Thomas Weber Email & Phone Number

I’m an experienced leader in gene therapy and a specialist in molecular virology, particularly the cell biology of AAV transduction. My work is focused on efficiently delivering therapeutic DNA to the desired tissue and cell types, which is still the most formidable hurdle to harness gene therapy to help patients. This is even the case with the arguably most promising DNA delivery vehicle, recombinant AAVs. I’m using cutting-edge technology to isolate and design novel AAV variants that efficiently deliver their therapeutic payload to the desired tissue or cell type. For this, I draw on my extensive experience in capsid engineering and my in-depth knowledge of molecular virology and cell biology. Rigorous validation of novel AAV variants in non-human primates and human cells and organoids is critical to utilize these AAV variants in the clinic and commercialize them to help patients.Areas of Expertise:• Foster a dynamic, inclusive and supportive work environment.• Promote collaborative efforts among teams with different skill sets to achieve company goals.• Ensure that the research meets the most rigorous standards.• Identify the most important barriers and devise strategies to overcome them.• Utilize my research experience in life sciences to reach company objectives and meet timelines.Selected Career Achievements• As Senior Director of Viral Platforms built the capsid engineering team at Spark Therapeutics.• Established a combination of two AAV library-based screening approaches to isolate AAV variants that display the same tissue/cell tropism in humans and NHPs.• Started a selection campaign for tissue-specific AAV variants in NHPs.• Contributed to preclinical studies for the treatment of heart failure with AAV gene therapy. These studies resulted in a phase 1 trial and an ongoing phase 2 clinical trial.• Validated a promoter/enhancer combination for the exclusive expression of a therapeutic gene in the heart.• Established an approach to deplete anti-AAV antibodies from blood.• Proved that mosaic AAV capsids are useful tools to alter AAV tropism• As a postdoctoral fellow in the laboratory of Dr. James E. Rothman I identified the minimal machinery for membrane fusion resulting in a cover article in Cell. This article is among the top 1% cited articles and was prominently featured in Dr. Rothman’s 2013 Nobel Prize lecture.