9:00am

Opening Address & Keynote:

Back and Forward Translational Research – How to close the circle?

Massimiliano Bianchi

Founder / President & CEO
Ulysses Neuroscience

9:05 – 10:05am

Clinical Studies topic:
Paediatric development in CNS disorders

Pediatric indication selection
Pediatric dosing
Pediatric endpoints

Frank Tennigkeit

Senior Director Pediatric Development
UCB

Drug Development expert with >12 years experience in the pharmaceutical industry.
• Medical background in Pharmacology and Neuroscience. Strong experience in clinical development, Neurology (Alzheimer’s Disease, Epilepsy, Parkinson’s Disease and PSP) and orphan autoimmune disorders.
• Design, negotiation and implementation of pediatric development plans and pediatric clinical studies phases 1-3.
• Extensive experience in planning, executing, publishing and submitting clinical development programs for new chemical entities, biologics, and vaccines. NDA and submission experience, 5 successful approvals (US, EU, J, CN).

10:10 – 10:40am

1-2-1 Meetings / Networking Break

10:40 – 11:10am

1-2-1 Meetings / Networking Break

11:10 – 11:20am

Morning Refreshments

11:20am – 12:20pm

Interest of EEG biomarkers to address challenges in CNS drug discovery

Explore the translational aspects of EEG biomarkers from animal to human
Discuss, where are the current limits of EEG applications?
What novel solutions do we need to develop with EEG in the near future?

Hedi Gharbi

Head of EMEA/APAC Sales & Head of Marketing
Synapcell

He’s holding a Master’s degree in Biomedical imaging and a MBA on Biotech and Pharmaceutical management. After 10 years working across cutting-edge medical devices, molecular imaging systems and consulting firms, Hedi joined SynapCell in 2016 as Marketing Manager. In this role, he initiated a 360° revamp of the company’s value proposition, branding and launched Cue®, the company’s ultimate EEG platform acting as a Go/No-Go asset for drug developers. In 2018, Hedi was promoted Head of Europe and Asia-Pacific Sales on top of his Marketing role to get closer to clients’ needs. This dual role allows him today to have an extensive knowledge of the current needs of biopharma industry towards drug discovery for CNS disorders, and how to address them.

Venceslas Duveau

Head of Product and Portfolio Management
Synapcell

Venceslas holds a PhD in Neuroscience and Neuropharmacology from the University of Bordeaux, France. He then did a post-doc at the Institute of pharmacology and toxicology at the University of Zurich. Venceslas has been working at SynapCell since 2012, first as Neuroscientist then as Head of Science. In May 2021, he was appointed Head of Product and Portfolio Management. Venceslas therefore brings more than 10 years’ experience in drug discovery and brain disorders. As SynapCell’s Head of product and portfolio management, he is at the cutting edge of Neuroscience research through the implementation of drug discovery programs for customers or for internal R&D projects.

12:20 – 1:20pm

Networking Lunch

1:20 – 1:50pm

Spotlight Presentation

Introducing diversity in non-clinical and translational CNS models for better prediction of clinical outcomes

Sigal Meilin

Chief Scientific Officer
MD Biosciences

1:55 – 2:55pm

Improved human-relevant models (including brain organoids)

What have we learned from working with human iPSC cells?
What are the current applications of brain organoid models, are any ready for disease-modelling?
What can we expect the next 10 years of research to deliver in this space?
How can academia and industry work together to advance this field?

Peter Atkinson

Senior Director, Head of External Innovation, Neurology Business Group
Eisai

Peter Atkinson, PhD is Senior Director and Head of External Innovation Neurology based at Eisai’s EMEA HQ in Hatfield. He leads a global team responsible for scouting and alliance management of early discovery collaborations and investment opportunities established to accelerate Eisai’s Neurology portfolio. Prior to joining Eisai Peter contributed to a variety of neuroscience drug-discovery programmes in Wyeth (later Pfizer) based in the USA, and SmithKline Beecham (later GlaxoSmithKline) based in the UK.

3:00 – 3:30pm

1-2-1 Meetings / Networking Break

3:30 – 4:00pm

1-2-1 Meetings / Networking Break

4:00 – 4:10pm

Afternoon Refreshments

4:10 – 4:40pm

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?

Jack Prenderville

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

Preclinical Development Strategies to De-Risk Clinical Translation

How much confidence can you gain in the biology of a target using preclinical studies
What value do genetics and data from human tissues bring
What are the relevant human and clinical endpoints that are equivalent to your preclinical measurements?

Janet Brownlees

Director, Discovery Research
MSD

Dr Janet Brownlees is a Director of Neuroscience Biology at the Merck Sharpe and Dohme (MSD) Discovery Research Centre in London, UK with 18 years of drug discovery experience. She currently leads a biology team working on the mechanisms associated with neurodegeneration and diseases of ageing. Dr Brownlees received a BSc (Hons) and PhD in Biochemistry from the Queen’s University of Belfast. Before joining MSD in 2019, she has held roles of increasing responsibility in academia and the drug discovery industry at King’s College London, Glaxo Smith Kline and LifeArc. She currently leads internal projects, external collaborations and serves on grant review boards.

5:45 – 6:45pm

Evening Drinks Reception

Clinical Studies

Time

Titles and Bullets

Facilitator

8:00 – 8:30am

Registration

8:30 – 9:00am

Opening Address & Keynote:

Back and Forward Translational Research – How to close the circle?

Massimiliano Bianchi

Founder / President & CEO
Ulysses Neuroscience

9:05 – 10:05am

Best approaches to patient stratification in psychiatric disease

How to de-risk clinical development by ensuring your program’s trial patient population composition is correct
How to effectively utilize biomarkers, multi-omics profiling and patient data to rationally define patient subgroups from heterogeneous patient populations?
What are the current possibilities of AI and machine learning in identifying treatment responders and predicting disease progression?

Gary Gilmour

Vice President, Preclinical Research
COMPASS Pathway

Gary Gilmour is Vice-President of Preclinical Research at COMPASS Pathways. Gary studied Pharmacology at the University of Glasgow before completing a DPhil in Neuroscience at the University of Oxford. He has built his career in the pharmaceutical industry and has led novel target and discovery projects across a broad range of CNS indications. His approaches to drug discovery place high emphasis on translational validation and establishing a deeper understanding of the biological substrates underlying different disease-related symptoms. Gary is a strong advocate for pre-competitive scientific exchange, and his leadership has significant shaped research strategies on common problems in neuroscience drug discovery.

10:10 – 10:40am

1-2-1 Meetings / Networking Break

10:40 – 11:10am

1-2-1 Meetings / Networking Break

11:20am – 12:20pm

Approaches to patient-centricity in pharma: a journey from stakeholders to allies

What is the aim of patient-centricity? (e.g. integrating research seamlessly and effectively into their lives)
What is currently being done in the pharma industry to engage with patients (e.g. awareness days, focus groups)?
What kind of patient-centric initiatives should we focus on (e.g. accessible sample collection, advisory boards)?

Carolina De Pasquale

Patient Insight Manager
Ulysses Neuroscience

Carolina De Pasquale, originally from Italy, began her academic career with a B.A in Multimedia at Dublin Institute of Technology before falling in love with research. She then pursued a PhD in behavioural signal processing at the Technical University Dublin (TUD) where she also worked as a teaching assistant and lecturer.Carolina has worked on speech acoustic coordination in human-human interactions in clinical settings to measure interpersonal rapport in psychotherapy. She worked in Bianchi’s Lab in molecular neuroscience for two years prior to moving to Ulysses Neuroscience Ltd. Having developed a strong interest in neuroscience and psychology during her career so far and in her personal life, she has come to

Ulysses Neuroscience Ltd. as Patient Insight Manager and Research Associate. As Patient Insight Manager she is responsible for working on patient relations and creating opportunities for
patients to express their priorities and needs, and for helping their communities to come together, working on advocacy and awareness alongside them. As Research Associate she works on the in vivo side of things, mostly with rats and the IFN-alpha model. She is also the lab designated trainer for rat procedures and she is part of the new rat welfare committee in Trinity College Dublin.

12:20 – 1:20pm

Networking Lunch

1:20 – 1:50pm

Spotlight Presentation

Introducing diversity in non-clinical and translational CNS models for better prediction of clinical outcomes

Sigal Meilin

Chief Scientific Officer
MD Biosciences

1:55 – 2:55pm

Next Generation CNS Biomarkers Discovery – Current Opportunities with Multi-Omics and Patient Data

How can we qualify novel biomarkers and their clinical relevance for use in early phase clinical trials?
What are the advantages and disadvantages of current biomarker discovery platforms?
What data are necessary to ensure that novel biomarkers have good clinical translatability?
• Usefulness preclinical biomarker discovery

Astrid Valles-Sanchez

Associate Director, Translational Biology
uniQure

Within the Preclinical Biology team at uniQure, Astrid Vallès-Sánchez is leading one of the early-stage research groups, from idea to preclinical proof-of-concept, focusing on central nervous system (CNS) indications. As a molecular neurobiologist, she has a broad interest in the molecular basis of brain functioning in health and disease. Dr. Vallès-Sánchez obtained her PhD at the Universitat Autònoma de Barcelona (Spain), and held postdoctoral positions at Solvay Pharmaceuticals, Radboud University and Maastricht University (The Netherlands). Her previous work in both academic and pharma settings includes basic research and translational activities, from target discovery to preclinical disease modeling, in the fields of neuroendocrinology, neurodegeneration and neuroplasticity. Since joining uniQure at the end of 2016, she has been involved in uniQure’s Huntington Disease (HD) gene therapy program, with focus on preclinical work in small and large animal models to evaluate biofluid and imaging candidate efficacy measures. Next to HD, her team leads the preclinical activities of the spinocerebellar ataxia 3 (SCA3) and other undisclosed CNS programs. With almost 30 peer-reviewed publications (ORCID iD 0000-0002-9377-2880), her H-index is 16.

3:00 – 3:30pm

1-2-1 Meetings / Networking Break

3:30 – 4:00pm

1-2-1 Meetings / Networking Break

4:00 – 4:10pm

Afternoon Refreshments

4:10 – 4:40pm

Preclinical Topic Poster Session

A neural circuits platform to accelerate and de-risk preclinical neurotherapeutic drug development

Abstract:

Correcting aberrant circuit signatures will be essential for the generation of new neurotherapeutics. The power of the Inscopix neural circuit platform is a complete sample prep-to-results workflow for brain mapping that enables our clients to expedite and de-risk preclinical neurotherapeutic drug development with a quantitative, data-driven approach that informs better decisions predictive of clinical success.

Basile Siewe

Senior Director, Business Development-Global Biopharma
Inscopix

Basile joined Inscopix in 2021. Basille worked previously at Jackson Labs where he was Director of Business Development for the in vivo services. Prior to that he had roles at Thermo and Affymetrix. Basile holds a Ph.D. from the Technical University of Munich in Germany in Molecular Biology/Immunology and was an Instructor and Research Fellow at the Rush University Medical Center in Chicago. At Inscopix, Basile works closely with the Commercial and Translational Science teams to grow our pharma and biotech business.

4:45 – 5:45pm

Selecting Clinical Endpoints and Assessing Clinical Outcomes

How does one assess on-target effects in the CNS using imaging, fluid biomarkers and objective functional measures? What are the relevance of these measures to disease?
Which digital biomarkers align with meaningful clinical endpoints?
What are regulatory perspectives on the incorporation of objective biomarkers in the context of clinical endpoints?
What are the best methods for tackling variability in clinical endpoints? Are there opportunities here for unique clinical designs, wearables, in home monitoring, AI or other approaches?

Karla Allebrandt

Senior Clinical Biomarker Lead CNS / Director
Boehringer Ingelheim

Karla Allebrandt is a Director of Experimental Medicine Studies and acts as Clinical Biomarker Lead in Early Clinical Development Projects in the CNS TA at Boehringer Ingelheim. Her work is centered around Precision Psychiatry, both through leading large external collaboration studies, as well as, in tailoring strategies for clinical trials. In addition, as Digital Biomarker Strategy Lead, she brought BI stakeholders together for review and decision making on remote patient monitoring solutions in the CNS TA. As a result, she leads studies to explore and validate digital biomarkers in combination with neurophysiological markers and conventional clinical assessments. Before joining BI, Karla developed and patented approaches to identify biomarkers of CardioMetabolic disease progression at Sanofi-Aventis. She holds a PhD in Neurogenetics, and a Habilitation in Medical Psychology from the University of Munich (LMU). There she worked as a Junior Group Leader on Epidemiology and Genetics of Sleep & Circadian Rhythms, acting as a KOL in that field.

5:45 – 6:45pm

Evening Drinks Reception

Gene Therapies

Time

Titles and Bullets

Facilitator

8:00 – 8:30am

Registration

8:30 – 9:00am

Opening Address & Keynote:

Back and Forward Translational Research – How to close the circle?

Massimiliano Bianchi

Founder / President & CEO
Ulysses Neuroscience

9:05 – 10:05am

Effective Vector Engineering and Drug Delivery Approaches for Gene Therapies

Discuss the strengths and drawbacks of viral vs. non-viral vectors
Explore the promotors and regulators needed for cell-specific expression
What drug delivery and novel technologies will enable gene therapy that maintains efficacy with low off-target effects

Michael Linden

UCB Gene Therapy Platform leadership
UCB

After receiving his Ph.D. in Biochemistry and Molecular Biology at the University of Zürich, Switzerland, Prof. Linden trained in Molecular Virology at Cornell University Medical College, N.Y. After his postdoctoral training he joined the faculty at Mount Sinai School of Medicine in New York, where he continued to study the biology and gene therapy potential of adeno-associated virus (AAV). In 2007, he moved continents to set up a new research group at King’s College London as the chair in Molecular Virology.
His contributions to the field include the biochemical and structural determination of the AAV Rep proteins, which orchestrate all aspects of the AAV life cycle, the characterization of AAVS1 and its description as a human safe harbor locus as well as the proposal of a mechanism of site-specific integration. During these studies Prof. Linden’s laboratory has demonstrated the potential of using embryonic stem cells for gaining insights into complex viral mechanisms. Finally, the laboratory designed a novel, neurotropic AAV capsid, AAV-TT.

10:10 – 10:40am

1-2-1 Meetings / Networking Break

10:40 – 11:10am

1-2-1 Meetings / Networking Break

11:10 – 11:20am

Morning Refreshments

11:20am – 12:20pm

No session

12:20 – 1:20pm

Networking Lunch

1:20 – 1:50pm

Spotlight Presentation

Introducing diversity in non-clinical and translational CNS models for better prediction of clinical outcomes

Sigal Meilin

Chief Scientific Officer
MD Biosciences

1:55 – 2:55pm

Translational imaging techniques for gene therapy (AAV, ASO, siRNA)

Current tools for assessing translational biodistribution from mouse to man – what are the advantages and shortcomings
Correlation between the efficacy of gene modulation and biodistribution by non-invasive imaging modalities?
Additional biomarkers needed to assess target engagement and efficacy following gene therapy?

Peter Larsen

Principal Scientist, Head of Preclinical Imaging, Neuroscience
Janssen

Peter obtained his Ph.D. from the University of Calgary where his work focused on aspects of remyelination and multiple sclerosis. He followed this with postdoctoral studies at the University of Southern Denmark in the area of neuroimmunology. In 2007, he joined H. Lundbeck where he worked for 9 years within psychosis and in implementation of novel imaging technologies. Since 2017, he has continued his career in the Neuroscience Therapeutic Area at Janssen Pharmaceutica, leading the preclinical imaging team to support internal Alzheimer’s and Parkinson’s projects with various imaging techniques.

3:00 – 3:30pm

1-2-1 Meetings / Networking Break

3:30 – 4:00pm

1-2-1 Meetings / Networking Break

4:00 – 4:10pm

Afternoon Refreshments

4:10 – 4:40pm

Preclinical Topic Poster

Everything’s backwards: Rethinking translation in Fragile X Syndrome

Overview on preclinical models of neurodevelopmental disorders and Fragile X Syndrome (FXS)
Ulysses Neuroscience Ltd. behavioural and molecular data using the Fmr1 knockout (KO) mouse as a model of FXS
How can researchers use back-translation from patients with FXS to design better experiments in these mice?
A brief overview of the clinical projects on FXS currently undergoing at Ulysses Neuroscience Ltd

John Kealy

CSO
Ulysses Neuroscience

Dr. John Kealy received his degree in neuroscience from Trinity College Dublin before conducting a PhD focused on synaptic plasticity at Maynooth University. John spent over 10 years working in academia using electrophysiological, neurochemical and genetic techniques to understand brain activity and metabolism across multiple levels – the neuron, glia, and the vasculature – in a number of disorders including schizophrenia, 22q11 deletion syndrome, delirium, and dementia. At Ulysses Neuroscience, John is Chief Scientific Officer and Academic Liaison Manager, and he oversees translational projects on Fragile X Syndrome, Charcot-Marie-Tooth disease, and depression. He also supervises the EEG / Operant tasks projects currently under development in the new Ulysses Neuroscience department at Maynooth University.

4:45 – 5:45pm

CNS gene therapy – current drug delivery and clinical development challenges & future opportunities

What drug delivery- and novel technologies (will) enable CNS gene therapy that achieves efficacy with low off-target effects (e.g. cell-type specificity)?
What strategies can be deployed to manage potential immune responses and dose-dependent toxicities to gene therapy?
What would be an ideal translational strategy (pre-clinical models, biomarkers etc) to develop CNS gene therapies for polygenic CNS diseases in the future?

Vishal Sahni

Director, Corporate Business Development & Strategy
Lundbeck

Vishal is a Corporate Business Development & Strategy Director at H. Lundbeck A/S, where he is searching for in-licensing and collaboration opportunities for neurological and psychiatric indications.
He has expertise in developing novel drugs for stratified patient populations using human genetics and in applying machine learning and artificial intelligence approaches to drug discovery and development. Prior to joining Lundbeck, Vishal led teams at several stages of the drug discovery-, development- and commercial value chain, initially at Eisai and subsequently at GlaxoSmithKline and MSD, working across Europe, the United States, the Middle East and Asia to support the development of small molecule-, biologics- and gene therapy-medicines.
Vishal holds a PhD in Developmental Neurobiology from University College London.

5:45 – 6:45pm

Evening Drinks Reception

Drug Delivery

Time

Titles and Bullets

Facilitator

8:00 – 8:30am

Registration

8:30 – 9:00am

Opening Address & Keynote:

Back and Forward Translational Research – How to close the circle?

Massimiliano Bianchi

Founder / President & CEO
Ulysses Neuroscience

9:05 – 10:05am

Exploring the Current Opportunities of Shuttle Vehicles and Transcytosis Drug Delivery Technologies

Discuss the most relevant targets in drug delivery to the CNS
Explore chemical modification and ligand conjugation as a way to shuttle therapeutics across the BBB
Explore viral and non-viral particle-based shuttling of therapeutics across the BBB
Discuss which of these strategies can help existing drug candidates

Habib Baghirov

Senior Scientific Business Analyst
Roche

Originally a medical doctor, I first worked in medical research in my home country before moving to Finland where I completed an MSc in Biomedical Imaging, investigating nanoparticle transport across the BBB. After graduation, I researched cell invasion in glioblastoma, primarily using advanced light microscopy and image analysis. Later on, I moved to Norway to do a Ph.D. in focused ultrasound-mediated transport across the BBB, using medical technology, imaging and image analysis. After spending a year in Ireland where I researched siRNA delivery into the brain, I moved to Roche in Switzerland to continue working on the delivery of biologics across the BBB, with a strong focus on microscopy, image analysis and data analysis. At that time, I became fascinated with computational biomedicine and eventually moved to Roche in Poland, to expand my skillset and gain new perspectives by working on digital biomarkers and therapeutics in neurodegenerative diseases.

10:10 – 10:40am

1-2-1 Meetings / Networking Break

10:40 – 11:10am

1-2-1 Meetings / Networking Break

11:10 – 11:20am

Morning Refreshments

11:20am – 12:20pm

Scientific and operational challenges to blood-brain administration

Discuss the scientific challenges to delivering macromolecules and advanced therapeutics to the brain: vascular, LP and transcranial
Exploring the operational site oversights and training requirements for single administrations and implantable infusion pumps
Considerations for maintaining the blind: sham controls, imaging, coordination with pharmacy, and blinding plans
How should procedural and investigational product related adverse events be managed and documented

Laura Omoboni

Senior Director, Clinical Trial Management
Medpace

Laura Omoboni, MS, is Senior Director, Clinical Trial Management, with over 20 years of clinical trial experience. She has managed multiple global, complex Phase I-III clinical trials as Program Director and Global Project Manager for CNS, oncology, acute-care and gene therapy disease studies involving multidisciplinary teams and vendors. Laura is a Subject Matter Expert for Medpace’s rare disease activities. She received her Master of Science in Chemistry and Pharmaceutical Technologies from University of Milan.

12:20 – 1:20pm

Networking Lunch

1:20 – 1:50pm

CNS Therapeutics Xchange
Europe 2021, December 2nd

Novel Targets

Preclinical

Clinical Studies

Gene Therapies

Drug Delivery

Lead Partner

Partners

© Copyright 2023 by Weblify & Webtec

CNS Therapeutics XchangeEurope 2021, December 2nd

Novel Targets

Preclinical

Clinical Studies

Gene Therapies

Drug Delivery

Lead Partner

Partners

CNS Therapeutics Xchange
Europe 2021, December 2nd