Gate2Brain, a cutting-edge technological platform for improving the transport of drugs in the brain, has achieved a decisive milestone in the fight against pediatric brain tumors by receiving Orphan Drug Designation (ODD) from the European Agency for Medicines (EMA) for its innovative product, G2B-002.
This designation means that G2B-002 has been recognized as a potential treatment for a rare and serious disease, and is given to medicines for conditions that affect fewer than 5 in 10,000 people in the European Union and that offer significant benefits against of limited or non-existent options.
Three key points of Gate2Brain's achievement
1. EMA Orphan Drug Designation: G2B-002 has received EMA Orphan Drug Designation, a milestone achievement that accelerates its progress toward clinical trials for the treatment of pediatric brain tumors, supported by a team of experts in biotechnology and pediatric neuro-oncology.
2. Innovation in the transport of drugs: Gate2Brain has developed a patented technology that allows drugs to cross the blood-brain barrier (BHE) more effectively, increasing the accuracy and effectiveness in the treatment of brain tumors.
3. Social and scientific commitment in pediatric oncology: in its first indication, G2B-002 is specifically aimed at diffuse intrinsic pontine glioma (DIPG) and pediatric glioblastoma (pGBM), aggressive brain tumors that represent one of the main causes of mortality for childhood cancer
Innovation driven by an Industrial Doctorate project
Gate2Brain actively participates in the Industrial Doctorates Plan , an initiative that promotes applied research in collaboration with the academic and business fields. The company collaborates with the Sant Joan de Déu Research Institute (IRSJD) in an Industrial Doctorate project led by doctoral student Clàudia Resa , under the supervision of researcher Dr. Ángel Montero and the CEO of Gate2Brain, Dra. Meritxell Teixidó .
This project explores the use of peptide shuttles to improve the transport of chemotherapeutic agents across the blood-brain barrier, with the aim of combating pediatric brain tumors that, so far, have no effective treatment. This collaborative approach is an outstanding example of how Industrial Doctorates connect academic knowledge with its practical application to generate a real impact on people's health.
Impulse to Transform the Treatment of Pediatric Brain Cancer
The designation of G2B-002 as an orphan drug by the EMA marks a decisive step in the development of new therapies for complex pediatric cancers, particularly those affecting the brain. Gate2Brain's patented technology facilitates drugs to cross the blood-brain barrier (BBB) with high precision, increasing both the efficacy and safety of brain tumor treatment.
G2B-002, Gate2Brain's most advanced product and proof-of-concept of its technology, is based on a peptide transport system that delivers an anticancer agent. This system has proven to be effective against pediatric brain tumors with intact BHE, such as pediatric glioblastoma (pGBM) and diffuse intrinsic pontine glioma (DIPG), the first indications to benefit from this technology.
In addition, the results obtained have the potential to be applied to other therapeutic agents and biological barriers, with significant clinical implications.
Innovation and Commitment to the Child Patient
With a research program focused on pediatric brain tumors as its first indication, Gate2Brain seeks to leverage the EMA recognition to bring G2B-002 into clinical trials and open a new era of therapeutic options in an area of high unmet need. . " We are moving towards clinical trials that could transform the treatment of pediatric brain tumors ", says Meritxell Teixidó, CEO of Gate2Brain, highlighting the firm commitment of the company to pediatric patients and their families.
About Gate2Brain Gate2Brain is a technology platform, spin-off of IRB Barcelona, the University of Barcelona and Hospital Sant Joan de Déu, founded in 2020. Gate2Brain uses its patented technology based on peptides to cross biological barriers, such as the blood-brain barrier (BHE), with the aim of improving the effectiveness of drug transport in the brain and reducing side effects. The company has received the support of institutions such as Fundació Botín (Mind the Gap), Banco Sabadell (BStartup Health), the CDTI, the European Innovation Council (EIC Accelerator), ACCIÓ's Startup Capital and CaixaResearch Consolidate "la Caixa" Foundation. Gate2Brain has the vision to improve the quality of life of patients through a technology that provides greater efficiency in the transport of drugs.
