Highlights from Gene Therapy in Rare Diseases Europe

London October 25-27, 2022

CombiGene’s Senior Program Director Alvar Grönberg recently attended the conference Gene Therapy in Rare Diseases Europe. Here are his main take-aways from the conference and its many workshops.

“Gene Therapy in Rare Diseases proved to be very useful. One thing that really struck me was how vibrant the gene therapy community is. There are so many people working hard to develop novel therapies with the goal of improving the life of patients suffering from any of the rare disease for which there is still no treatment. Breaking new ground is never easy.
As Oscar Segurado of AsC Therapeutics put it at the conference: ‘Development of a gene therapy product is like building an international space station while small molecule drug development is much simpler, like building a car.’
I find this statement to be very true. There are so many details that all require meticulous attention. The conference was filled with several interesting themes, such as new gene therapies for rare diseases, safety concerns and challenges in gene therapy, the regulatory path for orphan drugs, the importance of patient advocacy groups, ideas for long-term follow up of patients, and much more. All in all, I had three very useful and productive days at the conference, and my report back to the company was unusually long.”