AI in Medicine: Transforming the Landscape of Tissue-Based Diagnostics
Keynote Speaker: Behzad Najafinam, MD
University of Washington, Seattle, WA, USA
Day 1
1. Nanotechnology.
08:00
Arrival/Breakfast
08:00
09:00 - 09:20
Opening Remarks
09:20 – 10:00
Keynote Lecture
09:20 – 10:00
10:00 - 10:20
Autophagy and lysosomal dysfunction as emerging mechanisms of nanomaterial toxicity.
Stephan Stern
10:20 - 10:40
Blood-brain barrier delivery for lysosomal storage disorders with IgG-lysosomal enzyme fusion proteins.
Ruben Boado
10:20 - 10:40
10:40 – 11:00
Coffee Break
11:00 - 11:20
Nanoliposome-Based Enzyme Replacement Therapy for Fabry Disease: Progress Toward Clinical Translation
Elisabet González-Mira
11:00 - 11:20
11:20 - 11:40
A precision, tissue-targeted siRNA conjugate: the first substrate reduction therapy in Pompe disease.
Susan Sparks
11:40 - 12:00
Q&A
11:40 - 12:00
2. Organoids and Lab-Grown Models.
12:00 - 12:20
Modeling neuronopathic storage disorders with patient-derived culture systems.
Joe Mazzullii
12:00 - 12:20
12:20 – 12:40
Synthetic human heart models for the study of cardiac development and disease.
Aitor Aguirre
12:40 – 13:00
Generation of a brain organoid platform to study neuronopathic Gaucher disease and therapeutic strategies.
Ying Sun
12:40 – 13:00
13:00 - 13:20
Q&A
13:20 - 14:30
Lunch and Selected Presentations of posters
13:20 - 14:30
3. The Expanded Applications of AI in Lysosomal Disorders.
14:30 - 14:50
Virtual Basophils.
Oral Alpan
14:30 - 14:50
14:50 - 15:10
An AI-based approach to identify lysosomal cystine export regulating mTORC signaling
Svenja Keller
15:10 - 15:30
Artificial Intelligence in Clinical Trials: Opportunities and Challenges in Lysosomal Diseases
Shoshana Revel-Vilk
15:10 - 15:30
15:30 - 15:50
Coffee break
15:50 - 16:10
Evaluating osteonecrosis using AI-based technology.
Patrick Deegan
15:50 - 16:10
16:10 - 16:50
Assessment of bone involvement using the applications of Machine Learning.
Ravi Kamath
16:30 - 16:50
Q&A
16:30 - 16:50
17:30 - 18:00
Cocktail
18:00 - 20:30
Dinner
18:00 - 20:30
Day 2
4. Theranostics in Lysosomal Disorders.
09:00 - 09:20
Lysosome Exocytosis as a Therapeutic Target in Cancer Progression
Duarte Barral
09:00 - 09:20
09:20 - 09:50
Mitochondria-lysosome crosstalk in physiology and disease.
Nuno Raimondo
09:50 - 10:10
Targeting Mechanisms for bone-directed therapies.
Betul Celik
09:50 - 10:10
10:10 - 10:30
Base editing and prime editing to address genetic disease
Gregory Newby
10:30 - 10:50
Q&A
10:30 - 10:50
10:50 - 11:00
Coffee Break
5. Current issues in Gene Therapies for Lysosomal Disorders.
11:00 – 11:20
AAV Gene therapy and clinical trial for MPS IVA
Shunji Tomatsu
11:00 – 11:20
11:20 – 11:40
Sialidosis: Mechanisms of Pathogenesis and Therapeutic Modalities
Alessandra D’Azzo
11:40 – 12:00
MPSIIIC Gene Replacement Therapy with scAAV9/HGSNAT Vector.
Merve Emecen Sanli
11:40 – 12:00
12:00 – 12:20
Patient preferences for Gene Therapy or Future of the funding and reimbursement of Gene Therapy for adult-onset LDs
Ryan Colburn
12:20 - 12:50
Q&A - closing remarks
12:20 - 12:50
12:50
Lunch box to go
01:30 - 05:00
GD & Bone clinic
By invitation only
01:30 - 05:00