Mathew Pletcher
@thepetridish.bsky.social
From Allyson Berent of FAST. “What you think we want may not be what we want. The risk we are willing to accept may not be the risk you think we are willing to take. Ask us” paraphrased
#GATC2025 finishes with the patient perspective. Emphasizing that this is a partnership. All considerations of risk needs to be contextualized by the patients needs. Transparency is a must.
November 11, 2025 at 9:47 PM
From Allyson Berent of FAST. “What you think we want may not be what we want. The risk we are willing to accept may not be the risk you think we are willing to take. Ask us” paraphrased
#GATC2025 finishes with the patient perspective. Emphasizing that this is a partnership. All considerations of risk needs to be contextualized by the patients needs. Transparency is a must.
November 11, 2025 at 9:32 PM
#GATC2025 finishes with the patient perspective. Emphasizing that this is a partnership. All considerations of risk needs to be contextualized by the patients needs. Transparency is a must.
Said that we are past Era 1 (treating Rare Disease) and need to focus on how to bring it the tech to larger more complex diseases. Very clear articulation of what the Rare Disease community has been seeing (as sad as it is). Rare Disease is not something they are focused on.
Really helpful presentation by Sylke Poehling from #Roche that nicely encapsulated the thinking by Big Pharma on #genetherapy. #GATC2025
November 11, 2025 at 7:58 PM
Said that we are past Era 1 (treating Rare Disease) and need to focus on how to bring it the tech to larger more complex diseases. Very clear articulation of what the Rare Disease community has been seeing (as sad as it is). Rare Disease is not something they are focused on.
Really helpful presentation by Sylke Poehling from #Roche that nicely encapsulated the thinking by Big Pharma on #genetherapy. #GATC2025
November 11, 2025 at 7:52 PM
Really helpful presentation by Sylke Poehling from #Roche that nicely encapsulated the thinking by Big Pharma on #genetherapy. #GATC2025
George Church reminds us that we went 7 months from diagnosis to cure for baby KJ. It can be done but scaling beyond this rare one offs still escapes us. #GATC2025
November 11, 2025 at 7:12 PM
George Church reminds us that we went 7 months from diagnosis to cure for baby KJ. It can be done but scaling beyond this rare one offs still escapes us. #GATC2025
To paraphrase Sonia, what we are truly up against in accessing treatments are arbitrary decisions by individual people. Patients should have the choice to access these treatments. #GATC2025
Put on partial clinical hold while asking for an additional monkey study despite having robust data in other species. And lacking the money for that monkey study, that partial hold becomes a permanent hold.
November 11, 2025 at 1:58 PM
To paraphrase Sonia, what we are truly up against in accessing treatments are arbitrary decisions by individual people. Patients should have the choice to access these treatments. #GATC2025
Put on partial clinical hold while asking for an additional monkey study despite having robust data in other species. And lacking the money for that monkey study, that partial hold becomes a permanent hold.
November 11, 2025 at 1:56 PM
Put on partial clinical hold while asking for an additional monkey study despite having robust data in other species. And lacking the money for that monkey study, that partial hold becomes a permanent hold.
Sonia Vallabh speaking to the heart of the patient experience. Often told there is nothing you can do while your genetic information is actively kept from you. #GATC2025
November 11, 2025 at 1:49 PM
Sonia Vallabh speaking to the heart of the patient experience. Often told there is nothing you can do while your genetic information is actively kept from you. #GATC2025
Genetic Agency - the concept that patients should be provided the option to access the genetic medicines that could make a difference in their lives - but how do we make it happen?
Eric Kelsic of Dyno Tx kicking off what should be an interesting day discussing the concept of genetic agency.
November 11, 2025 at 1:38 PM
Genetic Agency - the concept that patients should be provided the option to access the genetic medicines that could make a difference in their lives - but how do we make it happen?
Eric Kelsic of Dyno Tx kicking off what should be an interesting day discussing the concept of genetic agency.
November 11, 2025 at 1:37 PM
Eric Kelsic of Dyno Tx kicking off what should be an interesting day discussing the concept of genetic agency.
Reposted by Mathew Pletcher
Eric Topol interviews Jennifer Doudna on the bright future of genome editing therapy in curing disease (Apple Podcasts). There’s an FDA-approved CRISPR therapy for sickle cell disease out now with others to come. [youtube.com]
Jennifer Doudna: The Exciting Future of Genome Editing
The most significant life science breakthrough of our time is just getting started.
Subscribe for more Ground Truths: https://erictopol.substack.com/
www.youtube.com
April 16, 2024 at 5:06 PM
Eric Topol interviews Jennifer Doudna on the bright future of genome editing therapy in curing disease (Apple Podcasts). There’s an FDA-approved CRISPR therapy for sickle cell disease out now with others to come. [youtube.com]
@thepressbox.bsky.social While I have your attention…You talk a lot about the crumbling business model of traditional media. I would be interested in hearing about the new models that meet the moment and you think could provide a path forward.
October 8, 2025 at 9:18 AM
@thepressbox.bsky.social While I have your attention…You talk a lot about the crumbling business model of traditional media. I would be interested in hearing about the new models that meet the moment and you think could provide a path forward.
@thepressbox.bsky.social If you are putting together an evolutionary tree of Sports Center anchors, isn’t Bob Ley at the top? Representing the CNN of sports concept that started the channel.
October 8, 2025 at 9:13 AM
@thepressbox.bsky.social If you are putting together an evolutionary tree of Sports Center anchors, isn’t Bob Ley at the top? Representing the CNN of sports concept that started the channel.
I guess I missed the announcement of the new candy corn tariff.
September 30, 2025 at 11:07 PM
I guess I missed the announcement of the new candy corn tariff.
Reposted by Mathew Pletcher
Reposted by Mathew Pletcher
Great news!
Experimental gene therapy for Huntington’s markedly slowed disease progression in key trial www.statnews.com/2025/09/24/h... via @statnews.com
Experimental gene therapy for Huntington’s markedly slowed disease progression in key trial www.statnews.com/2025/09/24/h... via @statnews.com
Experimental gene therapy for Huntington’s markedly slowed disease progression in key trial
An experimental gene therapy slowed the progression of Huntington’s disease by 75% after three years — study results likely to support the first approval of a genetic treatment for the condition.
www.statnews.com
September 24, 2025 at 12:28 PM
Great news!
Experimental gene therapy for Huntington’s markedly slowed disease progression in key trial www.statnews.com/2025/09/24/h... via @statnews.com
Experimental gene therapy for Huntington’s markedly slowed disease progression in key trial www.statnews.com/2025/09/24/h... via @statnews.com
I, for one, will not repeat the sins of my parents and visit this agony on my own offspring.
It is irresponsible to allow your kid to believe the Pirates are a legitimate team purposefully trying to win. Like believing in the Tooth Fairy.
Pirates Under Fire For Directly Marketing Team To Children theonion.com/pirates...
August 18, 2025 at 4:37 PM
I, for one, will not repeat the sins of my parents and visit this agony on my own offspring.
It is irresponsible to allow your kid to believe the Pirates are a legitimate team purposefully trying to win. Like believing in the Tooth Fairy.
Pirates Under Fire For Directly Marketing Team To Children theonion.com/pirates...
August 18, 2025 at 3:36 PM
It is irresponsible to allow your kid to believe the Pirates are a legitimate team purposefully trying to win. Like believing in the Tooth Fairy.
Reposted by Mathew Pletcher
Pirates Under Fire For Directly Marketing Team To Children theonion.com/pirates...
August 18, 2025 at 3:00 PM
Pirates Under Fire For Directly Marketing Team To Children theonion.com/pirates...
Reposted by Mathew Pletcher
Re $SRPT, huge doses are used, for Elevidys approx 10exp16 genomes for 70kg+, 100x > # of cells in the human body, or an MOI of 100 for every cell the patient has, ie trying to force AAV to do something it's not really capable of: Is it really surprising if tox issues may result?
July 19, 2025 at 12:40 PM
Re $SRPT, huge doses are used, for Elevidys approx 10exp16 genomes for 70kg+, 100x > # of cells in the human body, or an MOI of 100 for every cell the patient has, ie trying to force AAV to do something it's not really capable of: Is it really surprising if tox issues may result?
“It’s unconscionable that it now will take even longer for the FDA to rule on this drug for our very small population with no other specific therapies,” Barth Syndrome Foundation executive director Emily Milligan said in a statement.
endpoints.news/fda-rejects-...
endpoints.news/fda-rejects-...
FDA rejects Stealth's rare disease drug, but offers potential for accelerated approval
After a slight delay, the FDA rejected Stealth BioTherapeutics' Barth syndrome drug application for elamipretide, but will consider a resubmission via the accelerated approval pathway.
endpoints.news
June 23, 2025 at 1:10 AM
“It’s unconscionable that it now will take even longer for the FDA to rule on this drug for our very small population with no other specific therapies,” Barth Syndrome Foundation executive director Emily Milligan said in a statement.
endpoints.news/fda-rejects-...
endpoints.news/fda-rejects-...
Noooooooo! I guess I get to take this year off from following football. Or is this a secret plan to finally bottom out the team so we can get a high draft pick and get a real QB?
June 5, 2025 at 8:31 PM
Noooooooo! I guess I get to take this year off from following football. Or is this a secret plan to finally bottom out the team so we can get a high draft pick and get a real QB?
Reposted by Mathew Pletcher
Reposted by Mathew Pletcher
wonderful to see so many companies celebrating pride month this year
June 2, 2025 at 2:13 PM
wonderful to see so many companies celebrating pride month this year