Catalyst Award
Rebecca Ahrens-Nicklas, MD, PhD, Kiran Musunuru, MD, PhD, MPH, ML, MRA, Fyodor Urnov, PhD, and Danaher Corporation
May 12, 2:32 - 2:59 p.m. EST

George Stamatoyannopoulos Memorial Outstanding Achievement Award
Beverly Davidson, PhD 
May 14, 1:30 - 3p.m. EST

May 12

William Peranteau, MD 
Germline Editing: Technical Background Perinatal
10:15 - 10:35 a.m. EST

Denise Sabatino, PhD
Dissecting SUMOylation-mediated mechanisms that regulate AAV transgene expression
4 - 4:15 p.m. EST

May 13

Lindsey George, MD 
Evolving understanding of AAV toxicities 
10:15 - 10:41 a.m. EST

Benjamin Samelson-Jones, MD, PhD (Session Co-Chair) 
Recent toxicities in AAV gene therapies: Translational lessons from preclinical and clinical studies 
10:15 a.m. - noon EST

Rebecca Ahrens-Nicklas, MD, PhD, and Kiran Musunuru, MD, PhD, MPH, ML, MRA
Clinical translation of somatic genome editing as a therapeutic platform 
3:30 - 3:45 p.m. EST

Lindsey George, MD 
AAV and systemic toxicities in clinic trials and clinical practice  
3:30 - 3:52 p.m. EST

Top Abstract: Lindsey George, MD
Neuroepithelial Tumor Associated with AAV Integration After Intracisternal Magna Vector Delivery

May 15

Peter Kurre, MD 
Positive clonal selection of curative HSCs - introductory component 
10:15 - 10:41 a.m. EST

Benjamin Samelson-Jones, MD, PhD
Multiyear stable transgene factor IX levels after AAV hepatocyte-directed gene in neonatal and juvenile hemophilia B dogs 
3:30 - 3:45 p.m. EST

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