DCRT was represented by Annemieke Aartsma-Rus at a meeting of the European Medicines Agency (EMA) on RNA therapy
On February 2nd Annemieke Aartsma-Rus presented at a meeting on RNA therapy organized by the European Medicines Agency (EMA). She presented the state of the art for single stranded oligonucleotides, how splice modulating oligonucleotides can be exploited for individual mutations for patients with progressive brain diseases, and introduced how these are being developed within the setting of the DCRT and the 1 Mutation 1 Medicine network.
During the panel discussion and the EMA press conference on February 3rd she stressed the importance of starting a dialogue with regulators early, especially for academics as they have generally very little expertise with the regulatory system. She underlined her positive interaction with the Innovation Task Force and constructive feedback provided to the DCRT.
posted on February 9, 2023
