Editas 基因编辑疗法 EDIT-401 获批首次人体试验,单次注射上调肝脏 LDL 受体
CRISPR vs. the world's #1 killer. Editas just got cleared for the first human trial of EDIT-401 it is a one-time gene edit that turns up your liver's LDL receptors instead of cutting a gene.
Editas 的基因编辑疗法 EDIT-401 获准开展首次人体试验,它通过单次注射上调肝脏 LDL 受体,而不是剪切基因。该疗法在猴子实验中使坏胆固醇下降 90%,10 个月后仍保持下降;首位患者将于今年年底前在澳大利亚接受给药,首批安全性数据预计在 2027 年初公布。
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数字来自平台,为最近一次抓取时的数据。
以下是视频口播的自动转写,可能有错字。
Your liver already knows how to fix your cholesterol. It just isn't doing it hard enough. A heart disease kills about 18 million people a year, more than anything else on earth, which is pretty wild to put in perspective.
Now, a company called editas just got cleared to go after it with one injection. So, your liver cells are covered in tiny hooks called ldl receptors.
They grab bad cholesterol out of your blood and pull it in to be destroyed. More hooks means less cholesterol floating around.
Now, a statin essentially nudges your liver to grow a few extra hooks. You take it every day for the rest of your life. And the day you stop, the extra hooks go away.
Editas packs a gene editor into essentially a fat bubble, injects it, and it rides your bloodstream straight to the liver.
From there, it leaves the gene intact, and turns up the volume on it. So your cells keep building hooks on their own. Once that edit is made, it stays made.
Now, this was tested in monkeys. Bad cholesterol dropped 90 percent. 10 months later, it was still down. Oh wait, so yes, that's monkeys.
No humans have had this yet. The first patient gets dosed in australia before the end of this year. And the first safety numbers will come in early 2027.
But let's focus on the version that works here. One appointment instead of a pill, you refill 500 times plus plus plus for the rest of your life.
I mean, if this works, i'd be curious who gets it first. How much is going to cost? Is it worth the trade-off of pills? It definitely is in the right direction.
Share this with someone who loves biotech.
来源:TikTok · Tiff in Tech · tiktok.com