Gene therapy, a term that often appears in science fiction, is now entering people's lives.
There is a wave of gene therapy boom all over the world, and overseas gene therapy drugs are emerging one after another.
It is a pity that although China's gene therapy drugs came out on top at the time, due to internal reasons, they have been dormant for twenty years and are still absent from this important medical battlefield.
The idea of gene therapy actually appeared very early, as early as 1972. The idea at that time was to insert healthy genes into cells to replace disease-causing gene variants through DNA-based drugs.
The idea is beautiful, but it is not difficult to realize it. After decades of experimental failures, route exploration, and even the death of some patients, the journey has been extremely bumpy.
The world's first gene therapy drug, Jinyousheng, 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 money for subsequent clinical development and the introduction of capital, it fell into a patent battle between scientists and entrepreneurs, and long-term internal friction completely destroyed this first-in-class drug, and it is now a thing of the past.
In 2014, the second gene therapy drug was first launched in Europe to treat the rare genetic disease lipoprotein lipase deficiency. However, because there are so 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 example was sold after it was listed, and it was delisted 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.
After that, the pace of marketing of gene therapy drugs accelerated rapidly.
Gene therapy covers cell therapy, gene therapy and nucleic acid therapy. Today, more than 20 gene drugs are on the market around the world for the treatment of cancer, viral infections, and genetic diseases.
After nearly half a century, gene therapy has moved from concept to reality.
According to predictions, the global market size of gene therapy will increase 44 times from US$1 billion in 2017 to US$44 billion in the next two years.
Over the past few years, and in the coming years, gene therapy has become, and will continue to be, one of the most talked about areas of pharmaceuticals.
Countless patients will benefit from it. Faced with such a grand occasion, people can even stretch their imagination 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 in a direction that people cannot imagine.
"Bah bang bang", a burst of warm applause sounded.
One by one, employees stood up from their seats and clapped excitedly.
They had just seen the genomics achievements made by foreign research teams, and they were all excited and wished they could make such achievements themselves.
What is the biggest dream of every biology student?
Isn’t it just about unlocking the genetic code, uncovering the mystery of life, and thus changing the destiny of mankind?
And now, the entire world has taken a solid step towards this goal.
They must also ride on the wind, catch up with this wave, and soar and dance on the wind.
Everyone looked at Tang Que with expectant eyes.
He stood in the center, holding a DNA double helix structure model in his hand.
Amidst the applause, he had calmed down and looked at the crowd surrounding him.
"Okay, I have declared war. What are your thoughts and questions?"
Employees looked at each other, looking at each other.
They had been working under Tang Que for a while, but sometimes they still couldn't keep up with the leader's racing ideas.
Although this declaration sounds exciting, it seems that it is not up to them to decide whether they can talk about the details, and which direction to work on.
A gentle young man raised his hand: "Dr. Tang, I have a few questions. Do we already have a goal? Which rare disease should we start with?"
"Should we have a standard for selecting target diseases, such as starting from rare diseases with the most patients, or from other aspects?"
"The generic drug department is developing many drugs for rare diseases. Should we communicate more with them?"
Tang Que looked at him with admiration and nodded: "Jiang Kai, the question you raised is very good. It proves that you have your own thinking, which is very rare. 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 follow our friends across the river, imitate their treatment paths and delivery routes, and then make improvements. When everyone is familiar with it and can go ashore, we can start running freely."
"There are about 20 kinds of gene therapy drugs on the market now. Let's start with the most mature and expensive ones."
"Generic drugs have no technical content. They are all working on small molecule drugs. They are different from the biomacromolecule drugs we want to study, and the direction is completely different. They treat the symptoms, and we treat the root cause."
"You can communicate with them about other aspects besides R&D."
"Okay, everyone should understand now."
"I have another question to ask you," Tang Que put his raised fingers in front of his face and scanned the audience with his sharp eyes.
"Who can tell me what is the most expensive drug in the world today?"
The scene fell into silence for a moment.
After a few seconds, a short man wearing glasses looked around and timidly raised his hand: "I, I know."
Tang Que shook his head in disappointment, and his voice was extremely cold: "I asked a question. If you know it, just say it."
He suddenly shouted loudly: "When doing things here, you must be direct, do you understand?"
A sparse voice sounded: "Understood."
Tang Que's eyes widened: "Fart, the sound is so small. I can't hear it!"
The whole audience instantly shouted: "I understand!"
The little man with glasses also puffed up his chest and shouted loudly: "We understand!"
Tang Que clapped loudly: "Okay, this is enough energy, this is manliness, this is crazy."
"Now tell me, what is the most expensive drug in the world?"
He asked loudly again, this time using all his strength, and the deafening sound swept through the scene like a strong wind.
Everyone held their heads high and shouted: "zolgensma"
"Which company? What disease does it treat?"
"Novartis, spinal muscular atrophy sma."
Everyone's lungs were beating like bellows, spitting out these names loudly.
After they said it, they stared at Tang Que, waiting for him to continue.
Tang Que put his hand to his ear, fanned himself, and asked, "Where's the price? I didn't hear the price."
The little man with glasses was the first to blurt out, and each word shot out of his mouth like a machine gun.
"The selling price is as high as US$2.1 million, totaling about 14 million yuan."
Tang Que looked at him this time and nodded: "Very good. I'm making progress. Next time you know the answer, tell me directly and don't waste any words."
The little man with glasses swallowed nervously, nodded, pressed his lips tightly, and said no more.
The DNA molecule model in Tang Que's hand kept spinning and jumping as he continued talking.
"Spinal muscular atrophy (SMA) is a very rare neuromuscular genetic disease that affects only about 10,000 people worldwide. The therapy developed by Novartis uses an adeno-associated virus (AAV) to infect the correct human The smn1 gene is delivered into the patient's cells, thereby restoring the patient's ability to exercise."
"This is a true 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 obtained a clinical trial application from the Food and Drug Administration."
"Because it works so well, its market sales are growing, reaching $1.3 billion last year."
"But this is not the only drug to treat SMA disease. There are three SMA treatment drugs on the market globally, 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."
"You may still have some memories of last year's 700,000-yuan-a-shot drug incident. This drug is Bogen'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 lifelong medication through lumbar puncture. The price was too expensive before and the sales were not good. Later, after negotiation, it was included in the medical insurance catalog. The original 700,000 injections were reduced to 30,000. In this way, the patient's first-year treatment cost will be about 100,000, and it will cost another 50,000 per year for subsequent years. After reimbursement, it should be even lower."
"The last one is Roche's evrysdi. This is an oral drug that is also very expensive. It is priced at 60,000 per 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 yuan."
Tang Que suddenly looked serious, and the model in his hand stopped: "What I, Mr. Tang, dislike the most are the sky-high prices of drugs. If Bogen can lower their prices, why can't Roche and Novartis lower their prices?"
"The price is so ridiculously expensive, 14 million for one shot. It's simply outrageous to open the door to outrageous people. It's so outrageous."
"Isn't there a reason for monopoly in this? Pharmaceutical companies need profits, but they shouldn't be so outrageous."
"Of the 10,000 patients in the world, how many can afford this price? Are those who can't afford it have to watch themselves die?"
Tang Que shook his head and took a deep breath: "The development of 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, we have always had this glorious tradition."
"Our goal is to develop our own SMA gene therapy drugs, bring down these sky-high price drugs, restore their use value, and return them to the essence of drugs - treating diseases and saving lives."
"Only drugs that people can afford are good drugs and should be our goal."
“If no patient can afford a drug, or only a very small number of people can afford it, what’s the point of bringing it to this world?”
