7:55 am
Networking & Registration

Investigating Targeted Therapeutic Approaches Against Alzheimer’s

8:55 am Chair’s Opening Remarks

9:00 am Targeting the NLRP3 Inflammasome for the Treatment of Alzheimer’s

Synopsis

  • Learning how persistent NLRP3 inflammasome signaling propagates neuroinflammation in aged brains, and promotes AD pathogenesis and progression
  • Hearing how recent data confirms the essential role of NEK7 in the assembly of NLRP3 complex
  • Discovering how Halia is the first to target NEK7 to block the formation of NLRP3 inflammasome complex

9:30 am Update on Animal Models of Alpha-Synucleinopathy for Use in Therapeutic Drug Development

Synopsis

  • Investigating neuroinflammation with a focus on alpha-synuclein specific T cell responses
  • Exploring preformed fibrils such as motor and non-motor models
  • Deep Divining into AAV-alpha synuclein in rodent and non-human primate models and exploring motor and non-motor endpoints

10:00 am Precision Medicine Driven by the SPECTRA Network Biology Platform in Neurodegenerative Disorders

  • Johanna Withers Vice President, Clinical Development and Operations, Scipher Medicine

Synopsis

  • Learning about SPECTRA: A propriety integrative multi-omics network biology platform bringing first-in-class precision medicines to patients by identifying novel targets, drug repurposing candidates, drug response predictions, drug downstream molecular effects, biomarker discovery and patient stratification.
  • Through SPECTRA analyses, we help inform treatment selection to guide patients to therapies most likely be effective for them, with the overall goal of improving drug responses early in the disease timeline, preventing the progression of disease.
  • Scipher’s therapeutics pipeline has demonstrated the ability to identify promising drug targets as evidenced by successful experimental preclinical validation in disease models, an approach now being applied to neurodegenerative diseases.

10:30 am
Morning Break & Networking

Discovery & Translational Track

Zoning in on Neurodegenerative Disease Targets

11:00 am Differentiated iPSC Co-Culture Model Development for Neurodegenerative Diseases

Synopsis

  • Presenting data to show the development of novel coculture of peripheral motor neuron assays
  • Describing improvements to culture conditions that enable rapid maturation of motor neurons
  • Showcasing functional data from human iPSC-derived cortical neuron assays

11:30 am Uncovering Microbiome-Derived Therapeutic Targets to Treat Alzheimer’s Disease

  • George Tetz Chief Executive Officer, CLS Therapeutics & The Human Microbiology Institute

Synopsis

  • Investigating Alzheimer’s disease in the context of the microbiome: what do biopharma need to know?
  • Analyzing how microbial DNAs involvement in Alzheimer’s disease can be exploited as a treatment strategy against it
  •  Revealing how DNA of some species triggers protein misfolding and learning how this influences Alzheimer’s development and progression

Late Translational & Early Clinical Track

Upgrading Your Therapeutic Approach Against Alzheimer’s & Parkinson’s

  • Chan Tan Principal Scientist, BridgeBio Pharma

11:00 am Discovering Gene therapy for APOE4-Associated Alzheimer’s Disease

  • Jay Barth Executive Vice President & Chief, Medical Officer, Lexeo Therapeutics

Synopsis

  • Reviewing the role of APOE in the pathogenesis of Alzheimer’s Disease
  • Understanding the approach of AAV-based gene therapy to treat Alzheimer’s Disease patients who are APOE homozygous
  • Describing the Phase 1 study design of LX1001

11:30 am Enhancing Lysosomal Function as a Therapeutic Strategy for Parkinson’s Disease

  • Magdalene Moran President & Chief Scientific Officer, Carraway Therapeutics

Synopsis

  • Investigating lysosomal dysfunction in neurodegenerative diseases and what this means from a drug development perspective
  • Exploring activating TRPML1 as potential treatment for GBA-PD
  • Looking through the crystal ball: Outlining future Caraway directions

Exploring Biomarkers for Neurodegenerative Disease From a Clinical Standpoint

12:00 pm Panel Discussion: Integrating Biomarkers Throughout the Trajectory of CNS Drug Discovery & Development to Guide Clinical Decisions

  • Alejandro Rangel Associate Professor of Medicine, Universidad Panamericana
  • Chris Winrow Vice President & Head of Translational Medicine, Cyclerion
  • Magdalene Moran President & Chief Scientific Officer, Carraway Therapeutics

Synopsis

  • Discussing & debating whether biopharma should strive to achieve disease modification or improvement in quality of life when developing therapies for Alzheimer’s
  • Exploring the impact of Aducanumab on international approval for neurodegenerative disease
  • Learning what steps you must take to tailor your drug development approach to meet regulatory standards

12:30 pm
Lunch Break & Networking

Deep Diving into Therapy Delivery Across the Blood Brain Barrier to Drive Therapeutic Success

2:00 pm Analyzing Translational Approaches for Brain Targeted Drug Delivery

  • Lisa Shafer Chief Scientific Officer, Cerebral Therapeutics

Synopsis

  • Exploring challenges, success’ and failures in drug distribution for neurodegenerative disease
  • Understanding what steps you must take to enable personalized dosing and designing a biomarker strategy which works across neurodegenerative disease indications
  • Hearing common mistakes in effective clinical translation of therapeutic delivery across the blood brain barrier and learning how to overcome them

2:30 pm Showcasing LRRK2 Inhibitors as Treatment for Parkinson’s Disease

Synopsis

  • Exploring the role of LRRK2 in the pathophysiology of Parkinson’s disease
  • Uncovering case studies which showcase the benefit of therapeutic approaches to inhibit LRRK2
  • Revealing how new small molecule inhibitors of LRRK2 can be used to treat Parkinson’s disease

3:00 pm Panel Discussion: The New Landscape in NDD investing: How the 2021 Events Have Changed Outlooks

Synopsis

  • Hearing how new mechanisms and modalities have opened doors for early stage investment
  • Exploring new regulatory approvals, and their effect on the funding environment for clinical stage opportunities
  • Public market reception for neuro focused companies, and the effect on M&A

3:30 pm
Afternoon Break & Networking

Discovering Emerging Therapeutic Approaches & Treatment Strategies for CNS Diseases

4:00 pm Exploring CNS Application of Protein Degraders to Determine Whether They Can be Utilized in a Selective Manner to Minimize Side Effects & Maximize Therapeutic Outcome

  • Beth Hoffman Founder, President & Chief Executive Officer, Origami Therapeutics

Synopsis

  • Deep diving into targeted protein degraders (TPDs) from neurodegenerative therapeutic perspective
  • Exploring the use of TPDs to minimize side effects and maximize therapeutic outcome
  • Deep diving into the future of TPDs in CNS disorders to determine their impact on the CNS therapeutic landscape

4:30 pm Metabolic Dysfunction in Neurodegenerative Disorders: Spotlight on Parkinson’s, Huntington’s & Alzheimer’s

Synopsis

  • Understanding the impact of brain energy metabolism on health and disease
  • Exploring the advantages and disadvantages of metabolic interventions in CNS diseases
  • Discovering the power of ketogenic therapies can be harnessed against Huntington’s, Alzheimer’s & Parkinson’s

5:00 pm Uncovering Targeted Neural Cell Therapies for Chronic Disorders of the Nervous System

Synopsis

  • Developing neuronal cell therapy targeting dysregulated neural activity as a broad opportunity for the potential treatment of multiple neurodegenerative and developmental disorders
  • Revealing NRTX-1001: an inhibitory neuron cell therapy for chronic focal epilepsy (Phase I/II; NCT05135091)
  • Examining the preclinical evaluation of NRTX-1001 for Alzheimer’s disease

5:30 pm RNA interference (RNAi) Approaches for Allele Specific, Gain of Function Mutations in Rare, Monogenic Neurodevelopmental Disorders

Synopsis

  • Advances in DNA sequencing has rapidly accelerated the identification and characterization mutations at the heart of rare neurodevelopmental and neurodegenerative disorders. More than 7,000 rare, monogenic disorders are known, of which 40% are neurological in nature.
  • Knowing the root causes of monogenic disorders has allowed mutationspecific therapies possible. To that end, use of RNAi as a gene silencing therapy is an attractive approach for disorders where gain-of function mutations are the underlying etiology such as the case with KIF1Aassociated Neurological Disorder (KAND). Technologies that can address
    allele-specific mutations will be critical for therapeutic development.
  • Ovid Therapeutics is focused in areas of significant unmet medical need and where opportunities to leverage academic, patient organizations and patient data are available to accelerate discovery and development of novel and innovative therapies.

6:00 pm End of Day Two