Research and Innovation

Research Overview
Biogenera is at the forefront of biopharmaceutical research and is dedicated to developing innovative, effective, and safe therapies. We focus on serious and currently incurable diseases, with the goal of bringing treatments where none yet exist.
Innovative Biotechnologies
The proprietary MyGenera™ platform is at the core of Biogenera’s innovation. This revolutionary biotechnology enables the rapid and precise identification of new “anti-gene” drug candidates, specifically targeting a single gene identified as the cause of a particular disease, offering a pioneering approach to treatment. Unlike traditional “antisense” approaches that aim to inhibit RNA translation, MyGenera™ acts directly at the source—the gene—by blocking the transcription of DNA into RNA, known as the anti-gene approach.
At the core of the process is a proprietary bioinformatic approach that enables the rapid identification of a limited number of PNA-based anti-gene oligonucleotides (Ag-PNA) capable of specifically blocking the target gene and meeting predefined selection criteria. The platform allows for fast and accurate evaluation, ensuring that only compounds with the highest therapeutic potential, specificity, and safety proceed through the development process. The subsequent refinement involves further molecular and cellular pharmacodynamic evaluations, following increasingly stringent criteria to select the optimal Ag-PNA (the drug candidate).
The Advantages of the MyGenera™ Platform
This approach not only increases treatment effectiveness but also offers an improved safety profile, which is essential for treating serious diseases. The use of the MyGenera™ platform has already led to the discovery of promising drug candidates, such as BGA002, which has successfully completed preclinical studies and has proven to be effective and safe in preclinical models against aggressive pediatric and adult tumors expressing the MYCN gene—known for its involvement in several cancer characteristics such as metastasis, drug resistance, immunosuppression, and neoangiogenesis. BGA002 has received orphan drug designation from both the EMA and FDA for the treatment of Neuroblastoma, Soft Tissue Sarcomas, and Small Cell Lung Cancer.
01
Precision

Specifically targets the causal gene of the disease, maximizing therapeutic efficacy while avoiding off-target toxic effects.
02
Efficiency

By acting at the source (gene inhibition), it allows for a reduced drug dosage and lower frequency of administration compared to traditional therapies, improving patient compliance.
03
Versatility

Suitable for any disease in which the selective silencing of a target gene can have a therapeutic effect.
We work in the oncology field with main focus on
Pediatric tumors
BIOGENERA's research has led to the identification and development of the first specific drug, the anti-MYCN anticancer agent BGA002, for the treatment of childhood cancers with alterations in the MYCN oncogene (which is associated with a poor prognosis). Phase 1 clinical trials are expected to begin in the coming months, particularly for Neuroblastoma (currently the deadliest childhood cancer), as well as other pediatric solid tumors and some acute leukemias. Demonstrating its high level of innovation, the drug has already received Orphan Drug designation from both the European Medicines Agency (EMA) and the U.S. FDA for the treatment of Neuroblastoma and Soft Tissue Sarcomas (including Rhabdomyosarcoma and Synovial Sarcoma).

The second application
Adult tumors
Subsequently, the new anti-MYCN anticancer drug BGA002 will be used for the treatment of adult cancers with alterations in the MYCN oncogene (which is associated with a poor prognosis). In particular, in Small Cell Lung Cancer (SCLC), the deadliest form of lung cancer, for which the drug has already received Orphan Drug designation from the U.S. Food and Drug Administration (FDA).

A concrete answer for
All pathologies
The innovation offered by MyGeneraTM is applicable to any disease, acting at the level of mutations or gene-specific alterations. BIOGENERA’s research is currently focused on the most aggressive tumors, currently without treatment, and on pathologies that are fatal or very disabling for the quality of life, and have a high impact on the patients’ families and on healthcare services.
