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Meritxell Teixidó, CEO y CSO de Gate2Brain. Foto / FBG
 17.06.2025

Gate2Brain obtains FDA orphan drug designation for its drug G2B-002

The biotechnology company Gate2Brain, headquartered at the Barcelona Science Park, has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for its lead candidate, G2B-002, an innovative therapy aimed at treating diffuse intrinsic pontine glioma (DIPG) and pediatric glioblastoma (pGBM), two of the most lethal pediatric brain tumors with very limited treatment options. This designation adds to the one granted by the European Medicines Agency (EMA) at the end of 2024 and underscores the significant potential of this therapy in both Europe and the United States.

The company, a spin-off from the University of Barcelona (UB), the Institute for Research in Biomedicine (IRB Barcelona), and the Sant Joan de Déu Research Institute (IRSJD) – Sant Joan de Déu Hospital (SJD), specializes in the development of drug delivery systems across biological barriers. In this context, its lead candidate, G2B-002, enhances drug transport and penetration across the blood-brain barrier, increasing efficacy while minimizing systemic side effects.

Endowed with this FDA designation, G2B-002 benefits from enhanced regulatory support and strategic momentum, smoothing its transition from advanced preclinical development into clinical trial phase.

“Receiving ODD from both the EMA and US FDA reaffirms our conviction in G2B-002’s potential to address critical unmet needs in pediatric oncology,” said Meritxell Teixidó, CEO & CSO of Gate2Brain. “This dual recognition brings us a step closer to clinical trials and, ultimately, to offering new hope for young patients and their families.”

Gate2Brain remains dedicated to transforming the treatment landscape for pediatric brain tumors through innovative delivery solutions. The company has received support from institutions including Fundación Botín (Mind the Gap), Banco Sabadell (BStartup Health), CDTI (Neotec, Multipaís), the European Innovation Council (EIC Accelerator), Startup Capital d’ACCIÓ and CaixaResearch Consolidate de la “Fundació la Caixa”.

» Link to the news: Gate2Brain website [+]