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Meritxell Teixidor, CEO of Gate2Brain. Image / IRB Barcelona.
 20.11.2024

Gate2Brain receives EMA Orphan Drug Designation for groundbreaking pediatric cancer treatment

Gate2Brain -based in the Barcelona Science Park and focused on enhancing drug delivery to the brain through its cutting-edge technology platform- has achieved a significant milestone. The European Medicines Agency (EMA) has granted Orphan Drug Designation (ODD) to its innovative product, G2B-002, a crucial step forward, accelerating its progress toward clinical trials for pediatric brain tumors treatment.

Gate2Brain’s most advanced product, G2B-002, is based on a peptide transport system that delivers an anti-cancer agent that targets diffuse intrinsic pontine glioma (DIPG) and pediatric glioblastoma (pGBM), two aggressive brain tumors that are among the leading causes of childhood cancer mortality.

With a research programme initially focused on pediatric brain tumors.  Gate2Brain aims to leverage EMA’s recognition to advance G2B-002 to clinical trials and pave the way for a new era of treatment options in a high unmet need area.

We are moving toward clinical trials that could transform the treatment of pediatric brain tumors”, explains Meritxell Teixidó, CEO of Gate2Brain, underscoring the company’s strong commitment to pediatric patients and their families.

A Boost to Transform Pediatric Brain Cancer Treatment

Gate2Brain -founded in 2020 as a spin-off from the Institute for Research in Biomedicine (IRB Barcelona), the University of Barcelona (UB) and the Sant Joan de Déu Research Institute (IRSJD)- Hospital Sant Joan de Déu (SJD)- has developed a patented technology that allows drugs to cross the blood-brain barrier (BBB) more effectively, increasing precision and efficacy in treating brain tumors.

G2B-002, Gate2Brain’s most advanced product and a proof of concept for its technology, is based on a peptide transport system that delivers an anti-cancer agent. This system has demonstrated efficacy against pediatric brain tumors with an intact BBB, such as pediatric glioblastoma (pGBM) and diffuse intrinsic pontine glioma (DIPG), the first conditions to benefit from this technology.

Furthermore, these results show potential applications for other therapeutic agents and biological barriers, with significant clinical implications.

» For further information: Gate2Brain website [+]