*All times listed are in EST

Targeting Adult Neurodegenerative Disorders

7:40 am Chair’s Opening Remarks

7:45 am Session Reserved for PTC Therapeutics

8:15 am Gene Therapy for Parkinson’s Disease: Bridge to the Future


  • Past approaches for Parkinson’s Disease gene therapy: dopamine enhancement, neuromodulation, and neuroprotection
  • An ongoing journey: what we have learned from prior studies and how changes are being implemented in current Parkinson’s Disease gene therapy studies
  • Role of gene therapy as standard of care for patients with Parkinson’s Disease: a vision for how to achieve approvals and beyond

8:45 am Adeno-associated Virus Gene Therapy for Hereditary Neurologic Disorders

  • Ronald Crystal Founder & Chief Scientific Advisor (President & Chairman, Department of Genetic Medicine), Lexeo Therapeutics (Weill Cornell Medical College)


  • Exploring the vectors used to target hereditary neurologic disorders
  • Assessing the delivery methods and challenges for AAV; where does my vector go?
  • Evaluating gene therapy for CLN2 disease and APOE4 Alzheimer’s disease

9:15 am Clinical Trial Update: AAV2-BDNF Gene Therapy in Alzheimer’s Disease

  • Mark Tuszynski Professor & Director, Translational, Neuroscience Institute, UC San Diego School of Medicine


  • Showcasing how BDNF prevents neuronal death, stimulates neuronal function, and builds new synapses in animal models of Alzheimer’s disease
  • Highlighting the AAV2-BDNF gene therapy as the only potential therapy for Alzheimer’s disease that might improve memory by building new synapses
  • Evaluating the administration of AAV2-BDNF under MRI guidance to humans

9:45 am Panel Discussion: Targeting Adult Patient Populations with Neurodegenerative Disorders

  • Amber Van Laar Vice President, Clinical Development , AskBio
  • Ronald Crystal Founder & Chief Scientific Advisor (President & Chairman, Department of Genetic Medicine), Lexeo Therapeutics (Weill Cornell Medical College)
  • Mark Tuszynski Professor & Director, Translational, Neuroscience Institute, UC San Diego School of Medicine

10:15 am Structured Networking

11:00 am Morning Break

Next Generation Vectors For Improved Cell-Specificity & CNS Tropism


Moderator: Melissa Kotterman, Chief Scientific Officer, Iris Medicine

11:15 am Evaluating AAV Delivery for Neuroscience Gene Therapy

  • Junghae Suh Vice President, Gene Therapy Accelerator Unit , Biogen


  • Gene therapy for neuroscience continues to be challenging
  • Discussing AAV capsid considerations for neurological disorders
  • Discovering AAV capsid innovations for improved CNS specificity and tropism

11:45 am Engineered AAVs with Targeted & efficient Noninvasive Gene Delivery to the Primate CNS

  • Nick Goeden Co-Founder, Vice President of Technology , Capsida


  • High-throughput screening of AAV capsid libraries directly in non-human primates yields multiple generations of improved AAVs for delivery to the CNS
    Combinatorial engineering of multiple surface-exposed loops confers organ and cell-type level tropism and transduction
  • The resulting engineered capsids show robust and uniform DNA, RNA, and protein expression throughout the primate CNS, and dramatically increased transduction and transcription in human neurons
  • The engineered capsids are highly scalable on our suspension culture GMP-manufacturing platform, with high productivity and product quality

12:15 pm Machine Guided AAV Capsid Engineering for Improved CNS Delivery

  • Eric Kelsic Founder & Chief Executive Officer, Dyno Therapeutics


  • Machine-guided capsid engineering represents a new approach to overcome the current challenges of in vivo gene delivery
  • This approach combines three advanced technologies:
    • Next-gen library synthesis
    • Next-gen sequencing
    • Machine learning
  • With this workflow the search for improved capsids can be dramatically accelerated

12:45 pm Panel Discussion: Vector Discovery for Improved CNS Delivery

Optimizing Translational Strategies of Doses


Moderator: Swati Tole, Chief Medical Officer, Capsida

11:15 am Considerations for Translational Dose Selection for Gene Therapies

  • Sergey Aksenov Senior, Preclinical & Early Development Pharmacometrics, Novartis Institutes for BioMedical Research (NIBR)


  • Reviewing pharmacological concepts for gene therapy and outline a preclinical dose selection research program for gene therapy programs (i.e what needs to be measured in what species NHP vs other species, what are objectives of NHP biodistribution studies)
  • Describing a mathematical model approach to analyzing data from biodistribution studies, with the goal of informing first-in-human gene therapy dose range
  • Illustrating the modeling approach with NHP data

11:45 am How Regulatory-ready Biosimulation Advances Pre-clinical and Clinical CNS Drug Development


  • Predict dose-response relationship between administered viral load and target protein expression
  • For Gene Therapy and Neurological Diseases, these tools increase confidence around dosing, immunogenicity, and special populations
  • QSP modeling using virtual patients/trials supported by global regulators

12:00 pm Clinical Trial Strategies in the Rare Neurodevelopmental Disorders: Challenges & Opportunities


  • Summarizing latest thoughts on clinical trial approaches in RNDDs (including Angelman Syndrome, Dup15q Sydrome, Dravet syndrome, Rett Syndrome, Tuberous Sclerosis Complex, CDKL5-deficiency disorder and Fragile X Syndrome)
  • Sharing insights from both industry and academic perspectives on the future of translational medicine for the next generation of therapies in this field
  • Considering not only broader clinical trial design elements relevant to these populations but also of emerging commonalities and opportunities specific to RNDDs more broadly

12:30 pm Panel Discussion: Dose Optimization for Neurological Gene Therapies

1:15 pm Lunch Break

Enhancing Preclinical Model Value

2:15 pm Use of Computational and In Vitro Modelling to Predict CSF Drug Biodistribution

  • Bryn Martin Vice President, Research, Precision Delivery, & CSF Sciences, Alcyone Therapeutics


  • Problems associated with pre-clinical study power, reliability, and translation to humans
  • Need for precision dosing of drugs to the brain: where we are now and where we need to go
  • Key elements of CSF anatomy and physiology for drug modeling
  • In vitro modeling systems and results
  • Computational modeling systems and results

2:45 pm Optimizing Preclinical Models to Determine Gene Therapy Safety & Efficacy

  • Monica Bennett Head of Gene Therapy Unit, Neuroscience, Novartis Institutes for BioMedical Research (NIBR)


  • Preclinical development of AAV gene therapies for neurological disorders
  • Considerations for the development of AAV gene therapies
  • Preclinical development of AAV gene therapies

3:15 pm Session Reserved for Gubra

3:25 pm Efficacy of Gene Therapy Using a Dual Route of Administration in a Large Animal Model of Batten Disease

  • Jennifer Daily Executive Director, Preclinical Development, Neurogene


  • A large animal model of disease is more clinically translational due to the brain structure and size and CSF flow
  • Selecting the right delivery route is critical for efficacy in alarge animal model
  • A dual route of administration was essential for targeting the major phenotypes characteristic of CLN5 Batten disease

3:55 pm Panel Discussion: Optimizing Preclinical Predictions for CNS Gene Therapies

  • Bryn Martin Vice President, Research, Precision Delivery, & CSF Sciences, Alcyone Therapeutics
  • Monica Bennett Head of Gene Therapy Unit, Neuroscience, Novartis Institutes for BioMedical Research (NIBR)
  • Jennifer Daily Executive Director, Preclinical Development, Neurogene

Approaching the Clinic: Trial Design

2:15 pm The Multiple Ethical Challenges of Designing Pivotal Clinical Trials for Pediatric Rare Neurological Disorders


  • Limitations of randomized controlled trials
  • Limitations of single-arm trials
  • The potential of biomarkers and composite endpoints in rare

2:45 pm International, Multi-Center, Gene Therapy Trials in Rare Neurological Diseases: Opportunities & Challenges


  • Complex study designs pose operational challenges
  • Regulatory scrutiny of gene therapy trials introduces operational burden
  • Rare conditions and young populations require creative solutions and attention to patient experience

3:15 pm Session Reserved

3:45 pm Panel Discussion: Clinical Challenges for Neurological Gene Therapies

4:25 pm Afternoon Break & Networking

Gene Therapy For Neurological Disorders: Toxicity & Safety

5:00 pm Addressing Disease Progression in CNS: Moving from Rare to Common Disorders


  • How are development paths for more common disease best managed?
  • Key areas of challenge with conducting trials
  • Are endpoints for gene therapies the same as endpoints for small molecules?

5:30 pm Showcasing the Latest CNS Toxicity Knowledge After Direct Parenchymal Administration


  • Assessing the delivery procedure: risks and improvements
  • Exploring local toxicity and AAV capsids, and transgene expression
  • MRI follow-up and evaluation of local tolerance: experience from large animals studies and patients
  • Evaluating the immune reaction and redosing strategies
  • Next steps: disease selection and protocol design

6:00 pm CNS Immunogenicity & Toxicity Panel


  • Discussing the latest CNS immunogenicity data
  • Debating DRG toxicity
  • Translation of toxicity research into the clinic

6:30 pm Biotech Quick-Fire Round


As the gene therapy landscape expands and more companies target the CNS, this session will showcase the latest companies to join the scene with a series of short presentations from the conference attendees.

7:00 pm Chair’s Closing Remarks

7:05 pm Scientific Poster Session


Once the presentations finish, join your colleagues to share your latest neurological research and view your fellow attendees gene therapy progress, to forge new connections and catch up with familiar faces over a drink.

8:00 pm End of Day