Doctoral School

Gene Therapy

Cluster

Research and Valorization

Target audience

PhD students, postdocs and lab technicians working in the field of gene therapy

Teacher

Prof. Dr. Frauke Coppieters, Ghent Therapy and RNA group, UGent.

Prof. Coppieters is associate professor within RARE-MED, a Ghent University multidisciplinary consortium for basic and translational research on precision medicine for rare diseases. She is also member of the steering committee of Gene, Cell Tissue engineering (GATE), the European Retinal Disease Consortium (ERDC, www.erdc.info) and the ProgRET doctoral network (www.progret.eu). Her current research focuses on retinal disease modelling and new genetic therapies for inherited blindness. In specific, the group uses antisense oligonucleotides (ASOs) to modulate gene expression, and aims to develop a platform for functional evaluation of ASO treatment in patient-derived 2D and 3D retinal models.

 

Prof. Dr. Stefaan De Smedt, Laboratory of General Biochemistry and Physical Pharmacy, UGent

Prof. De Smedt founded the Ghent Research group on Nanomedicines involving pre-clinical research on nanomedicines, more specifically nanoscopic carriers to improve targeted and intracellular delivery of nucleic acids (siRNA, µRNA, mRNA, pDNA), being a whole class of molecules which are not yet clinically used though under intensive development. He is member of the steering committee of GATE and guest professor at various universities in Belgium and China. In addition, he serves as Editor-in-Chief of the Journal of Controlled Release.

Prof. Luk Vandenberghe, Grousbeck Gene Therapy Center, US

Prof. Vandenberghe is an associate professor at Harvard Medical School and associate member of the Broad Institute of Harvard and MIT in Boston, MA, USA. He directs the Grousbeck Gene Therapy Center at Massachusetts Eye and Ear Infirmary in Boston, USA, which is part of the Ocular Genomics Institute, a bench to bedside research program to study, diagnose, and develop treatments for diseases of the eye. His previous work led to the discovery of novel AAV serotypes such as AAV9, novel insights into AAV structure-function, and vector immunobiology. His laboratory aims to gain a deeper understanding of mechanisms of gene transfer to develop technologies to overcome hurdles to gene therapy clinical applications, and to translate specific gene therapy programs in vision, hearing, and other fields.  Prof. Vandenberghe has over 50 peer reviewed publication and more than a dozen licensed patents, mostly related to gene therapy methods, technologies, and applications.

 

Prof. Dr. Robrecht Raedt, 4Brain, UGent

Prof. Raedt is an associate research professor and principal investigator of the 4BRAIN lab at Ghent University. He performs research on the neural basis of behavioral control using intracranial recording during cognitive testing in human as well as in animal models. Currently, he is exploring the potential of in vivo gene therapy and photopharmacology as a potential targeted therapy for focal epilepsy. In addition, he is director of the animal facility of the faculty of Medicine and Health Sciences, member of the GATE steering committee and secretary of the Belgian Society for Neuroscience.

Prof. Dr. Frederic Acke, Otology and Audiology group, UGent & UZ Gent

Prof. Acke is an otorhinolaryngologist with a PhD on genetic hearing loss. His main research focus is the exploration of mechanisms and innovative treatment strategies, including gene therapy, for inner ear disorders. He is member of the otology-audiology group (department of Head and Skin, UGent) that built up expertise in the field of hearing loss, with a focus on genetic research of deafness, (re)habilitation of hearing loss and balance.

 

Prof. Dr. Bart Leroy, Dept. of Ophthalmology, UGent & UZGent

Prof. Leroy is clinical geneticist and ophthalmologist at UZ Ghent. He founded the Ophthalmic Genetics and Visual Electrophysiology division at Ghent University hospital and is involved in the development of several gene therapies. He was part of the team that developed Luxturna, the first approved gene augmentation therapy, and subsequently helped bring it to market. He is also part of the nuclear team investigating the first oligonucleotide therapy in man for CEP290-associated inherited retinal disease. He is organiser of several multi-stakeholder meetings with IRD experts, patient organisations, pharmaceutical industry and regulatory agencies to improve mutual understanding of potential outcomes of innovative retinal gene therapies.

 

Dr. Tim Desmet, Advanced Cell & Tissue Engineering.

Dr. Tim Desmet is business developer in advanced cell and tissue therapies at Ghent University. He obtained a Master of Science in Business Administration and is an early investor in two start-ups. He co-founded two companies and is actively involved in an investment fund. Additionally, he is member of the business team of GATE.

 Dr. Ineke Imbo, Direction communication and marketing

Dr. Imbo was a post-doctoral researcher at the Experimental Psychology lab in Ghent University and at the centre for applied cognitive research at the Carleton University in Ottawa, Canada. Currently, she is involved in science communication at Ghent University, working on several projects promoting science for both children and adults such as Wetenschapscafe or Dag van de wetenschap.

 

Abstract

Gene therapy is rapidly emerging to treat rare diseases and includes various strategies such as gene augmentation, antisense oligonucleotides and gene editing. This doctoral school course aims to provide a comprehensive overview of current gene therapy technologies complemented by examples from our university and beyond. Additionally, we will cover the gene therapy development process, from early-stage research and valorisation to human clinical trials.

Objectives

  1. current gene therapy technologies
  2. gene therapy delivery systems
  3. the valorisation process and clinical trials
  4. how to effectively communicate your research to patients and the general public the perspectives of patients as well as companies on gene therapy

Dates and venue

07 February 2025

Auditorium C, UZ Gent (entrance 48)

Programme

Part 1: Gene therapy - technologies

  • 9u-9u35: Overview of genetic therapy strategies (prof. Frauke Coppieters, Ghent Therapy and RNA Group, UGent)
  • 9u35-10u10: Gene therapy delivery through nanomedicines (prof. Stefaan De Smedt, Laboratory of General Biochemistry and Physical Pharmacy, UGent)
  • 10u10-10u55: Delivery through viral vectors (prof. Luk Vandenberghe, Grousbeck Gene Therapy Center, US)

Break (15min)

 

Part 2: Gene therapy - applications

  • 11u10-11u45: Gene therapy for brain disease with a focus on epilepsy (prof. Robrecht Raedt, 4Brain, UGent)
  • 11u45-12u20: Gene therapy for inherited deafness (prof. Frederic Acke, Otology and Audiology group, UGent & UZGent)
  • 12u20-12u55: Gene therapy for inherited blindness (prof. Bart Leroy, Dept. of Ophthalmology, UGent & UZGent)

Lunch (13:00-14:00)

 

Part 3: Gene therapy - development

  • 14u-14u35: Valorization and Good Manufacturing Practices (GMP) (dr. Tim Desmet, UGent)
  • 14u35-15u10: Gene therapy clinical trials (HIRUZ, UZGent)
  • 15u10-15u45: A company’s perspective (TBD)

Break (15min)

  • 16u-16u35: Science communication (Ineke Imbo, UGent)
  • 16u35-17u10: A patient’s perspective (TBD)

Registration

Registration fee

Free of charge for Doctoral School members.

Number of participants

Maximum 100

Language

English

Training method

Presentations by international and faculty professors

Evaluation method

After successful participation, the Doctoral School Office will add this course to your curriculum of the Doctoral Training Programme in Oasis. Please note that this can take up to one to two months after completion of the course.