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ASGCT
United States
Registrace 29. 09. 2011
Congressional Briefing: How Gene Therapies are Transforming the Hemophilia Landscape
On July 24, ASGCT hosted a virtual briefing, How Gene Therapies are Transforming the Hemophilia Landscape, for House and Senate staff. The briefing focused on the potential of gene therapies to change the hemophilia treatment paradigm and inform on real-world implications and considerations for gene therapy recipients.
During the briefing, experts provided a comprehensive overview of the gene therapy landscape for hemophilia, from basic mechanisms and delivery methods to patient experiences and clinical outcomes. The panel included: ASGCT Board members Matt Porteus, MD, PhD and Lindsey George, MD; ASGCT member Leonard Valentino, MD; and patient advocates and gene therapy recipients Bobby Wiseman, Jr. and Bill Maurits.
During the briefing, experts provided a comprehensive overview of the gene therapy landscape for hemophilia, from basic mechanisms and delivery methods to patient experiences and clinical outcomes. The panel included: ASGCT Board members Matt Porteus, MD, PhD and Lindsey George, MD; ASGCT member Leonard Valentino, MD; and patient advocates and gene therapy recipients Bobby Wiseman, Jr. and Bill Maurits.
zhlédnutí: 140
Video
Policy and Advocacy Shorts with Katy Spink, PhD
zhlédnutí 30Před 21 hodinou
Katy Spink, PhD, COO at Dark Horse Consulting, emphasizes ASGCT's unique focus on policy issues and its role in facilitating learning and collaboration among professionals in the cell and gene therapy field.
Policy and Advocacy Shorts with Ashley Hume
zhlédnutí 19Před 28 dny
Ashley Hume, President at Emerging Therapy Solutions, explains how ASGCT fosters collaboration between different stakeholders and emphasizes the importance of staying ahead of new therapies to ensure patient access.
Policy and Advocacy Shorts with Jennifer Wellman
zhlédnutí 13Před 28 dny
Jennifer Wellman, Chief Development Officer at Akouos, discusses ASGCT's role in addressing manufacturing and access challenges in gene therapy, and shares her excitement about the field's rapid progress.
Policy and Advocacy Shorts with Keith Wonnacott, PhD
zhlédnutí 10Před 28 dny
Keith Wonnacott, PhD, Vice President of Regulatory Affairs at Lexeo Therapeutics, shares insights on ASGCT's role in connecting academia and industry, and discusses the evolution of regulatory approaches in cell and gene therapy.
Policy and Advocacy Shorts with Diane Berry, PhD
zhlédnutí 14Před 28 dny
Diane Berry, PhD, Executive Vice President, Chief Global Policy & Advocacy Officer at Sarepta Therapeutics, highlights ASGCT's unique role in bringing together diverse stakeholders and providing evidence-based advocacy to advance policies in cell and gene therapy.
Policy and Advocacy Shorts with Terry Flotte, MD
zhlédnutí 19Před měsícem
Dr. Terry Flotte, Provost and Dean at UMass Chan Medical School, reflects on ASGCT's history and its crucial role in fostering collaboration between scientists, regulators, and policymakers to accelerate the development of gene therapies.
Policy and Advocacy Shorts with Jeff Chamberlain, PhD
zhlédnutí 9Před měsícem
ASGCT Past President Jeff Chamberlain, PhD, discusses the Society's role in bringing together diverse expertise, the importance of patient involvement, and his excitement about gene therapy becoming a routine part of medicine.
Policy and Advocacy Shorts with Federico Mingozzi, PhD
zhlédnutí 19Před měsícem
ASGCT Board Member, Federico Mingozzi, PhD, discusses the excitement of new clinical data in gene therapy, the challenges of attracting investors for rare diseases, and the importance of engaging with policymakers to advance the field.
Policy and Advocacy Shorts with Hans Peter Kiem, MD, PhD
zhlédnutí 12Před měsícem
Hans-Peter Kiem, MD, PhD, ASGCT Past President, discusses ASGCT's evolution, its role in fostering collaboration, and the exciting future of in vivo gene therapies.
Policy and Advocacy Shorts with Paula Cannon, PhD
zhlédnutí 7Před měsícem
ASGCT President, Paula Cannon, PhD, talks about the importance of having a space for patients, policymakers, and CGT professionals to interact - and how those interactions lead to real change and inspire hope.
Policy and Advocacy Shorts with Wilson Bryan, MD
zhlédnutí 30Před měsícem
Wilson Bryan, MD, former FDA regulator, explains the importance of ASGCT in bridging gaps between researchers, regulators, and policymakers, and shares his optimism about the future of cell and gene therapies.
CRISPR and Beyond with Vertex CSO David Altshuler, MD, PhD-ASGCT's The Issue
zhlédnutí 250Před měsícem
Join host Emily Walsh Martin, PhD, and David Altshuler, MD, PhD, Chief Scientific Officer of Vertex Pharmaceuticals. David came to Vertex after a lengthy career in medicine and academic research. David and Emily discuss how David and the team at Vertex think about innovation in drug development and how that impacts how they select programs and approaches for the diseases they focus on. They als...
Targeting STAT3 in Tumors with Oligo-PROTAC with Dr. Marcin Kortylewski-The Molecular Therapy Pod...
zhlédnutí 95Před měsícem
Join ASGCT and the Molecular Therapy family of journals for a conversation between Drs. Paloma Giangrande and Marcin Kortylewski. They discuss a recent article published in Molecular Therapy Nucleic Acids by Dr. Kortylewski and colleagues titled "Oligo-PROTAC strategy for cell-selective and targeted degradation of activated STAT3 (www.cell.com/molecular-therapy-family/nucleic-acids/fulltext/S21...
AAV Capsid Production with Drs. Mario Mietzsch and Robert McKenna - Molecular Therapy Podcast
zhlédnutí 203Před 2 měsíci
Dr. Daniel Stone speaks with Drs. Mario Mietzsch and Robert McKenna from the University of Florida to discuss a recent article published in the Biomanufacturing in Gene and Cell Therapy special issue of Molecular Therapy Methods & Clinical Development (www.sciencedirect.com/special-issue/10RZ8HRVPL2) by Drs. Mietzsch, McKenna, and colleagues titled Production and characterization of an AAV1-VP3...
Gene Insertion Tech with Jason Cole, CEO of SalioGen-ASGCT's The Issue
zhlédnutí 204Před 2 měsíci
Gene Insertion Tech with Jason Cole, CEO of SalioGen-ASGCT's The Issue
Meet Dr. Mohamed Abou-el-Enein, Editor-in-Chief of Molecular Therapy Methods & Clinical Developme...
zhlédnutí 42Před 2 měsíci
Meet Dr. Mohamed Abou-el-Enein, Editor-in-Chief of Molecular Therapy Methods & Clinical Developme...
Gene Therapy Targets and Biomarkers: Molecular Therapy Presents
zhlédnutí 478Před 3 měsíci
Gene Therapy Targets and Biomarkers: Molecular Therapy Presents
Increasing Equitable Access with Hayley Lam, PhD
zhlédnutí 18Před 2 měsíci
Increasing Equitable Access with Hayley Lam, PhD
An Explosive Year for Gene Therapy with ASGCT President Paula Cannon
zhlédnutí 35Před 2 měsíci
An Explosive Year for Gene Therapy with ASGCT President Paula Cannon
Reporting on Regulatory Considerations with Journalist Jef Akst
zhlédnutí 11Před 2 měsíci
Reporting on Regulatory Considerations with Journalist Jef Akst
CNS Gene Therapies with Todd Carter, PhD
zhlédnutí 24Před 2 měsíci
CNS Gene Therapies with Todd Carter, PhD
New Frontiers in Hemophilia Treatments with Lindsey George, MD
zhlédnutí 21Před 2 měsíci
New Frontiers in Hemophilia Treatments with Lindsey George, MD
Cystic Fibrosis Gene Therapies with CDA Winner Ashley Cooney, PhD
zhlédnutí 31Před 2 měsíci
Cystic Fibrosis Gene Therapies with CDA Winner Ashley Cooney, PhD
Patient Advocacy with Leah Myers + Jenny Burke
zhlédnutí 12Před 2 měsíci
Patient Advocacy with Leah Myers Jenny Burke
Stem Cell Therapy with Charles Murry, MD, PhD
zhlédnutí 37Před 2 měsíci
Stem Cell Therapy with Charles Murry, MD, PhD
Communicating the Science with Benjamin McLeod
zhlédnutí 6Před 2 měsíci
Communicating the Science with Benjamin McLeod
New Technologies for Gene Targeting + Gene Correction with David Segal, PhD
zhlédnutí 25Před 2 měsíci
New Technologies for Gene Targeting Gene Correction with David Segal, PhD
Welcome to the Annual Meeting with ASGCT President Jeff Chamberlain, PhD
zhlédnutí 125Před 3 měsíci
Welcome to the Annual Meeting with ASGCT President Jeff Chamberlain, PhD
Welcome to the Annual Meeting with ASGCT President Jeff Chamberlain, PhD
zhlédnutí 36Před měsícem
Welcome to the Annual Meeting with ASGCT President Jeff Chamberlain, PhD
Great explanation, thanks!
1 in 800 will have an adverse event. Responsable for unexpected deaths on a grand scale espessaly using the liponano particles. The long term studies are not completed and unknown. In itself leads to cansers of all kinds. This is a bunch of greedy buggers making money on eugenics, not helping anybody, just a profitable ideoligy that will never work as it in all the years never has not lead to a disaster, Sickning.
my baby brother has this. he's only 3. he hasn't left the hospital since he was born. it's been so rough.
So sad... Four year old video yet no update anywhere for treatment using gene editing!
Why do you say mRNA vaccines for Covid fit YOUR definition of "Gene Therapy?" Elaborate.
beautiful
Lecker Lungenbrötchen
Grüße an alle
dear sir or mam, if exon 45,46, and 47 missing how will it reframe it...plz give me suggestions...
It would be great if there was a video on how Excision-only PiggyBac Transposase Expression vector works
Dr Osade on CZcams has the best remedy for sickle cell ❤❤❤❤❤❤❤❤
MIMOSA-DI CAPSULES from Planet Ayurveda acts as coolant and useful in blood disorders
Any updates on this? I have MTM and would love some updates.
I wonder what is the latest update of this research. Thank you for this presentation!
Treating disease by changing the blueprint to our bodies at a microscopic level 🧬🩺🥼❤️ (Our Genetic Make-up)
Eat turd
They illicit immune response because it’s all toxic to the human body that is NOT immunity!
This helped me so much! I couldn't understand viral vectors but this video go me to understand.
شكرا كثيرا 🤍🤍
That's a great report. What's relevant to me is , is that if the gene therapy doesn't work , I can still infuse . Is there any food that that helps. I know personally that sny food that's a blood thinner doesn't help. Vitamin K green type smoothies are safe. It seems like 95 percent of things that are good for blood are risky for bleeders. Thank God for clotting factor. I infuse once a week to control hip both ankles and right knee target joints. Survived a brain aneurysm in 05 . My faith in Jesus got me through that . I'm carb free by about 90 percent of the time. I'm checking into this . This could be wonderful news fot us . And yes I agree that it's unimaginable what people world wide suffer with out clotting factor.
What caused your brain aneurysm? Is it cholesterol or hypertension? brain bleed is scary😢
They are using us like laboratory rats. The Covid mRNA genetic experiment, in addition to not working as it should, is causing numerous side effects. Don't believe advertisements with pretty designs because it's not that simple. Big Pharma's goal is money.
Wow it's very interesting
Any cure for tis disease? If anyone knows please please let ne know.thank you
how long do the EVs persist in the system ? how long can the "exosomes" be detected in the blood system ? therapeutic application dose would be at what numbers/level of "exosomes" ? the medical application requires a single dose ?, or do the doses need to be sequential and at what rate ? how many ? how does one measure the EVs in a 5ml glass vessel ?
This hypothesis is toxic! Mrna from any other species isn’t meant to go in the human body along with all these chemicals no wonder people were getting sick after they were injected with this toxic combination claiming it’s a vaccine when it’s actually causing all the diseases to escalate!
Beautiful presentation for beginners learning gene therapy regarding DMD
Funny - "The subtitling of these videos was funded in part by a donation from Pfizer, Inc." Good to know.
Super useful! Thanks for sharing!
🥰🥰 fell proud as a LGMD patient 🥰🥰
wooooow you are amazing
So about making me immortal. ...
I have pigment dispersion syndrome. I really need a cure.
😢
Exciting and well explained
Good elaborate
I know nothing about viral vectors nor any biology really. Just stumbled across the word viral vector and youtubed it. and I am both astonished and greatful for how much science help humanity, and the heroes that dedicate their life to science and research to bring us these amasing treatments.
Which species Messenger RNA is this from and what’s the danger of sick animals mRNA controlling human cells? Oh yes zoonotic disease & sickness!
I find a high protein diet along with light exercise really helps. Dont excercise till your muscles cramp or get sore...but exercise keeps the muscles strong. Watch your weight but dont get too thin ....more weight = more effort to move. Its a losing battle but try keep mobile and safe as long as you can
Gene therapy
It’s in the jab
Look, I feel sorry about those suffering from this but since this IS a genetically inherited disease then along with treatment should also come sterilization. Why create new generations of people with this? Adopt a healthy child instead.
THANK YOU SO MUCH
Immortality
Hi. Iam a 29 years old women and i havemyotubular myopathy, i hope for a cure. Do you have an e mail adress?i want to ask some questions or more information,
Poison.
Gene therapy is started any where in world?
Hi my elder brother is suffering from eb since 30 years there is no cure we tried everything literally everything my cousin was also butterfly baby but he was died in 13 years of his age
Combined with crisper technology we could use this to completely rewrite our entire biological systems
How and where in india take gene therapy fo hemophilia A
It's the same for suicide gene therapy!