I can extract side effects

Gene therapy, a term that often appears in science fiction, is now entering people's lives.

The whole world is setting off an upsurge of gene therapy, and overseas gene therapy drugs are emerging one after another.

It's a pity that although Huaxia's gene therapy drugs won the first place back then, due to internal reasons, they have been silent for a full twenty years, and they are still absent from this important medical battlefield.

The idea of ​​gene therapy actually appeared very early. As early as 1972, there was this idea. At that time, the idea was to insert healthy genes into cells to replace disease-causing gene variants through DNA-based drugs.

The idea is very beautiful, but it is not ordinary difficult to realize it. After decades of experimental failures, route exploration, and even the death of some patients, the journey along the way has been extremely bumpy.

The world's first gene therapy drug "Jin Yousheng" was actually launched in China in 2003. It was an anti-tumor gene therapy drug, which was groundbreaking at the time.

However, due to the lack of funds for subsequent clinical development and the introduction of capital, it fell into a patent battle between scientists and entrepreneurs. The long-term internal friction completely destroyed this first-line drug, and it has been a thing of the past.

In 2014, the second gene therapy drug was launched in Europa for the treatment of the rare genetic disease lipoprotein lipase deficiency. However, because there are too few patients with this disease, the incidence rate is only one in a million, and the price is as high as 1 million US dollars, only one case was sold after listing, and it was withdrawn from the market four years later.

In 2017, the first in vivo gene therapy drug in the Eagle Country market was approved by the FDA, marking the official arrival of the era of gene therapy.

Since then, the pace of gene therapy drug listing has accelerated rapidly.

Gene therapy covers cell therapy, gene therapy, and nucleic acid therapy. Today, more than 20 gene drugs have been launched around the world for the treatment of cancer, viral infections, and genetic diseases.

After nearly half a century, gene therapy has gone from concept to reality.

According to forecasts, the global market size of gene therapy will increase from 1 billion US dollars in 2017 to 44 times in just a few years, reaching 44 billion in the next two years.

Gene therapy has been, and will continue to be, one of the most talked about areas of pharma over the past and the years to come.

Countless patients will benefit from it, and in the face of such a grand occasion, people can even stretch their imaginations further and imagine the real future.

The future that only appears in science fiction may be gradually being reproduced in reality, and it is flying towards a direction that people cannot imagine.

"Papa papa", a burst of warm applause rang out.

One by one, the employees stood up from their seats and applauded excitedly.

They just saw the genomics achievements made by foreign scientific research teams, and they were all excited, wishing they could make such achievements themselves.

What is the biggest dream in every biological child?

Isn't it just to unravel the genetic code and unravel the mystery of life, so as to change the destiny of mankind?

And now, the whole world has taken a solid step towards this goal.

They also want to ride the wind, catch up with this wave, and soar and dance on the wind.

Pieces of expectant eyes looked at Tang Que.

He stands in the center, holding a model of the DNA double helix.

Amid the applause, he had already calmed down and looked at the crowd gathered around him.

"Okay, I've declared war, what are your thoughts and questions?"

Employees, you look at me, I look at you, and look at each other.

They have been working under Tang Que for a while, but sometimes they still can't keep up with the leader's way of thinking.

Although this statement sounds exciting, but can you talk about the details, and it seems that they can't decide which direction to focus on.

A well-mannered young man raised his hand: "Dr. Tang, I have a few questions. Do we have a goal? Which rare disease should we start with?"

"Should we have a standard for selecting target diseases, such as starting with the rare disease with the most patients, or from other aspects?"

"The generic drug department is developing drugs for many rare diseases. Should we communicate with them more?"

Tang Que looked at him with admiration and nodded: "Jiang Kai, the question you asked is very good. It is very rare to prove that you have your own thinking. I appreciate you very much."

He raised a finger and said slowly: "I have already chosen the target."

"As for the selection criteria, it's very simple. We first cross the river by feeling our friends, imitating their treatment and drug delivery routes, and then making improvements. When everyone is familiar with it and can go ashore, we can start running freely."

"There are about 20 gene therapy drugs on the market now, and we will start with the most mature and expensive ones."

"There is no technical content in generic drugs, they are all engaged in small molecule drugs, which are different from the biomacromolecular drugs we want to study, and the direction is completely different. They treat the symptoms, but we treat the root cause."

"You can talk to them about other aspects besides R\u0026D."

"Okay, everyone should understand now."

"I have another question to ask you." Tang Que put his raised fingers in front of him, his sharp eyes swept across the audience.

"Who can tell me, what is the most expensive drug in the world today?"

The scene fell into silence for an instant.

After a few seconds, a small man wearing glasses looked around and raised his hand timidly: "I, I know."

Tang Que shook his head in disappointment, his voice was extremely cold: "I asked a question, if you know it, just say it."

He suddenly shouted loudly: "When you do things here, you have to be direct, do you understand?"

A sparse voice sounded: "Understood."

Tang Que stared: "Fart, the sound is so low. I can't hear it!"

The audience immediately shouted: "I understand!"

The little man with glasses also puffed out his chest and shouted, "We understand!"

Tang Que applauded loudly: "Okay, this is enough, this is man, this is crazy."

"Now tell me, what is the most expensive drug in the world?"

He asked loudly again, this time with all his strength, the deafening voice swept across the scene like a gust of wind.

Everyone held their heads high and yelled, "Zolgensma"

"Which company? What disease does it treat?"

"Novartis, Spinal Muscular Atrophy SMA."

Everyone's lungs were flapping like a bellows, and they spit out these names loudly.

After saying it, they stared at Tang Que, waiting for him to continue.

Tang Que put his hand next to his ear, fanned it, and asked, "Where's the price? I didn't hear the price."

The little man with glasses was the first to blurt out, each word shot out of his mouth like a machine gun.

"The price is as high as 2.1 million US dollars, a total of about 14 million yuan."

Tang Que looked at him this time and nodded his head: "Very good, there is improvement, next time you know the answer, just say it, don't waste any saliva."

The little man with glasses swallowed nervously, nodded, closed his lips tightly, and did not speak again.

The DNA molecular model in Tang Que's hands kept spinning and jumping, and he continued to talk incessantly.

"Spinal muscular atrophy (SMA) is a very rare neuromuscular genetic disease affecting only tens of thousands of people worldwide. The therapy developed by Novartis uses adeno-associated virus (AAV) to inject the correct human Delivery of the SMN1 gene into cells in the patient's body restores the patient's ability to exercise."

"This is a real gene therapy drug that can completely cure patients with just one injection."

"This drug is not currently on the market in China, but it has already obtained a clinical trial application from the Food and Drug Administration."

"Because it works so well, it's a growing market, reaching $1.3 billion last year."

"But this is not the only drug for the treatment of SMA. There are three SMA drugs on the market worldwide, and the other two are already on the market in China."

"The price is also very expensive, but it is cheaper than the Novartis one."

"Everyone may still have a little impression of last year's 700,000 injection drug incident. This drug is Biogen's antisense oligonucleotide drug Spiraza for the treatment of SMA."

"It has been on the market in China for more than two years. Patients need life-long medication through lumbar spine puncture. The price was too expensive before and it has not been sold well. Later, it was included in the medical insurance catalog after negotiation. The original 700,000 injections were reduced to 31,000. Acupuncture. In this way, the treatment cost for the patient is about 100,000 in the first year, and 50,000 per year in the subsequent years. After reimbursement, it should be lower.”

"The last one is Roche's Evrysdi, which is an oral drug. It is also very expensive. It is priced at 60,000 a bottle in China. It is not yet covered by medical insurance. The annual treatment cost for a child is about 40,000 to 50,000."

Tang Que's face suddenly turned serious, and the model in his hand stopped: "What I, Tang, don't like the most is the sky-high price of medicine. Biogen can lower the price, why can't Roche and Novartis lower the price?"

"The price is so outrageously expensive, 14 million yuan per injection, it's simply outrageous to open the door for outrageously, outrageously home."

"Is there no reason for monopoly? Pharmaceutical companies need profits, but they shouldn't be so outrageous."

"Of the 10,000 patients in the world, how many of them can afford this price? Do those who can't afford it have to watch themselves die?"

Tang Que shook his head and let out a deep breath: "The development of science and technology should make everyone better and better, instead of dying in pain while looking at the technological products in front of us."

"The more mature the technology, the more cost can be reduced. Now, let us be the cost killer. Made in China has always had this glorious tradition."

"Our goal is to develop our own SMA gene therapy drugs, knock down these high-priced drugs from high places, restore their use value, and let them return to the essence of drugs - curing diseases and saving lives."

"Only medicines that people can afford are good medicines and should be our goal."

"If no patients can afford a drug, or only a very small number of people can afford it, what's the point of it coming into this world?"

I wrote 6400 tonight. I really tried my best. I hope I can maintain this state in the future.

In fact, sometimes I have less, because in addition to work, I have to read some biological and pharmaceutical materials, so that the technical aspects can be written smoothly, and the plot will not be too outrageous. I am not a professional either, and my knowledge is limited. I have to read books to understand a lot of knowledge in order to write the logic behind drug production. After all, the male protagonist can't make that kind of low-level low-level logic error when he can't develop a drug. Another: This book is more like a scientific research paper on black technology. The hero does not just take out a black technology product and make money directly. Instead, he wants to decipher the theory and knowledge in it, so as to use it for himself. If you can't be self-reliant, how is this different from buying advanced technology from Europe and America, and how is it different from a comprador of that kind of technology? The male lead of a black-tech novel should have ambition and ideals, should he be a comprador of extraterrestrial technology if he has a system?