Bioindustry Focuses on Optogenetics Nobel Prize... Will the First Therapy Emerge for Retinal Diseases?

Market Reaction Remains Tepid on Clinical Outcomes Despite Nobel Prize
First Commercialization Expected in Retinal Disease... FDA Approval Under Review
Optogenetics Used to Identify New Drug Targets in Brain Disorders

This year, the Nobel Prize in Physiology or Medicine was awarded to researchers who contributed to the development of optogenetics, drawing increased attention to the bioindustry utilizing related technologies. However, since there have been no cases where therapeutics based directly on optogenetics have been commercialized, the industrial potential of this technology is currently being put to the test.

According to the bioindustry on the 7th, optogenetics is a technology that controls the activity of specific neurons using light and has become a key tool in neuroscience research over the past 20 years. In drug development, it is being used to identify brain circuits responsible for diseases and to select drug targets.

MapLight, co-founded in 2018 by Nobel laureate and Stanford University professor Karl Deisseroth, is also developing new drugs by identifying brain circuits associated with symptoms in animal experiments using optogenetics and discovering drug targets within those circuits through spatial transcriptomics technology. MapLight was listed on Nasdaq in the U.S. in October last year, raising $296.3 million (about KRW 398.1 billion).

However, this year’s clinical results have failed to meet market expectations. The candidate substance for autism spectrum disorder did not meet the primary endpoint in its phase 2 clinical trial in June. The candidate substances for schizophrenia and Alzheimer’s-related psychosis achieved the primary endpoint only in the twice-daily dosing group in the phase 2 trial for schizophrenia in July, while the once-daily dosing group—with greater commercial potential—did not reach statistical significance.

On the day of the Nobel Prize announcement, October 5 (local time), MapLight’s stock price also closed at $9.28 (about KRW 12,470), down 5.55% from the previous trading day. This is about half of the IPO price and not far from its 52-week low. Despite the positive news of the Nobel Prize, the market appears to value clinical success of pipeline candidates more than the optogenetics platform itself.

Currently, the therapeutic field closest to commercialization for optogenetics is retinal diseases. This involves inserting genes for light-sensitive proteins into retinal cells, enabling eyes that have lost vision to respond to light once again.

Last month, the U.S. Food and Drug Administration (FDA) accepted a new drug application for Nanoscope Therapeutics’ optogenetic gene therapy ‘Mogene’. Approval is expected to be decided in the first half of next year. In phase 2b/3 clinical trials, 40% of patients showed medically meaningful improvement in vision 52 weeks after administration of Mogene.

Optogenetics is expected to show results in retinal diseases first, because the retina is neural tissue that directly receives light. While the brain requires invasive procedures like fiber-optic implants to deliver light, the eye allows genes to be delivered with a single injection and light targeted precisely to the intended cells. This approach also overcomes limitations of conventional gene therapies that are restricted to certain mutations by utilizing remaining retinal cells regardless of genetic causes. Kim Kihyun, professor of ophthalmology at Seoul St. Mary’s Hospital, explained, “It is possible to directly observe how the protein is expressed, which makes the process of verifying therapeutics easier.”

Experts predict that the use of optogenetics in drug development will further increase in the future. Kim Junki, professor at Ulsan University College of Medicine, said, “Neurological diseases are challenging to treat by targeting specific circuits because multiple brain regions are intertwined, but optogenetics is highly promising as a tool for finding new therapeutic targets in diverse diseases.”

There is also attention on whether Korean researchers will achieve new results in the field of optogenetics. Professor Deisseroth received the Basic Medicine Prize at the Asan Medical Awards in March last year, as selected by the Asan Foundation. Prior to this, he taught optogenetics technology in person to over 20 researchers in Korea and distributed research genetic clones for free to over 80 research institutions in the country, forging ties with Korea’s research community. It is said that Professor Deisseroth was nominated for this year’s Nobel Prize at the recommendation of Seo Pangil, former director of the Korea Brain Research Institute and operating committee member for the Asan Medical Awards. The Korean academic community recognized and engaged with the value of optogenetics well before the Nobel Prize was awarded.

This content was produced with the assistance of AI translation services.