For BIOGENERA SpA, DNA, the distinctive element that makes everyone unique, is the real target of gene-specific therapy. While traditional pharmacological approaches act on downstream therapeutic targets (protein or mRNA), the proprietary biotechnological platform MyGeneraTM permits the identification of new candidate medicines capable of acting directly at the origin (DNA), or rather at the gene level and in a highly specific manner.
Candidate medicines, identified through MyGeneraTM, are constituted of oligonucleotides of DNA capable of blocking, in a powerful and highly selective way, the transcription of mutated and pathological genes. This new and unique pharmacological approach allows the causes and not the symptoms of the illness to be directly acted upon (as occurs with traditional medicines). Thus opening the doors for curing the onset of serious pathologies such as tumors, genetic, cardiovascular, neurodegenerative and infective diseases.
Gene-specific therapies and their mechanisms
The new category of oligonucleotide gene-specific medicines is characterised by a short chemical sequence of modified DNA that acts on a specific pathological target gene.
An anti-gene is an oligonucletoide able to inhibit the transcripstion of a target gene ino mRNA
(two copies per cell)
The anti-gene oligonucletoide works by forming a stable duplex with the complementary DNA sequence, binding to the open DNA structure of an active gene, thereby blocking transcription
(Thousands of copies)
The technology is applicable to any disease where selective silencing of a target gene will have therapeutic effect eg. oncology, dominant genetic diseases, viral/bacterial infections.
(Thousands of copies)
The first application:
The research done by BIOGENERA SpA has led to the identification and development of the first specific drug, the anti-tumoral anti-MYCN BGA002, for the cure of tumors of children with alterations of the tumoral gene MYCN (given a poor prognosis). Within a few months BIOGENERA will start the phase 1 clinical studies, specifically in Neuroblastoma (currently the more lethal tumor in children). On demonstrating its high level of innovation, the medicine has already obtained status of Orphan Drug from the European Medicines Agency (EMA) for the cure of Neuroblastoma and of the group of Soft Tissue Sarcomas (that include Rhabdomyosarcoma and Synovial Sarcoma)
A concrete answer
for all pathologies
The innovation offered by MyGeneraTM is applicable to any illness, acting specifically at the level of mutated and altered genes. BIOGENERA’s research is at present concentrated on the more aggressive tumors (those currently lacking treatment) and on pathologies that are lethal or highly debilitating on quality of life, with a high impact on patients’ families and on the health service.