Gene therapy brings new hope for those suffering from heart failure, Parkinson's

PAIN—For many Filipinos, diseases like heart failure and Parkinson’s disease remain life-changing diagnoses with few lasting solutions.

- By ELLALYN DE VERA-RUIZ

Current treatments can ease discomfort and slow progression, but they often fall short of addressing the root cause of illness.

Gene therapy is emerging as one of the most promising frontiers, offering the possibility of correcting the root of disease instead of simply managing symptoms.

Atthe heart of this effort is Bayer, the multinational pharmaceutical and biotechnology company investing heavily in emerging science through its subsidiaries AskBio and Viralgen, both based in San Sebastian, Spain.

Together, they are working to transform gene therapy from theory into treatment.

Redefining viruses as medicine

AskBio Chief Executive Officer (CEO) Gustavo Pesquin said the concept of gene therapy came from looking at viruses in a different way.

Instead of seeing them solely as agents of illness, scientists realized the same ability that allows a virus to enter human cells and reach the nucleus could be used to deliver something beneficial.

“They started with this virus that is highly prevalent in society called AAV (adeno-associated virus). It is very innocuous. It does not cause any particular disease. Basically, they clean all the genetic material inside, and that was the ship used to get inside the cell and the nucleus and try to create the medicine,” Pesquin explained.

Inside this carrier is what he described as a cassette, made up of three essential parts: the transgene, which carries the instructions to repair what is broken; the promoter, which directs the therapy to the right part of the body, whether it is the brain, the heart, or another organ; and the capsid, the protective shell that allows the therapy to travel safely into the cells.

“This combination allows us to target the cause of the disease. Instead of taking medicine every day for life, the goal is to intervene once and have a lasting effect,” he added.

Scaling innovation