Thomas Weber Email and 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.
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Ceo And FounderGene Therapy ConsultingPhiladelphia, Pa, Us -
Senior Director Of Viral PlatformsSpark Therapeutics, Inc. Sep 2021 - Sep 2024Philadeliphia, Pa, UsaEstablished and lead the first Spark capsid engineering team and planned and managed a yearly Opex budget of ~$1.5M. Guide capsid team in developing novel approaches to select AAV variants that transduce specific tissues / cell-types. Directing team to achieve company’s KPIs and meet project timelines. -
Senior Director Of Viral PlatormsSpark Therapeutics, Inc. Sep 2021 - Sep 2024Philadelphia, Pennsylvania, United StatesBuilt 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. -
Associate ProfessorIcahn School Of Medicine At Mount Sinai Jan 2016 - Sep 2021New York, Ny. United States Of AmericaPerformed research to study AAV Biology, enabling AAV gene therapy to treat heart failure and cardiomyopathies. Designed AAV vector genomes that result in cardiac-specific overexpression of gene of interest; designed a method to deplete neutralizing anti-AAV antibodies from blood, collaborating with colleagues to achieve research goals. Assisted graduate students and postdoctoral fellows in becoming independent researchers and guided them in presenting their work. -
Assistant ProfessorMount Sinai School Of Medicine Dec 1999 - Dec 2015Nyack, New York, United StatesSet up a laboratory to study biology of adeno-associated viruses and develop methods to improve properties of current AAV variants. Secured external funding for research from sources such as March of Dimes, Department of Defense and National Institutes of Health (NIH). Taught cell biology classes to graduate students. -
Research InstructorMemorial Sloan Kettering Cancer Center Jan 1997 - Nov 1999New York, United StatesContinuing my work during my postdoctoral fellowship in the laboratory of Nobel Laureate Dr. James E. Rothman, I demonstrated that SNAREs are the minimal machinery for membrane fusion. The cover article in Cell describing this work is among the top 1% in citations in published journal articles. -
Postdoctoral FellowMemorial Sloan Kettering Cancer Center Apr 1994 - Dec 1996New York, New York
Thomas Weber Education Details
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Biochemistry -
Biochemistry And Organic Chemistry
Frequently Asked Questions about Thomas Weber
What company does Thomas Weber work for?
Thomas Weber works for Gene Therapy Consulting
What is Thomas Weber's role at the current company?
Thomas Weber's current role is CEO and Founder.
What schools did Thomas Weber attend?
Thomas Weber attended Eth Zürich, Eth Zürich.
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Thomas Weber
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Thomas Weber
Political Science And History Student At University Of Illinois Urbana-ChampaignPark Ridge, Il -
Thomas Weber
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