Friedreich’s Ataxia Drug Developer RaNA Therapeutics to Present Data at Conference

RaNA Therapeutics, a Cambridge, Massachusetts-based biotech company that develops next-generation RNA-targeted medicines and which currently has a lead program in Friedreich’s Ataxia, announced that its CEO Ronald Renaud will present a corporate overview on Feb. 8 at the 18th annual BIO CEO & Investor Conference in New York City. RaNA’s management will also make a corporate presentation at a conference at the Leerink Partners 5th annual Global Healthcare Conference on Feb. 11. Both conferences will take place at the Waldorf Astoria.

Friedreich’s Ataxia (FA), a rare and life-threatening neuromuscular disease, is usually caused by an expansion of the GAA repeat in the Frataxin (FXN) gene, which results in a reduction of the expression of the FXN protein. This protein is an important part of the function of mytochondria, the energy-generating organisms inside all cells. RaNA’s scientific technology is based on the development of RNA-targeted medicines that increase gene expression. These medicines consist of highly specific oligonucleotides, molecules that are designed by scientists to bind to certain areas of RNA (ribonucleic acid), the nucleic acid transcribed from DNA, in a process called transcription, that will give rise to the protein.

The oligonucleotides designed by RaNA can affect RNA activation in two ways. In the first approach, oligonucleotides act during transcription, the process where DNA gives rise to RNA, preventing the action of chromatin modifiers — complexes that can suppress the expression of genes.

In the second strategy, the process happens at a post-transcriptional level, after DNA is copied into RNA but before the protein is made (translation). This last approach is being used in Friedreich’s Ataxia research through oligonucleotides that stabilize the mRNA within the cells and increase the half-life, allowing for a higher availability of the proteins they give rise to. This approach is expected to result in a higher production of the FXN protein, the major default in Friedreich’s Ataxia. By raising the FXN protein to healthy levels, researchers expect to treat and prevent the disease.

Both conferences in New York City next week will be also webcasted through RaNA’s website.