Behind the glory of the Nobel Prize: Looking at the CRSPR from the rise of RNAi

Before the announcement of the Nobel Prize, the biomedical inventions that had the potential to win the awards were already being madly rendered by the media, but the blessings were based on the blessings. The transition from the invention of cutting-edge laboratories to real drugs is a long and tortuous process. Sometimes, the greater the expectations, the greater the disappointment.

Once, it was like sitting on a roller coaster, RNAi, as a Nobel Prize-winning technology, can be described as both fame and fortune, but there is no use of it. So far, scientific invention has not been translated into actual drugs, at least not fast enough for investors. This is very embarrassing.

After ten years of winning the Nobel Prize, RNAi once again stood tall: the first drug to silence the disease gene using RNAi technology will be approved by the federal government next year. However, such a slow progress has sounded the alarm for other over-promoted, widely acclaimed inventions, from invigorating immune vaccines to optogenetics to the CRISPR gene editing system. .

“I guarantee that God created RNAi not just for drug development,” said John Maraganore, CEO of Alaylam Pharmaceuticals, who conducts RNAi research in Cambridge. "We have solved it."

Behind the glory of the Nobel Prize: Looking at the CRSPR from the rise of RNAi

Exploration begins with locusts

The story of RNAi begins with the nematode class. In the early 20th century, some scholars studied how genes are expressed in small aphids and found that they can manipulate this process by interfering with the nematode genes by the RNA of the insects. Scientists Andrew Fire and Craig Mello published a major paper in Nature, showing that the aphid gene can be silenced.

This is of great significance to the medical profession. Many diseases are caused by malformed or ineffective protein conformations. Most traditional medicines aim to eliminate this protein from the body. In contrast, RNAi therapy can theoretically completely down-regulate this dysfunctional protein product—by silencing the corresponding coding gene. Excitingly, scientists began experimenting with the regulation of mammalian cells using Fire and Mello's aphid studies. In 2001, the code was finally cracked, and the use of biotechnology to turn RNAi into a drug "opened a maddening development."

Patients, researchers and, most importantly, investors, see it as the next major breakthrough in medical science. However, this baby said that she is still too happy~

“I think when most people don’t understand what they are really excited about, they don’t understand what the process will be like,” Dirk Haussecker, an independent analyst who followed the RNAi technology.

Nobel Prize

Mello is a researcher at the University of Massachusetts. Fire was a professor at Stanford University. They won the 2006 Nobel Prize. It was very rare to get this award in just eight years after they first published the results of the locust research.

Subsequently, this area began to break out. The VC has already given RNAi countless dollars. Now the investment of major pharmaceutical companies is flooding in the most famous – Merck has invested 1.1 billion knives to acquire Sirna Medical , which is based on RNAi research.

"It makes people excited because they guess that high prices mean that RNAi will really work long before," Haussecker said.

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