Crispr Pioneer Launches Startup to Make Tailored Gene-Editing Treatments

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Gene Editing Pioneer Leverages Crispr to Create Customized Treatments for Rare Diseases, Citing Breakthroughs in FDA Regulatory Pathway.

A new startup called Aurora Therapeutics, co-founded by Nobel laureate Jennifer Doudna, is pushing the boundaries of gene editing technology by developing personalized treatments for rare and fatal diseases. The company aims to capitalize on a new regulatory pathway announced by the US Food and Drug Administration (FDA) that allows for the approval of bespoke therapies for rare conditions based on data from a small number of patients.

This shift marks a significant change in the way drug trials are conducted, particularly for rare diseases where recruiting enough patients is a major challenge. According to FDA officials Marty Makary and Vinay Prasad, once a manufacturer demonstrates success with several consecutive patients with different bespoke therapies, the agency will consider granting marketing authorization for the product.

Aurora's initial focus will be on treating phenylketonuria (PKU), a metabolic disorder that requires a highly restrictive low-protein diet to prevent toxic levels of phenylalanine in the blood. Without early treatment and monitoring, PKU can hinder brain development and impair cognitive functions. The company plans to use base editing, a more precise form of Crispr technology, and will have a standardized process to streamline the design and manufacturing of its therapies.

Aurora's strategy involves swapping out guide RNAs to make several versions of a PKU therapy that address different mutations. This approach allows for the treatment of multiple mutations with less regulatory red tape compared to traditional drug development processes.

According to Fyodor Urnov, Aurora's co-founder and a genome editing scientist at UC Berkeley, the company is committed to making treatments available to all patients regardless of the mutation causing their condition. "We are very much about no mutation left behind," he says.

The Innovative Genomics Institute will continue to create bespoke gene-editing therapies for children with rare diseases, while a trial at the Children's Hospital of Philadelphia and Penn Medicine will test the same type of gene editor used in baby KJ's therapy in a group of similar disorders.
 
🤯 I'm low-key obsessed with this breakthrough! Did you know that 1 in 5 medicines are being reformulated to treat rare genetic disorders? 📊 The FDA's new regulatory pathway is gonna revolutionize the way we develop personalized treatments for these conditions! 💡 According to the CDC, there are over 7,000 known rare genetic disorders, and Aurora Therapeutics is tackling just one of them: PKU. 🤝

The stats on gene editing technology are insane! 📈 Crispr-Cas9 has already been approved for cancer treatment in China 🇨🇳, and now we're seeing its potential in treating rare diseases. 🚀 The base editing approach used by Aurora is way more precise than traditional CRISPR tech - it's like finding a needle in a haystack! 💻

Aurora's goal to "no mutation left behind" is music to my ears! 🎶 With their standardized process and commitment to making treatments available to all patients, I'm hopeful that we'll see some major breakthroughs in the fight against rare diseases. 👍
 
Just heard about this Aurora Therapeutics startup & I'm low-key hyped 🤩! Gene editing pioneer Jennifer Doudna is leading the charge & it's amazing to see her team creating customized treatments for rare diseases like PKU 💊. The fact that they're using base editing, a more precise form of Crispr tech, means they can design multiple therapies targeting different mutations 🧬. This approach could make a huge difference in the lives of kids with these conditions & I'm all about it 👏!
 
idk about this whole bespoke therapy thing... sounds like they're trying to game the system 🤔. I mean, a small number of patients is basically just enough data for the FDA to consider it a 'breakthrough'? that doesn't seem super rigorous to me. and what's up with all these different mutations? can't we just standardize something here? 🚫
 
I'm loving this new development 🤯! It's like the FDA is finally catching up with the private sector on innovation, right? I mean, who needs regulatory red tape when you can make progress for rare diseases that are literally killing people 🙅‍♂️? It's all about streamlining the process and letting scientists do their thing.

I'm also excited to see personalized medicine become a reality. No more one-size-fits-all treatments; now it's all about tailored therapies based on individual mutations 🧬💡. The fact that companies like Aurora are committed to making these treatments accessible to all patients, regardless of the mutation, is just awesome ❤️.

Of course, this also raises questions about equity and access. How will these new therapies be priced and funded? Will it be a public-private partnership or will governments need to step in? These are the kinds of debates we should be having 🤔💸.
 
I'm loving this new development in gene editing tech! It's like they're finally making progress on those super rare diseases that have been plaguing people for ages 🤯. I mean, think about it - with PKU, just a small protein adjustment can make all the difference. And the fact that they're using base editing and swapping out guide RNAs? That's some fancy stuff right there 💡.

I'm also super impressed by their approach to making treatments available to everyone, regardless of mutation 🌈. It's not about leaving anyone behind, it's about inclusivity! And with the FDA's new regulatory pathway, it looks like we're finally moving in the right direction 🚀. The thought of multiple mutations being tackled with less red tape? That's a game-changer for me 💥.

I just hope we see this tech become more accessible to everyone, not just those who can afford it 🤞. We need to make sure that innovation doesn't come at the cost of equality 💕.
 
I'm freaking out thinking about this! A personalized treatment for PKU? It's like they're playing with fire here, but also maybe saving lives? I mean, how many people are stuck on that super restrictive diet just because it's the only way to not poison themselves? And what about all those kids who can't get enough of that therapy because their mutation is a bit too weird for the current technology? "No mutation left behind" sounds like a beautiful slogan but is it really gonna happen?! I'm hyped they're pushing boundaries and trying new things, but also kinda scared we might not be ready for this level of precision...
 
💡 just read about aurora therapeutics and i'm blown away by their approach 🤯 they're using crisper tech to create customized treatments for rare diseases like pku 💊 and it's crazy how much faster they can develop therapies than traditional methods 🚀 with base editing, they can swap out guide rnas to make different versions of the therapy that address different mutations 🔧 it's gonna be a game changer for kids with pku and other rare conditions 👶 what i love about this is that they're committed to making treatments available to all patients regardless of their mutation 💖 and the fda regulatory pathway changes are a huge step forward 🚀 did you know that for every 1,000 people affected by a rare disease, there's only around 2-5 clinical trials? 📊 so this new approach is gonna make a huge difference in getting treatments to those who need it most 💕
 
I'm low-key hyped about this 🤩 Aurora Therapeutics is on it! With Crispr tech, they're creating customized treatments for rare diseases like PKU and I think this is the future of medicine 🌟. No more one-size-fits-all approach, just tailored therapies that actually work 💡. It's crazy how FDA's new pathway is gonna change the game, especially for rare diseases where patients are super hard to find. This is all about giving people with these conditions a fighting chance 🥊 and I'm here for it! The fact that they're using base editing is also a major win 🔒. Less regulatory red tape means more options for patients and that's what it's all about 🌈
 
🤯 I'm loving this new development! It's finally happening – we're talking about personalized medicine that actually works for real people, not just rich folks with deep pockets. PKU is such a harsh disease, and the fact that Aurora's working on base editing tech is gonna make a huge difference. They need to hurry up though, I've got an aunt who's struggling with it and she needs this ASAP 💔! And I'm loving the "no mutation left behind" vibe – it's about time we saw some real progress in gene editing. The whole FDA regulatory pathway thing is a game-changer too. Can't wait to see what other rare diseases Aurora takes on next 🚀
 
I think this is super cool 💡! Scientists like Jennifer Doudna are literally changing lives with this new gene editing tech 🧬💥. I mean, can you imagine having a treatment that's tailored just to YOU because of your specific mutation? It's mind-blowing 🤯. And the best part is that they're not leaving anyone behind – no matter what mutation they have, there's gonna be a treatment 🌈👍. The FDA's new pathway is a huge step forward and I hope it makes a difference in people's lives ASAP 💨. We need more innovative thinkers like Jennifer Doudna in the world of medicine!
 
This is massive 🤯! Finally, we're seeing some real change in how they're tackling these super rare diseases 💉 Aurora Therapeutics is on it, leveraging Crispr tech to create customized treatments and the FDA is stoked about it 🎉 I mean, who wouldn't want a treatment that's tailored just for them? It's like, no more one-size-fits-all approach anymore 👋 This breakthrough in gene editing could be a total game-changer, especially with base editing being way more precise than traditional Crispr 💻 And the fact that they're making these treatments available to all patients regardless of their mutation is just 🙌
 
🤔 this is a big deal i guess... finally someone doing something about those super rare diseases that are literally killing people. and it's not just about throwing money at the problem either, they're actually trying to make treatments available to everyone no matter what mutation they have. base editing is the key here, sounds like a game changer. but we'll see how this all plays out in the long run 🚀
 
omg i just started learning about Crispr 🤯 and it sounds like such cool tech but also kinda scary what if it goes wrong? 😅 anyway so this Aurora Therapeutics thing is trying to use it to help people with super rare diseases that no one else can treat... like PKU or whatever. isn't PKU a big deal where they have to eat like super special food all the time? 🍴 and what's up with the FDA making new rules for gene editing therapy? did we just get approved for some kind of fancy medicine or something? 💊
 
I'm really excited about this new development 🤩! Gene editing is going to change lives, especially for kids struggling with rare diseases like PKU. I mean, can you imagine having a treatment that can fix the specific mutation causing your child's condition? It gives me hope that we'll see more breakthroughs in treatments for these super-rare conditions 🙏. As a parent, it's been heartbreaking to watch my kid deal with health issues, and this technology could be a game-changer 💊. I'm all for innovation, but what really gets me is the emphasis on making treatment available to all patients, regardless of their mutation 🌈. It's about time we see some real progress in personalized medicine! The future looks bright, folks 😊
 
🤓 I gotta say, this Aurora Therapeutics startup is looking up! They're really pushing the boundaries with their customised treatments for rare diseases using Crispr tech 🧬. I mean, think about it, no more one-size-fits-all approach when it comes to treating genetic disorders 😊. With base editing and guide RNAs, they can create multiple versions of a therapy that target different mutations. That's like having a bespoke suit for every single person with PKU 👔.

And the fact that they're committed to making treatments available to all patients regardless of mutation is just amazing 💕. It's about no mutation left behind, right? 🤝 Fyodor Urnov's quote at the end sums it up nicely. I'm excited to see how this plays out and what kind of breakthroughs we can expect from Aurora Therapeutics 👀.
 
OMG u guys its like finally a light at the end of the tunnel 4 ppl suffering from rare diseases their gonna get custom treatments based on Crispr gene editing tech thats so cool! Aurora Therapeutics is leading the way & their goal is to make sure no mutation gets left behind they r using base editing which is more precise than regular Crispr tech its like a game changer for people with PKU who cant even eat regular food cuz of phenylalanine levels

im all about this innovation & hope it leads to more breakthroughs in the future
 
😱 this is amazing news 💥 imagine being able to fix the genetic mutation that causes PKU for someone like KJ who was born with it 🤝 and can finally live a normal life without having to follow such strict diet restrictions 😩

but at the same time its also kinda worrying how much control these companies have over our bodies 💉 and what happens when they say no mutation left behind 🤯 does that mean they'll be creating treatments for every possible genetic variation ? it seems like we're playing a huge game of genetic whack-a-mole 🎮 where companies keep finding new mutations to treat but never really addressing the root cause of these diseases 💔
 
I'm totally against this whole thing 🙅‍♂️. Gene editing is being pushed as a miracle cure, but what about all the unknown long-term effects? We're basically playing with fire here 💧. What if these treatments end up causing more harm than good? And what's to stop big pharma from exploiting this tech for profit and abandoning patients once they've made their money 💸? Not to mention the ethics of editing genes in human embryos – it's like messing with nature 🌿. I'm all about pushing boundaries, but let's not get ahead of ourselves here ⏰. We need to be more cautious and do our due diligence before we start tinkering with human DNA 🔬.
 
I'm so stoked that gene editing is finally being used to create customized treatments for super rare diseases 🤩... but wait, isn't this just playing god? I mean, we're already messing with the fundamental building blocks of life, can't we just stick to what works and not risk causing more harm than good? But on the flip side, think about all those kids who are struggling to breathe or walk because they don't have access to proper treatment... this could be a game-changer for them 💡. Or maybe it's just a bunch of hooey and we're gonna end up with another Big Pharma scam? Ugh, my brain hurts trying to wrap itself around all this 🤯.
 
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