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

Target gene
(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)

How Logo mygenerafunctions

The innovations

Development of oligonucleotide gene-specific medicines
Identification time of 2/3 months for the candidate-drug molecule instead of the conventional 2/3 years
Greater medicine efficacy
Reduced or absent therapy toxicity
Potentially efficacious applications in all therapeutic areas

The first application:
paediatric tumours

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 for the cure of Neuroblastoma from the European Medicines Agency (EMA) and from the Food and Drug Administration (FDA) in USA, and of the group of Soft Tissue Sarcomas (that include Rhabdomyosarcoma and Synovial Sarcoma) by EMA.

A concrete answer
for all pathologies

The second application:
adult tumors

Subsequently the the anti-cancer drug BGA002 will be used for the treatment of several adult tumors with MYCN gene alterations (that is associated with poor prognosis). In particular, the first indication will be for the treatment of the Small Cell Lung Cancer (the most deadly type of lung cancer), for which BGA002 has already obtained the Orphan Drug designation by the FDA.

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.