THANK YOU for making #ASGCT2025 a success! Access sessions on demand until July 18. We can't wait to see you May 12-16, 2026 in #Boston! #ASGCT2025 #CGT #ASGCTadvocacy
May 17, 2025 at 8:42 PM
THANK YOU for making #ASGCT2025 a success! Access sessions on demand until July 18. We can't wait to see you May 12-16, 2026 in #Boston! #ASGCT2025 #CGT #ASGCTadvocacy
Hello from #ASGCT2025! Visit our team at booth 1315 in the Exhibit Hall.
May 14, 2025 at 8:25 PM
Hello from #ASGCT2025! Visit our team at booth 1315 in the Exhibit Hall.
Incredible story that we learned about today at #ASGCT2025!
CHOP & @pennmedicine.bsky.social have delivered the first-ever personalized gene editing therapy for a patient with CPS1 deficiency, marking a major milestone in the application of CRISPR-based treatments.
Learn more about KJ & the future of personalized medicine: www.chop.edu/news/worlds-....
Learn more about KJ & the future of personalized medicine: www.chop.edu/news/worlds-....
May 15, 2025 at 8:43 PM
Incredible story that we learned about today at #ASGCT2025!
I was excited to attend and speak at #ASGCT2025 @asgct.bsky.social, but then incredibly disappointed to be told I can’t bring my 3 month old daughter. Being an early career scientist and mother is challenging enough without additional barriers #WomenInSTEM #AcademicTwitter #momademia
May 14, 2025 at 6:07 PM
I was excited to attend and speak at #ASGCT2025 @asgct.bsky.social, but then incredibly disappointed to be told I can’t bring my 3 month old daughter. Being an early career scientist and mother is challenging enough without additional barriers #WomenInSTEM #AcademicTwitter #momademia
A lipid nanoparticle–delivered base-editing therapy was custom designed for an infant with a ureacycle disorder. The affected infant was treated at approximately 7 and 8 months of age. Read the Brief Report: nej.md/42XF03F
#ASGCT2025 #MedSky @asgct.bsky.social
#ASGCT2025 #MedSky @asgct.bsky.social
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | NEJM
Base editors can correct disease-causing genetic variants. After a neonate had received
a diagnosis of severe carbamoyl-phosphate synthetase 1 deficiency, a disease with
an estimated 50% mortality ...
nej.md
May 15, 2025 at 5:06 PM
A lipid nanoparticle–delivered base-editing therapy was custom designed for an infant with a ureacycle disorder. The affected infant was treated at approximately 7 and 8 months of age. Read the Brief Report: nej.md/42XF03F
#ASGCT2025 #MedSky @asgct.bsky.social
#ASGCT2025 #MedSky @asgct.bsky.social
May 16, 2025 at 4:48 AM
A vaccine that generates antibodies that bind to cocaine and prevent it entering the brain has shown promise in an initial trial in ten addicts 🧪 #ASGCT2025
www.newscientist.com/article/2480...
www.newscientist.com/article/2480...
Vaccine may treat cocaine addiction by blocking drug's entry to brain
A vaccine that helps people overcome cocaine addiction has shown signs of being safe and effective in a small trial
www.newscientist.com
May 21, 2025 at 9:43 AM
A vaccine that generates antibodies that bind to cocaine and prevent it entering the brain has shown promise in an initial trial in ten addicts 🧪 #ASGCT2025
www.newscientist.com/article/2480...
www.newscientist.com/article/2480...
The Founders, Mendell, & Catalyst Award Symposium starts at 3:45 PM in Hall F! Celebrate CGT pioneers and their game-changing impact. #ASGCT2025
May 13, 2025 at 6:30 PM
The Founders, Mendell, & Catalyst Award Symposium starts at 3:45 PM in Hall F! Celebrate CGT pioneers and their game-changing impact. #ASGCT2025
Excellent story from @faylinphd.bsky.social on the hot news from #ASGCT2025 — @kiranmusunuru.bsky.social and colleagues tailor the world’s first bespoke gene editor to treat KJ, an infant with an ultra-rare genetic disease, heralding a new era in precision med.
www.genengnews.com/topics/genom...
www.genengnews.com/topics/genom...
May 15, 2025 at 8:06 PM
Excellent story from @faylinphd.bsky.social on the hot news from #ASGCT2025 — @kiranmusunuru.bsky.social and colleagues tailor the world’s first bespoke gene editor to treat KJ, an infant with an ultra-rare genetic disease, heralding a new era in precision med.
www.genengnews.com/topics/genom...
www.genengnews.com/topics/genom...
A gene editing drug custom-made for a critically ill baby in just a few months appeared to be safe & working as intended, according to newly published results in @nejm.org. Longer follow-up is needed to assess durability but read more about this in the story below: #ASGCT2025 #medsky #biosky #CRISPR
New: A #CRISPR gene editing drug custom-made for a critically ill baby in just a few months appeared to be safe and working as intended, according to results newly published in the New England Journal of Medicine. Details below:
#biotech #biosky #ASGCT2025
www.biopharmadive.com/news/crispr-...
#biotech #biosky #ASGCT2025
www.biopharmadive.com/news/crispr-...
A bespoke CRISPR therapy suggests a blueprint for treating ‘N-of-1’ diseases
A gene editing drug custom-made for a critically ill baby showed that, for some ultra-rare diseases, it’s possible to design and test a new CRISPR medicine in just a few months.
www.biopharmadive.com
May 15, 2025 at 8:03 PM
A gene editing drug custom-made for a critically ill baby in just a few months appeared to be safe & working as intended, according to newly published results in @nejm.org. Longer follow-up is needed to assess durability but read more about this in the story below: #ASGCT2025 #medsky #biosky #CRISPR
Do you have late-breaking data that is:
• high impact
• groundbreaking
• innovative
and
• newsworthy?
Send us your work through March 14 for the opportunity to present it at #ASGCT2025! #genetherapy #celltherapy annualmeeting.asgct.org/abstracts/la...
• high impact
• groundbreaking
• innovative
and
• newsworthy?
Send us your work through March 14 for the opportunity to present it at #ASGCT2025! #genetherapy #celltherapy annualmeeting.asgct.org/abstracts/la...
February 26, 2025 at 9:23 PM
Do you have late-breaking data that is:
• high impact
• groundbreaking
• innovative
and
• newsworthy?
Send us your work through March 14 for the opportunity to present it at #ASGCT2025! #genetherapy #celltherapy annualmeeting.asgct.org/abstracts/la...
• high impact
• groundbreaking
• innovative
and
• newsworthy?
Send us your work through March 14 for the opportunity to present it at #ASGCT2025! #genetherapy #celltherapy annualmeeting.asgct.org/abstracts/la...
Did you know that your high-impact #genetherapy + #celltherapy research could win you an award?
When you submit an abstract for #ASGCT2025, you may be eligible for a monetary award or a presentation opportunity. Send us your work through Jan. 31! bit.ly/3Oqtw0u
When you submit an abstract for #ASGCT2025, you may be eligible for a monetary award or a presentation opportunity. Send us your work through Jan. 31! bit.ly/3Oqtw0u
November 27, 2024 at 4:29 PM
Did you know that your high-impact #genetherapy + #celltherapy research could win you an award?
When you submit an abstract for #ASGCT2025, you may be eligible for a monetary award or a presentation opportunity. Send us your work through Jan. 31! bit.ly/3Oqtw0u
When you submit an abstract for #ASGCT2025, you may be eligible for a monetary award or a presentation opportunity. Send us your work through Jan. 31! bit.ly/3Oqtw0u
In a new editorial, Andrea L. Gropman, MD, and Alexis C. Komor, PhD, describe the foundations of an N-of-1 gene-editing study to treat an infant with a urea-cycle disorder. Read the editorial: nej.md/430hLWR
#ASGCT2025 @asgct.bsky.social #ScienceSky
#ASGCT2025 @asgct.bsky.social #ScienceSky
Personalized Gene Editing to Treat an Inborn Error of Metabolism | NEJM
In this editorial, the authors describe the foundations of an N-of-1 gene-editing study to treat an infant with a urea-cycle disorder.
nej.md
May 15, 2025 at 5:08 PM
In a new editorial, Andrea L. Gropman, MD, and Alexis C. Komor, PhD, describe the foundations of an N-of-1 gene-editing study to treat an infant with a urea-cycle disorder. Read the editorial: nej.md/430hLWR
#ASGCT2025 @asgct.bsky.social #ScienceSky
#ASGCT2025 @asgct.bsky.social #ScienceSky
@laurensarko.bsky.social gave a great talk at #ASGCT2025:
CRISPR-engineered CAR T cells targeting senescence-associated uPAR
Clear, compelling science—and a great discussion that followed. Exciting to see where this work is headed.
#CRISPR #CellTherapy
@warfnews.bsky.social
CRISPR-engineered CAR T cells targeting senescence-associated uPAR
Clear, compelling science—and a great discussion that followed. Exciting to see where this work is headed.
#CRISPR #CellTherapy
@warfnews.bsky.social
May 16, 2025 at 5:32 PM
@laurensarko.bsky.social gave a great talk at #ASGCT2025:
CRISPR-engineered CAR T cells targeting senescence-associated uPAR
Clear, compelling science—and a great discussion that followed. Exciting to see where this work is headed.
#CRISPR #CellTherapy
@warfnews.bsky.social
CRISPR-engineered CAR T cells targeting senescence-associated uPAR
Clear, compelling science—and a great discussion that followed. Exciting to see where this work is headed.
#CRISPR #CellTherapy
@warfnews.bsky.social
📢 Congratulations to these five new members of the Board of Directors! On June 1, we'll welcome to the Board:
- Barry Byrne, MD, PhD
- Marcela Maus, MD, PhD
- Laura Sepp-Lorenzino, PhD
- Steven Gray, PhD
- Allison Bradbury, PhD
Join them next month at #ASGCT2025. www.asgct.org/publications...
- Barry Byrne, MD, PhD
- Marcela Maus, MD, PhD
- Laura Sepp-Lorenzino, PhD
- Steven Gray, PhD
- Allison Bradbury, PhD
Join them next month at #ASGCT2025. www.asgct.org/publications...
Congratulations to Our New Board Members!
The incoming members of the Board of Directors will assume their roles on June 1, 2025.
www.asgct.org
April 18, 2025 at 4:05 PM
📢 Congratulations to these five new members of the Board of Directors! On June 1, we'll welcome to the Board:
- Barry Byrne, MD, PhD
- Marcela Maus, MD, PhD
- Laura Sepp-Lorenzino, PhD
- Steven Gray, PhD
- Allison Bradbury, PhD
Join them next month at #ASGCT2025. www.asgct.org/publications...
- Barry Byrne, MD, PhD
- Marcela Maus, MD, PhD
- Laura Sepp-Lorenzino, PhD
- Steven Gray, PhD
- Allison Bradbury, PhD
Join them next month at #ASGCT2025. www.asgct.org/publications...
PacBio is gearing up for #ASGCT2025 in New Orleans!
We’re excited to be part of the leading event for gene and cell therapy experts. Discover how HiFi sequencing is unlocking new possibilities in research and accelerating breakthroughs across the field.
#PacBio #ASGCT
We’re excited to be part of the leading event for gene and cell therapy experts. Discover how HiFi sequencing is unlocking new possibilities in research and accelerating breakthroughs across the field.
#PacBio #ASGCT
May 9, 2025 at 3:44 PM
PacBio is gearing up for #ASGCT2025 in New Orleans!
We’re excited to be part of the leading event for gene and cell therapy experts. Discover how HiFi sequencing is unlocking new possibilities in research and accelerating breakthroughs across the field.
#PacBio #ASGCT
We’re excited to be part of the leading event for gene and cell therapy experts. Discover how HiFi sequencing is unlocking new possibilities in research and accelerating breakthroughs across the field.
#PacBio #ASGCT
For a taste of #NewOrleans, stream #WWOZ for music from Louisiana while packing for #ASGCT2025
Also a good music calendar site – msg me for my music club suggestions. #Jazz #Zydeco #CRISPR
www.wwoz.org/schedule
Also a good music calendar site – msg me for my music club suggestions. #Jazz #Zydeco #CRISPR
www.wwoz.org/schedule
May 10, 2025 at 2:34 PM
For a taste of #NewOrleans, stream #WWOZ for music from Louisiana while packing for #ASGCT2025
Also a good music calendar site – msg me for my music club suggestions. #Jazz #Zydeco #CRISPR
www.wwoz.org/schedule
Also a good music calendar site – msg me for my music club suggestions. #Jazz #Zydeco #CRISPR
www.wwoz.org/schedule
Don't miss today's Outstanding New Investigator Symposium at #ASGCT2025! Rising stars in genetic medicine present breakthrough research spanning rare metabolic disorders, CAR T-cell innovations, and vector engineering advances. 10:15 AM - NOON, Hall F. #CGTInnovation
May 16, 2025 at 3:22 PM
Don't miss today's Outstanding New Investigator Symposium at #ASGCT2025! Rising stars in genetic medicine present breakthrough research spanning rare metabolic disorders, CAR T-cell innovations, and vector engineering advances. 10:15 AM - NOON, Hall F. #CGTInnovation
Got a weird look from someone at #asgct2025 when I took a spam timeshare call and told them I cant since I am incarcerated under house arrest for the next 5 years
Relax it is a joke!
Relax it is a joke!
May 15, 2025 at 6:52 PM
Got a weird look from someone at #asgct2025 when I took a spam timeshare call and told them I cant since I am incarcerated under house arrest for the next 5 years
Relax it is a joke!
Relax it is a joke!
Hear how gene therapies can address serious genetic diseases before birth. Dr. Tippi MacKenzie will deliver a George Stamatoyannopoulos Memorial Lecture at the @asgct.bsky.social Annual Meeting on May 15. #ASGCT2025 #GeneTherapy #PrenatalResearch #raredisease
May 14, 2025 at 1:44 PM
Hear how gene therapies can address serious genetic diseases before birth. Dr. Tippi MacKenzie will deliver a George Stamatoyannopoulos Memorial Lecture at the @asgct.bsky.social Annual Meeting on May 15. #ASGCT2025 #GeneTherapy #PrenatalResearch #raredisease
No adorable infants involved in my work, but I am really proud of what I've done so far and am excited for Dr. Xiao Wang's talk today at 1:30PM central at #ASGCT2025! I started our lab's work on PXE from the ground-up and have learned so much along the way.
May 16, 2025 at 2:51 PM
No adorable infants involved in my work, but I am really proud of what I've done so far and am excited for Dr. Xiao Wang's talk today at 1:30PM central at #ASGCT2025! I started our lab's work on PXE from the ground-up and have learned so much along the way.
🚨 The full collection of #ASGCT2025 abstracts is available NOW! 🚨
Check out the most innovative science in the #celltherapy and #genetherapy field, with 2,200 pieces of original research—over 10% more than last year!
Read + download the latest #CGT research. www.asgct.org/Publications...
Check out the most innovative science in the #celltherapy and #genetherapy field, with 2,200 pieces of original research—over 10% more than last year!
Read + download the latest #CGT research. www.asgct.org/Publications...
Now Available: 28th Annual Meeting Abstracts
Read the most innovative science in the field, with 2,200 pieces of original research@over 10% more than last year!@
www.asgct.org
April 28, 2025 at 10:13 PM
🚨 The full collection of #ASGCT2025 abstracts is available NOW! 🚨
Check out the most innovative science in the #celltherapy and #genetherapy field, with 2,200 pieces of original research—over 10% more than last year!
Read + download the latest #CGT research. www.asgct.org/Publications...
Check out the most innovative science in the #celltherapy and #genetherapy field, with 2,200 pieces of original research—over 10% more than last year!
Read + download the latest #CGT research. www.asgct.org/Publications...
📢 Calling all #raredisease + #CGT patient advocates: we want you to attend #ASGCT2025!
Patients, advocates, + caregivers can apply for free registration + nonprofit organizations can receive exhibitor discounts. Apply now! #PatientEducation #PatientAdvocacy annualmeeting.asgct.org/register/pat...
Patients, advocates, + caregivers can apply for free registration + nonprofit organizations can receive exhibitor discounts. Apply now! #PatientEducation #PatientAdvocacy annualmeeting.asgct.org/register/pat...
February 12, 2025 at 3:48 PM
📢 Calling all #raredisease + #CGT patient advocates: we want you to attend #ASGCT2025!
Patients, advocates, + caregivers can apply for free registration + nonprofit organizations can receive exhibitor discounts. Apply now! #PatientEducation #PatientAdvocacy annualmeeting.asgct.org/register/pat...
Patients, advocates, + caregivers can apply for free registration + nonprofit organizations can receive exhibitor discounts. Apply now! #PatientEducation #PatientAdvocacy annualmeeting.asgct.org/register/pat...
One can't present at #ASGCT2025 without a bit of nourishment and science discussion! Ready to meetup?
Good luck with the flight delays and I'll see you all shortly in #NewOrleans
Good luck with the flight delays and I'll see you all shortly in #NewOrleans
May 12, 2025 at 9:04 PM
One can't present at #ASGCT2025 without a bit of nourishment and science discussion! Ready to meetup?
Good luck with the flight delays and I'll see you all shortly in #NewOrleans
Good luck with the flight delays and I'll see you all shortly in #NewOrleans
👁️ Visit VisionGift at ASGCT 2025 in New Orleans – Booth #1915! Don’t miss Poster #580 on Tuesday, where Cerys Easton presents our latest findings on post-mortem retinal transcriptome & proteome changes. #ASGCT2025 #Retina #Proteomics #Transcriptomics #GeneTherapy
May 13, 2025 at 6:52 PM
👁️ Visit VisionGift at ASGCT 2025 in New Orleans – Booth #1915! Don’t miss Poster #580 on Tuesday, where Cerys Easton presents our latest findings on post-mortem retinal transcriptome & proteome changes. #ASGCT2025 #Retina #Proteomics #Transcriptomics #GeneTherapy