CNS THERAPEUTICS XCHANGE
EAST COAST 2023
Boston
May 24, 2023
May 24, 2023
Welcome to hubXchange’s CNS Therapeutics Xchange East Coast 2023, 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 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 an In-Person meeting.
VENUE DETAILS: Hilton Boston Woburn Hotel, 2 Forbes Road, Woburn MA 01801
SNAPSHOTS OF DISCUSSION TOPICS
- Novel Non-Invasive Targeted Drug Delivery to the CNS
- Emerging non-Amyloid targets for Alzheimer’s disease
- Opportunities from emerging therapeutic modalities: Target selection in an evolving druggability landscape
- Methods to increase the success rates of drugs entering clinical phases
- Relevant in vitro mechanistic profiling to guide SAR and downstream in vivo studies
- Utilizing clinical biomarkers to enhance drug development in neurodegenerative diseases
- Clinical trials for rare diseases: challenges and opportunities
- Non-invasive methods to increase brain drug uptake
- Transcytosis-mediated brain delivery for proteins, antibodies, oligonucleotides, and small molecules
- Novel strategies of drug delivery into the brain
Full Xchange Agenda
Click on each track for detailed agenda
Novel Targets
08:30 – 09:00
Opening Address & Keynote Address
Expanding the horizon of EEG applications to accelerate CNS therapeutics development
The process of drug discovery is both costly and time-intensive. Through this keynote, we will explore the high translational potential of electroencephalography (EEG), a technique that finds use in both clinical and preclinical settings. The relevance of this tool will be illustrated by examples from epilepsy research. We’ll discuss how the development of more advanced EEG technology and analysis methods is also anticipated to further enhance the usefulness of EEG in understanding brain function and developing new therapeutic strategies against neurodegenerative, neurodevelopmental or rare CNS disorders.
Head of Technology, SynapCell
Julien holds a PhD in Neuroscience from the University of Lyon, France. Julien joined SynapCell in 2017 after two post-docs in different structures in Toronto. Since then, he manages R&D Preclinical projects and develops new innovative solutions for sponsors. With over 12 years’ experience in the field, he is well-versed in the emerging technologies and industry trends.
Scientific Sales Manager, SynapCell
After a MSc in Neuroscience and in international business in science, Hugo now manages the USA and LATAM zone. His background allows him to understand the current needs of the biopharma industry towards drug discovery for CNS disorders, and how to address them.
09:05 – 10:05
Opportunities from emerging therapeutic modalities: Target selection in an evolving druggability landscape
- Emerging therapeutic modalities especially for targeting traditionally undruggable or
poorly druggable targets are opening new therapeutic avenues. - Beyond conventional small molecule and biologics, these approaches include
ASO/siRNA, small molecule RNA therapeutics, protein degradation approaches, gene
therapy and blood brain barrier delivery approaches. - Judicious pairing of target or target combinations with appropriate therapeutic
modalities has the potential to propel the next phase of innovation in CNS therapeutics.
Global Head, Neuroscience Search & Evaluation, AbbVie
Murali Gopalakrishnan is responsible for Search & Evaluation-in Neuroscience at AbbVie. In prior roles, Murali held positions of increasing responsibility at Abbott / AbbVie leading Discovery research programs across multiple therapeutic areas including neuroscience, pain, nephrology and urology and delivering multiple clinical candidates to the R&D pipeline. At Abbott, Murali led the Global External Research group with responsibility for sourcing Discovery research-focused external innovation & establishing early-stage partnerships. He also had
responsibility for the growth and leadership of Abbott China R&;D Center in Shanghai, while leading the Renal Discovery Research. Murali obtained his undergraduate training in pharmacy, PhD in Pharmacology with post-doctoral training in molecular biology and also holds an M.B.A degree.
Clinical Studies topic
Multimodal, remote assessments for decentralized CNS clinical trials
- Lowering burden on patients, CROs and clinic sites to allow robust enrollment and low early termination rates
- The importance of capturing day to day fluctuations in cognition and mood
- Ensuring remote assessments have high reliability and adherence and are equivalent to in-clinic assessments
Co-founder & Chief Scientific Officer, Cumulus Neuroscience.
Brian is a computational neuroscientist who is an expert in brain-reading technology, using machine learning methods to decode brain states from EEG, fMRI, MEG. His academic career a faculty position at Queen’s University Belfast, senior researcher at Carnegie Mellon University’s Department of Machine learning and as post-doc at the Centre for Mind/Brain Sciences, University of Trento, Italy. He has a Ph.D. in Cognitive Science from Trinity College Dublin, is a member of the Alzheimer’s Association EEG PIA committee, and organised the MLINI series of workshops on machine learning with neuroimaging data at NeurIPS.
Networking Lunch
Spotlight Presentation
AI-enabled phenotypic drug discovery delivers first-in-class treatments for major neuropsychiatric disorders
PsychoGenics’ has developed AI-enabled phenotypic platforms that have led to the discovery of numerous putative treatments for neuropsychiatric disorders including ulotaront, a novel antipsychotic discovered in partnership with Sunovion. Ulotaront, is a non-Dopamine D2 treatment for schizophrenia and other neuropsychiatric disorders in Phase III. It has produced robust effects across a broad range of symptoms of schizophrenia, including negative symptoms, with a placebo-like safety profile similar. The presentation will describe SmartCube and eCube, high throughput, mouse behavioral and physiological platforms that offer unique
opportunities to reinvigorate the drug discovery process by identifying promising first-in-class compounds with novel mechanisms of action.
President, Drug Discovery and Development, PsychoGenics Inc
David is a neuroscientist and expert in drug discovery and development who most recently held the position as Chief Scientific Officer of Redpin Therapeutics. Previously, David served in multiple leadership roles at Eli Lilly and Company, including Vice President and Chief Scientific Officer in neuroscience research.
1-2-1 Meetings / Networking Break
1-2-1 Meetings / Networking Break
15:00 – 16:00
Emerging non-amyloid targets for Alzheimer’s disease
- What are the targets that you are most excited about? What kind of patient population do you think that type of therapy should target?
- What kind of targets will be most suitable for early patients vs mild to moderate patients vs advanced patients?
- Any targets that are not currently in development, but you think we should focus on as well?
Senior Vice President, Research & Development, Annovis Bio
Dr. Cheng Fang is an accomplished neuroscientist with two decades of experience in neurodegenerative diseases with broad scientific knowledge and hands-on experience. She has a successful track record of scientific publications and contributions. Dr. Fang has deep drug development knowledge with both pre-clinical and clinical development experience.
16:00 – 16:30
Spotlight Presentation
Gold among the rubble: where digital innovation is finally impacting CNS medicine
The wave of digital innovation has at once inspired and frustrated those passionate for a medicine revolution. Santosh Shanbhang (CFO) will explain that, if you know where to look, investments are finally paying off. Among a host of false starts in the last decade, there’s also enough data to prove a few specific areas where digital is living up to the hype. Santosh will discuss these with examples, and discuss why CNS therapeutic areas are poised to realize the biggest immediate growth.
Chief Financial Officer, Akili, Inc.
CFO responsible for Finance, Corporate and Business Development, and Company Operations Shanbhag is a senior financial executive with 20 years of experience leading financial operations for U.S. and international organizations and executing complex business programs for transformative healthcare companies. Prior to joining Akili, Shanbhag held senior finance leadership roles at Vertex Pharmaceuticals, most recently as Vice President and Head of International Finance and Accounting, where he helped build-out the international business and secure reimbursement for novel medicines in key international markets. Prior to Vertex, Shanbhag served in positions of increasing responsibility at Capgemini Consulting and Texas Instruments. He holds an M.S. in Management & Engineering from MIT and Sloan School of Management and an M.S. in Mechanical Engineering from the University of Massachusetts, Amherst.
16:45 – 17:45
Novel non-invasive targeted drug delivery to the CNS
- Novel non-invasive delivery systems that can target within the CNS.
- Targets within the CNS for different diseases — which targets for PD or ALS or AD, etc.
- Are the targets on cell surface or cell interior — if so, how to access.
- Is there a difference delivering RNA, small molecules, or proteins, related to targets.
Chairman & Chief Executive Officer. Founder Lauren Sciences
Susan Rosenbaum, J.D., is the Founder, Chairman & CEO of Lauren Sciences; 10 years successful biotech visionary, entrepreneurial leader, strategic executive, motivated manager w/experience and expertise. Licensed breakthrough V-Smart nanotechnology for BBB delivery from BGU, Israel; built Lauren Sciences LLC, multiple medical and scientific collaborations, 5-member MAB/SAB of KOLs, 12 foundation grant awards, innovative V-Smart platform applications, transformative V-Smart pipeline products in pre-clinical development for CNS; co-authored DD&D March 2018 cover feature, presented at 42 scientific conferences, w/posters at 8 scientific meetings, made Lauren Sciences 1st Place Winner Global Healthcare Innovation at BIO 2019. Founder (present): Lauren Sciences LLC; Maya Sciences LLC; Founder (past): investment banking firm (Rosenbaum & Co.), corporate finance law firm (Rosenbaum, P.C.); Private and public company BODs; Nat’l/internat’l author/speaker.
Preclinical
08:30 – 09:00
Opening Address & Keynote Address
Expanding the horizon of EEG applications to accelerate CNS therapeutics development
The process of drug discovery is both costly and time-intensive. Through this keynote, we will explore the high translational potential of electroencephalography (EEG), a technique that finds use in both clinical and preclinical settings. The relevance of this tool will be illustrated by examples from epilepsy research. We’ll discuss how the development of more advanced EEG technology and analysis methods is also anticipated to further enhance the usefulness of EEG in understanding brain function and developing new therapeutic strategies against neurodegenerative, neurodevelopmental or rare CNS disorders.
Head of Technology, SynapCell
Julien holds a PhD in Neuroscience from the University of Lyon, France. Julien joined SynapCell in 2017 after two post-docs in different structures in Toronto. Since then, he manages R&D Preclinical projects and develops new innovative solutions for sponsors. With over 12 years’ experience in the field, he is well-versed in the emerging technologies and industry trends.
Scientific Sales Manager, SynapCell
After a MSc in Neuroscience and in international business in science, Hugo now manages the USA and LATAM zone. His background allows him to understand the current needs of the biopharma industry towards drug discovery for CNS disorders, and how to address them.
09:05 – 10:05
Methods to increase the success rates of drugs entering clinical phases
- Challenges to translate complex pre-clinical CNS disease models into early clinical proof of principle actionable studies
- The tools and options that available to improve the odds of successful translation from concept to clinical confidence
Vice President, Head of Biology, Cerevel Therapeutics
Phil did his PhD and a brief post doc in signal transduction mechanisms in CNS at the University of Nottingham in the UK. In 1994 he joined the Duman/Nestler lab at Yale investigating the molecular regulation of CRF receptors and, exploring the role the receptor plays in withdrawal from drugs of abuse. He joined Pfizer in 1997 as a lab head where he took on increasingly senior roles leading several programs incuding CB1 agonists/antagonists, psychosis disease space. In 2004 he joined the CNS management team and subsequently headed several programs in neuroscience, neurology and external collaborations which led to therapeutic area operational oversight as well as Kendall Sqauare MA site responsibilities. In 2018 Pfizer announced that it was exiting Neuroscience. Phil subsequently joined Cerevel Therapeutics as Head of Biology in October of 2018.
11:20 – 12:20
Unravelling the translational hurdles in neurotherapeutics: Can EEG be instrumental in this endeavor
- Discussion on the role of preclinical efficacy experiments in the high failure rate in clinical trials in CNS disorders
- Can preclinical testing abide with precision medicines prerequisite
- Foreseeing limitations in preclinical efficacy testing
Head of Technology, SynapCell
Julien holds a PhD in Neuroscience from the University of Lyon, France. Julien joined SynapCell in 2017 after two post-docs in different structures in Toronto. Since then, he manages R&D Preclinical projects and develops new innovative solutions for sponsors. With over 12 years’ experience in the field, he is well-versed in the emerging technologies and industry trends.
Scientific Sales Manager, SynapCell
After a MSc in Neuroscience and in international business in science, Hugo now manages the USA and LATAM zone. His background allows him to understand the current needs of the biopharma industry towards drug discovery for CNS disorders, and how to address them.
Spotlight Presentation
AI-enabled phenotypic drug discovery delivers first-in-class treatments for major neuropsychiatric disorders
PsychoGenics’ has developed AI-enabled phenotypic platforms that have led to the discovery of numerous putative treatments for neuropsychiatric disorders including ulotaront, a novel antipsychotic discovered in partnership with Sunovion. Ulotaront, is a non-Dopamine D2 treatment for schizophrenia and other neuropsychiatric disorders in Phase III. It has produced robust effects across a broad range of symptoms of schizophrenia, including negative symptoms, with a placebo-like safety profile similar. The presentation will describe SmartCube and eCube, high throughput, mouse behavioral and physiological platforms that offer unique
opportunities to reinvigorate the drug discovery process by identifying promising first-in-class compounds with novel mechanisms of action.
President, Drug Discovery and Development, PsychoGenics Inc
David is a neuroscientist and expert in drug discovery and development who most recently held the position as Chief Scientific Officer of Redpin Therapeutics. Previously, David served in multiple leadership roles at Eli Lilly and Company, including Vice President and Chief Scientific Officer in neuroscience research.
- Not having the right in vitro assay, or relying heavily on a single work-horse assay can lead to:
- Low quality hit rate from HTS (low confirmation)
- Not tractable SAR
- Unable to improve SAR
- SAR moving in the wrong direction
- Selecting the wrong molecule into in vivo
- Low quality hit rate from HTS (low confirmation)
- Should we invest more resources for deeper mechanistic profiling (kinetics, signaling pathways, etc.)? Or should we move molecules into in vivo models ASAP?
Senior Director, Head of In Vitro Pharmacology-Neuroscience, Cerevel
Sokhom Pin is currently the head of in vitro pharmacology at Cerevel Therapeutics, where his team is engaged in exploring novel targets, assay development/screening, as well as mechanistic profiling of late-stage molecules. Prior to Cerevel, he built and lead a similar team at Alkermes, Inc.. Sokhom has over 20 years of drug discovery experience at large and mid-sized pharma’s, including DuPont, BMS, and Novartis. He has a strong passion is GPCR pharmacology and neuroscience drug discovery. He received his Ph.D. in Pharmaceutical Sciences (Pharmacology/Toxicology) from the University of Connecticut and his M.S. from the Johns Hopkins University.
Spotlight Presentation
Gold among the rubble: where digital innovation is finally impacting CNS medicine
The wave of digital innovation has at once inspired and frustrated those passionate about a medicine revolution. Santosh Shanbang (CFO) will explain that, if you know where to look, investments are finally paying off. Among a host of false starts in the last decade, there’s also enough data to prove a few specific areas where digital is living up to the hype. Santosh will discuss these with examples, and discuss why CNS therapeutic areas are poised to realize the biggest immediate growth.
Chief Financial Officer, Akili, Inc.
CFO responsible for Finance, Corporate and Business Development, and Company Operations Shanbhag is a senior financial executive with 20 years of experience leading financial operations for U.S. and international organizations and executing complex business programs for transformative healthcare companies. Prior to joining Akili, Shanbhag held senior finance leadership roles at Vertex Pharmaceuticals, most recently as Vice President and Head of International Finance and Accounting, where he helped build-out the international business and secure reimbursement for novel medicines in key international markets. Prior to Vertex, Shanbhag served in positions of increasing responsibility at Capgemini Consulting and Texas Instruments. He holds an M.S. in Management & Engineering from MIT and Sloan School of Management and an M.S. in Mechanical Engineering from the University of Massachusetts, Amherst.
Clinical Studies
Registration
08:30 – 09:00
Opening Address & Keynote Address
Expanding the horizon of EEG applications to accelerate CNS therapeutics development
The process of drug discovery is both costly and time-intensive. Through this keynote, we will explore the high translational potential of electroencephalography (EEG), a technique that finds use in both clinical and preclinical settings. The relevance of this tool will be illustrated by examples from epilepsy research. We’ll discuss how the development of more advanced EEG technology and analysis methods is also anticipated to further enhance the usefulness of EEG in understanding brain function and developing new therapeutic strategies against neurodegenerative, neurodevelopmental or rare CNS disorders.
Head of Technology, SynapCell
Julien holds a PhD in Neuroscience from the University of Lyon, France. Julien joined SynapCell in 2017 after two post-docs in different structures in Toronto. Since then, he manages R&D Preclinical projects and develops new innovative solutions for sponsors. With over 12 years’ experience in the field, he is well-versed in the emerging technologies and industry trends.
Scientific Sales Manager, SynapCell
After a MSc in Neuroscience and in international business in science, Hugo now manages the USA and LATAM zone. His background allows him to understand the current needs of the biopharma industry towards drug discovery for CNS disorders, and how to address them.
Fluid biomarkers in pharmaceutical development – the good, bad and ugly
- Changing role of biomarkers – what makes a great biomarker, what’s working, gaps and expectations for the future
- Current experiences with fluid biomarkers in pharmaceutical development – successes and areas for improvement
- Bridging the gap between early-stage biomarker applications and routine/ large-scale use – key challenges & learnings
VP Sales & Marketing, Fujirebio Diagnostics
Matt Bell is the Vice President of Marketing & Sales at Fujirebio Diagnostics, where he is focused on the development and execution of the neuroscience roadmap, clinical development & commercialization of novel neurodegenerative biomarkers to support pharmaceutical and diagnostic clients, as well as the transition of novel, bespoke biomarkers to their development as IVD cleared products that can be deployed across countries. Prior to Fujirebio, he worked in leadership positions at LGC genomics and Thermo Fisher Scientific where he was responsible for the R&D roadmap, validation and commercialization of new products. Matt has worked extensively along the interface between R&D, manufacturing and commercialization.
Morning Refreshments
Integrating multimodal data to meet clinical challenges
- Clinical potential of multimodal assessments
- Validation and operational challenges
- Implications for clinical research and drug development
Head of Partnerships, Winterlight Labs (part of Cambridge Cognition)
Bill Simpson serves as Head of Partnerships at Winterlight Labs. He has spent the last 15 years working in digital health as a data analyst, clinical scientist and business development lead. He obtained his PhD in Neuroscience from McMaster University where he studied blood and behaviour based biomarkers of psychiatric disease.
Spotlight Presentation
AI-enabled phenotypic drug discovery delivers first-in-class treatments for major neuropsychiatric disorders
PsychoGenics’ has developed AI-enabled phenotypic platforms that have led to the discovery of numerous putative treatments for neuropsychiatric disorders including ulotaront, a novel antipsychotic discovered in partnership with Sunovion. Ulotaront, is a non-Dopamine D2 treatment for schizophrenia and other neuropsychiatric disorders in Phase III. It has produced robust effects across a broad range of symptoms of schizophrenia, including negative symptoms, with a placebo-like safety profile similar. The presentation will describe SmartCube and eCube, high throughput, mouse behavioral and physiological platforms that offer unique
opportunities to reinvigorate the drug discovery process by identifying promising first-in-class compounds with novel mechanisms of action.
President, Drug Discovery and Development, PsychoGenics Inc
David is a neuroscientist and expert in drug discovery and development who most recently held the position as Chief Scientific Officer of Redpin Therapeutics. Previously, David served in multiple leadership roles at Eli Lilly and Company, including Vice President and Chief Scientific Officer in neuroscience research.
15:00 – 16:00
Clinical trials for rare diseases: challenges and opportunities
- Best practice in ensuring your rare disease trial is a success
- Using precision medicine in clinical trials to advance therapeutics for rare diseases
- Assessing clinical data with biomarker-correlated outcomes in Rett Syndrome
Vice President of BD and Scientific Strategy, Anavex
Dr Daniel Klamer, Vice President of Business Development and Scientific Strategy of Anavex, has more than 15 years of experience in neuroscience and the orphan disease space, with acquisition, partnering and R&D experience in Europe and the USA. Prior to Anavex he worked at Retrophin and Neurosearch Sweden. At Neurosearch Sweden, Dr Klamer led and evaluated multiple discovery-phase neuropharmacological research products with an emphasis on strategic evaluation of preclinical and clinical development. Dr. Klamer earned his PhD in Pharmacology at The Sahlgrenska Academy at University of Gothenburg, Sweden, his MBA at Fordham Gabelli School of Business, and his Post-Doctoral training at the Department of Psychiatry, Yale University School of Medicine. In addition, Dr. Klamer holds a position as an Associate Professor at the Department of Pharmacology at The Sahlgrenska Academy at the University of Gothenburg.
Chief Financial Officer, Akili, Inc.
CFO responsible for Finance, Corporate and Business Development, and Company Operations, Shanbhag is a senior financial executive with 20 years of experience leading financial operations for U.S. and international organizations and executing complex business programs for transformative healthcare companies. Prior to joining Akili, Shanbhag held senior finance leadership roles at Vertex Pharmaceuticals, most recently as Vice President and Head of International Finance and Accounting, where he helped build-out the international business and secure reimbursement for novel medicines in key international markets. Prior to Vertex, Shanbhag served in positions of increasing responsibility at Capgemini Consulting and Texas Instruments. He holds an M.S. in Management & Engineering from MIT and Sloan School of Management and an M.S. in Mechanical Engineering from the University of Massachusetts, Amherst.
16:45 – 17:45
Utilizing clinical biomarkers to enhance drug development in neurodegenerative diseases
- Defining the path for Blood Based Biomarkers (BBBMs) in Clinical Trial and Real World Clinical Setting
- Addressing patient heterogeneity (ethnoracial diversity, age, gender, co-morbidities, co-pathologies)
- Developing diagnostic biomarkers for differential diagnosis (e.g. PD, DLB, FTD, etc.)
Executive Director, Translational Medicine, Eisai US
Pallavi Sachdev is Executive Director and Head of Translational Medicine in the Alzheimer’s Disease and Brain Health (ADBH) Division at Eisai Inc. Pallavi received her Ph.D. in Biochemistry and Molecular Biology from Mount Sinai-New York University School of Medicine and conducted her postdoctoral fellowship at The Rockefeller University focusing on cell signaling networks. Pallavi is recognized as a subject matter expert on implementation of clinical biomarkers for the development of tailored therapeutics. In recognition of her noteworthy achievements, Pallavi was the recipient of the 2021 Rising Star Award from the Healthcare Businesswoman’s Association (HBA). Her research interests include biofluidic biomarkers and their application in neuroscience with a focus on Alzheimer’s disease as well as optimal use of these fluid-based biomarkers with other modalities including imaging, genetic and digital. Withing her group, Pallavi has expanded the use of global proteomics to deliver target engagement and treatment response biomarkers for the diverse neurology pipeline. She is a champion of pre-competitive collaboration and is an active member of several public/private partnerships and consortia including DIAN-TU, ADNI PPSB, FNIH AMP-AD and the CORAL consortium.
Drug Delivery
08:30 – 09:00
Opening Address & Keynote Address
Expanding the horizon of EEG applications to accelerate CNS therapeutics development
The process of drug discovery is both costly and time-intensive. Through this keynote, we will explore the high translational potential of electroencephalography (EEG), a technique that finds use in both clinical and preclinical settings. The relevance of this tool will be illustrated by examples from epilepsy research. We’ll discuss how the development of more advanced EEG technology and analysis methods is also anticipated to further enhance the usefulness of EEG in understanding brain function and developing new therapeutic strategies against neurodegenerative, neurodevelopmental or rare CNS disorders.
Head of Technology, SynapCell
Julien holds a PhD in Neuroscience from the University of Lyon, France. Julien joined SynapCell in 2017 after two post-docs in different structures in Toronto. Since then, he manages R&D Preclinical projects and develops new innovative solutions for sponsors. With over 12 years’ experience in the field, he is well-versed in the emerging technologies and industry trends.
Scientific Sales Manager, SynapCell
After a MSc in Neuroscience and in international business in science, Hugo now manages the USA and LATAM zone. His background allows him to understand the current needs of the biopharma industry towards drug discovery for CNS disorders, and how to address them.
Non-invasive methods to increase brain drug uptake
- What in vitro and in vivo preclinical tools are useful to understand the blood brain barrier?
- What can be achieved by focused ultrasound?
- Novel synthetic AAVs and delivery to the CNS
- Will non-viral delivery be an alternative?
Senior Director, External Neuroscience Innovation Neuroscience Drug Discovery Unit, Takeda
Gabriele Proetzel, PhD, Senior Director, External Neuroscience Innovation in the Neuroscience Drug Discovery Unit at Takeda Pharmaceuticals, based in Cambridge, MA, focuses on gene therapy therapeutic approaches for neurological disorders.
Prior appointments were at The Jackson Laboratory, Scil Proteins, Deltagen Inc., and Boehringer Mannheim/Roche. Gabriele earned her Master’s degree in Life Sciences from the Ludwig-Maximilian University of Würzburg, Germany, and a Ph.D. from University of Cincinnati, Ohio studying homologous recombination in embryonic stem cells to develop mouse models for disease. She completed her postdoctoral at the Max Planck Institute for Biophysical Chemistry in Göttingen, Germany.
Preclinical topic
Early stage GLP safety assessment can be a deal breaker
- CNS drugs experience high attrition rates and safety concerns compared other therapeutic areas. Lack of translatability between pre-clinical experimentation and clinical studies and ADR’s are significant contributing factors. Early de-risking of any potential safety concerns is imperative.
- The ICH S7 Core battery is a pre-requisite for FIH. ICH S7A and M3(R2) guidelines only mandate a modified Irwin/FOB assessment for CNS safety but can a single dose neurofunctional test adequately address the complexity of the nervous system and the propensity of CNS drugs to cause nervous system AE’s.
- De-risking for potential CNS adverse effects in early drug development through in vivo supplementary assays (seizure liability, cognitive deficits, motor deficits and abuse liability).
- Incorporation of in vitro assessment of primary neurons, hiPSC, organoids and tissue slices into an early-stage comprehensive CNS safety assessment to improve de-risking.
Director of Business Development- North America, Porsolt S.A.S.
Kendall has more than a decade of experience as an in vivo behavioural pharmacologist in both industry and academia. Before taking a business development role at Porsolt, she held a study director role at Psychogenics managing their in vivo pharmacology team focused on neurodegenerative disorders. She holds a PhD in Veterinary Biology and Biomedical Science from Murdoch University in Australia and has completed post-doctoral research fellowships in the laboratories of Dr Robert H Brown Jr at Harvard Medical School/MGH and Dr Giuseppina Tesco at Tufts Medical School before transitioning into industry.
Spotlight Presentation
AI-enabled phenotypic drug discovery delivers first-in-class treatments for major neuropsychiatric disorders
PsychoGenics’ has developed AI-enabled phenotypic platforms that have led to the discovery of numerous putative treatments for neuropsychiatric disorders including ulotaront, a novel antipsychotic discovered in partnership with Sunovion. Ulotaront, is a non-Dopamine D2 treatment for schizophrenia and other neuropsychiatric disorders in Phase III. It has produced robust effects across a broad range of symptoms of schizophrenia, including negative symptoms, with a placebo-like safety profile similar. The presentation will describe SmartCube and eCube, high throughput, mouse behavioral and physiological platforms that offer unique
opportunities to reinvigorate the drug discovery process by identifying promising first-in-class compounds with novel mechanisms of action.
President, Drug Discovery and Development, PsychoGenics Inc
David is a neuroscientist and expert in drug discovery and development who most recently held the position as Chief Scientific Officer of Redpin Therapeutics. Previously, David served in multiple leadership roles at Eli Lilly and Company, including Vice President and Chief Scientific Officer in neuroscience research.
1-2-1 Meetings / Networking Break
1-2-1 Meetings / Networking Break
Transcytosis-mediated brain delivery for proteins, antibodies, oligonucleotides, and small molecules
- Challenges in crossing and transporting proteins, antibodies, oligonucleotides and small molecules across the blood brain barrier (BBB).
- BBB transcellular transport via receptor-mediated transcytosis targeting the transferrin, insulin, low density lipoprotein and other receptors.
- Nanoparticles and their surface modifications used for brain delivery including liposomes, polymer nanospheres, dendrimers, protein drug conjugates and other biological nanoparticles.
- Other transport systems for crossing the BBB and delivering proteins, antibodies, oligonucleotides and small molecules into the brain including virus like particles (VLPs) and targeting endothelial cells.
President & Chief Executive Officer, Aphios Corporation
Trevor P. Castor, President and CEO, Aphios Corporation, Woburn, MA, has over 30 years of diversified experience in technology and business development, management, marketing and finance in the biotechnology industry. He graduated from the University of California, Berkeley with a Ph.D. in Mechanical Engineering and a MSc in Chemical Engineering, and summa cum laude with a BSc in Chemical Engineering from the University of Toronto, Canada. He is the author of several peer-reviewed publications, and over 100 issued and pending patents. He has collaborated with several pharmaceutical and biotechnology companies including Eli Lilly, BMS, Novartis, Pfizer and Merck-Millipore-Sigma.
Trevor P. Castor, President and CEO, Aphios Corporation, Woburn, MA, has over 30 years of diversified experience in technology and business development, management, marketing and finance in the biotechnology industry. He graduated from the University of California, Berkeley
with a Ph.D. in Mechanical Engineering and a MSc in Chemical Engineering, and summa cum laude with a BSc in Chemical Engineering from the University of Toronto, Canada. He is the author of several peer-reviewed publications, and over 100 issued and pending patents. He has collaborated with several pharmaceutical and biotechnology companies including Eli Lilly, BMS,
Novartis, Pfizer and Merck-Millipore-Sigma.
Spotlight Presentation
Gold among the rubble: where digital innovation is finally impacting CNS medicine
The wave of digital innovation has at once inspired and frustrated those passionate for a medicine revolution. Santosh Shanbhang (CFO) will explain that, if you know where to look, investments are finally paying off. Among a host of false starts in the last decade, there’s also enough data to prove a few specific areas where digital is living up to the hype. Santosh will discuss these with examples, and discuss why CNS therapeutic areas are poised to realize the biggest immediate growth.
Chief Financial Officer, Akili, Inc.
CFO responsible for Finance, Corporate and Business Development, and Company Operations Shanbhag is a senior financial executive with 20 years of experience leading financial operations for U.S. and international organizations and executing complex business programs for transformative healthcare companies. Prior to joining Akili, Shanbhag held senior finance leadership roles at Vertex Pharmaceuticals, most recently as Vice President and Head of International Finance and Accounting, where he helped build-out the international business and secure reimbursement for novel medicines in key international markets. Prior to Vertex, Shanbhag served in positions of increasing responsibility at Capgemini Consulting and Texas Instruments. He holds an M.S. in Management & Engineering from MIT and Sloan School of Management and an M.S. in Mechanical Engineering from the University of Massachusetts, Amherst.
Novel strategies of drug delivery into the brain
- Current Challenges: Discuss the current challenges and obstacles that exist in developing genomic therapies, including technological limitations, ethical considerations, and funding constraints.
- Novel Technologies: Discuss the latest advancements in genomic technologies that are driving the development of new therapies, such as
CRISPR/Cas9, base editing, tRNAs and gene therapy vectors. - Delivery Methods: The delivery of genomic therapies to target cells and tissues is a critical factor in their success.
- Preclinical Testing: Discussing current strategies for preclinical testing of genomic therapies, including in vitro and in vivo models, and identifying areas where additional research is needed to improve the
predictive value of preclinical testing. - Clinical Trials: Discussing the design of clinical trials for genomic therapies, including endpoints and patient selection criteria, and identifying areas where improvements can be made to streamline the clinical trial process and accelerate the development of new therapies.
Vice President of R&D, Tevard Biociences
Peixin Zhu joins Tevard Biosciences as the VP of R&D. He has about two decades of drug discovery and gene therapy development, experience ranging from developing A.I. HTS platforms for small molecules to advancing SMA Type I gene therapy through IND. He has a strong background across multiple scientific fields, including computational neuroscience, molecular biology, and gene therapies for CNS and liver indications including SMA, Dravet, Batten, DMD, Fabry, and Pompe diseases etc. Prior to Tevard, Peixin was the VP of Discovery Research of LogicBio. Prior to joining LogicBio, Peixin Zhu led multiple teams at the Novartis Institutes for Biomedical Research; Applied Genetic Technologies Corp.; and Exegenesis Bio Inc. Peixin was responsible for establishing an in vivo A.I. HTS screening platform at Novartis, and for designing and bringing the 2nd generation SMA Type I gene therapy to early clinical stage at Exegenesis Bio. He and his team were also instrumental in several key partnerships including with the Stanley Center (Broad Institute), Florida State University.