SoVarGen unveils preclinical data of ASO drug for brain tumor at OTS

[Photo provided by SoVarGen]

SoVarGen, a Korean biotech startup that develops a pipeline of RNA therapeutics for brain diseases, said Wednesday it announced research results of a preclinical trial of its antisense oligonucleotide (ASO) drug for a pediatric brain tumor at the Oligonucleotide Therapeutics Society (OTS) meeting held in Phoenix, Arizona, USA on Oct. 2-5.

The academic conference which marks its 18th year is an open, nonprofit forum to foster research and development of oligonucleotide therapeutics. Global players like Biogen and Moderna were among major participants this year.

Oligonucleotide therapeutics are being used to successfully treat diseases and have the potential for widespread application. Over 100 treatments are in development for common conditions, including cancer and Alzheimer’s disease, as well as rare diseases.

Biogen which presented data on its investigational ASO drug for Lou Gehrig’s disease at the event has a pipeline of 27 drugs in development for brain disease, of which seven are in the ASO class. This means ASO technology has been established as a potential therapy for various brain and neurological diseases.

Biogen, a leader in the development of innovative therapies for brain diseases, recently gained attraction for lecanemab’s strong phase 3 data in Alzheimer’s disease.

SoVarGen’s chief science officer and KAIST professor Lee Jeong-ho was the only Korean expert invited to speak at the conference.

In a paper published in Nature Medicine in 2018, Lee found that brain stem cell-derived BRAF mutations are the cause of seizures and tumors in pediatric brain tumors. At this year’s OTS, he published data in a follow-up study that a single injection of the company’s BRAF ASO drug suppressed seizures and tumor growth for three months in an animal model that mimics a pediatric brain tumor with seizures.

SoVarGen also presented a poster detailing the status of ASO drug research for refractory epilepsy.

Focal cortical dysplasia is the most common pediatric refractory epilepsy, but there is no treatment yet. SoVarGen is leading the pack in the development of a drug for this disease using its ASO platform technology and is currently conducting a pre-clinical trial.

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