Aurora Therapeutics, a new startup co-founded by Nobel Prize-winning scientist Jennifer Doudna, is working on tailored gene-editing treatments for rare diseases. The company plans to use a novel FDA regulatory pathway that allows personalized treatments for rare and fatal diseases based on data from just a handful of patients.
The current process for approving new drugs requires testing in hundreds or thousands of patients, which can be difficult for rare diseases due to the small number of affected individuals. However, this new pathway, known as the "plausible mechanism pathway," provides an alternative route for these types of treatments to get regulatory approval.
Aurora is initially focusing on treating a metabolic disorder called phenylketonuria (PKU), which affects 13,500 people in the US and requires a highly restrictive low-protein diet. The company aims to develop multiple versions of a PKU therapy that address different mutations using base editing, a more precise form of Crispr.
The development of personalized gene-editing therapies is crucial for patients like baby KJ, who recently received treatment made just for him after just six months. The treatment likely saved his life and was approved using the new regulatory pathway.
Aurora's strategy involves swapping out the guide RNA to make several versions of a PKU therapy that address different mutations, allowing the company to use the same technology platform to treat many mutations with less regulatory red tape.
The Innovative Genomics Institute will continue to create bespoke gene-editing therapies for children with very rare diseases. 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.
Industry expert Fyodor Urnov believes that Crispr technology is finally maturing, and he predicts that we will see other children with their personalized editors within three to four years.
The current process for approving new drugs requires testing in hundreds or thousands of patients, which can be difficult for rare diseases due to the small number of affected individuals. However, this new pathway, known as the "plausible mechanism pathway," provides an alternative route for these types of treatments to get regulatory approval.
Aurora is initially focusing on treating a metabolic disorder called phenylketonuria (PKU), which affects 13,500 people in the US and requires a highly restrictive low-protein diet. The company aims to develop multiple versions of a PKU therapy that address different mutations using base editing, a more precise form of Crispr.
The development of personalized gene-editing therapies is crucial for patients like baby KJ, who recently received treatment made just for him after just six months. The treatment likely saved his life and was approved using the new regulatory pathway.
Aurora's strategy involves swapping out the guide RNA to make several versions of a PKU therapy that address different mutations, allowing the company to use the same technology platform to treat many mutations with less regulatory red tape.
The Innovative Genomics Institute will continue to create bespoke gene-editing therapies for children with very rare diseases. 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.
Industry expert Fyodor Urnov believes that Crispr technology is finally maturing, and he predicts that we will see other children with their personalized editors within three to four years.
. It's crazy how much more efficient it'll be compared to traditional methods. Just think about it - instead of testing a treatment on hundreds of people, you can create multiple versions tailored to specific mutations in just a handful! 
That baby KJ's story is insane, it's like they took a leap of faith and made it work 
. And with Crispr technology getting more mature, I'm expecting to see even more personalized treatments for kids with rare disorders within the next few years 
. It's like we're finally getting close to that sci-fi movie future where medicine is so precise it's like having your own superpower 
!
This new gene-editing pathway for rare diseases is lit 
. No more waiting for hundreds of patients to be tested – just a few and they're good to go! 
. Aurora's strategy is genius 
, making multiple versions of the therapy with less regulatory hassle 
. Can't wait to see these treatments become a reality for kids like baby KJ in no time 
.
i mean think about it... they're talkin bout makin therapies 4 rare diseases & not just 4 people but 1 patient at a time 
its like wow! my lil bro has PKU & his doc says hes one of the lucky ones cuz he can take meds on his own 
but what if ther was therapy 4 every single kid w/ that disease? thats the kinda thing u get excited bout 
i no its still in trial phase but omg the possibiliteez 
. So, Aurora Therapeutics is working on gene-editing treatments for rare diseases and I guess that's kinda cool... but what about the science? Like, how does it work exactly? Can someone break it down in a way that doesn't put me to sleep 
. And another thing, 13,500 people in the US affected by PKU? That's like, what's going on here? How many of those people are actually getting treated and how's the success rate? I need some concrete info, not just feel-good stories about baby KJ 
.
. We're already seeing some incredible advancements in CRISPR tech, and it's only going to get more exciting from here. Bring on those bespoke therapies! 
. I hope this tech keeps gettin better & more affordable 4 everyone 
! And I'm so glad to hear that baby KJ got treatment made just for him - it's crazy to think about how that could've been life-saving 
Fyodor Urnov's prediction of personalized gene editors being available in 3-4 years is SO reasonable - this tech is finally maturing and it's gonna change lives 
This new FDA pathway is like a game-changer for rare diseases! I mean, can you imagine not having to wait years for a treatment just because there's not enough people affected? It's crazy how much power they're giving Aurora Therapeutics with this "plausible mechanism pathway". 
Baby KJ's story gives me chills, I'm so stoked that this tech might've literally saved his life! 
. I mean, about time right? Like, who needs a bunch of random people in clinical trials when we have this genius stuff 
. And yeah, it's super cool that they're using the "plausible mechanism pathway" thingy to get around all those boring regulatory requirements. Sounds like a total game-changer for families dealing with these super rare and deadly diseases 
.
. Baby KJ's story is just the icing on the cake – poor dude almost didn't make it and now he gets his very own customized treatment 
. It's like having a supercomputer analyze the patient's DNA and create a personalized treatment plan in seconds 
.
. Fyodor Urnov is spot on with his prediction that we'll see more of these custom gene-editing therapies in the next 3-4 years 
its gonna save so many lives!! baby kj's story is literally heartwarming 
and im so hyped for the trial at chp and penn medicine 
