#genetherapy search results
We're honored that our #genetherapy treatment for #AADCdeficiency received the Therapeutics Innovation of the Year by BioTech Breakthrough Awards, a milestone in advancing care for patients with a rare disease.
🧠🔬 Tweaking a single gene in microglia can transform them from Alzheimer's culprits to protectors, shielding brain connections and slowing disease. Curious how one genetic switch could change the game? news-medical.net/news/20251106/… #Alzheimers #Neuroscience #GeneTherapy @Nature
We're excited to sponsor & attend the @HuntingtonSG Clinical Research Congress in Nashville this weekend, where we'll connect with the #huntingtonsdisease research community & share a scientific presentation on our investigational #genetherapy candidate AMT-130. #HDCongress2025
What sets biologics apart from traditional meds? 🧬 Discover how living cells are revolutionizing treatments for diseases once impossible to cure - from gene therapies to precision cancer drugs. Learn more: news-medical.net/whitepaper/202… #Biologics #HealthcareInnovation #GeneTherapy
Breakthrough in #GeneTherapy: @HopkinsMedicine researchers developed a tumor-targeted viral vector for treating NF1. It suppresses tumor growth in preclinical models while minimizing off-target effects. Honoring Dr. Verena Staedtke’s visionary leadership. buff.ly/5MYqcGc
We had a wonderful weekend at #HDCongress25, where we engaged with #huntingtonsdisease clinicians, researchers, and advocates and gained valuable insights as we plan the path forward for our investigational #genetherapy candidate AMT-130.
📢 We’re hiring! @NeurosurgeryOSU & the Gene Therapy Institute are recruiting tenure-track faculty at the Associate/Full Professor level. Join our interdisciplinary team advancing novel #GeneTherapy approaches from lab to clinic. Apply by Dec 15 👉 go.osu.edu/C8Qz
Interested in learning more about how our innovative #GeneTherapy delivery method bypasses the blood-brain barrier for precise dosing in the brain? Watch below to see how we administer gene therapy directly to the thalamus through a specialized neurosurgical procedure.
Today uniQure announced that we have received feedback from the U.S. Food and Drug Administration (FDA) during a recent pre-Biologics License Application (BLA) meeting regarding AMT-130, our investigational #genetherapy for #huntingtonsdisease. uniqure.gcs-web.com/news-releases/…
$OCGN Ocugen is making strides with OCU410 for geographic atrophy & Stargardt, aiming for a 2027 BLA submission. OCU400, targeting retinitis pigmentosa, is in a Phase 3 trial, with potential for US & European regulatory paths. #OCUGEN #Genetherapy
The FDA decided not to fast-track the approval of AMT-130 for the treatment of Huntington's. Henry Miller describes the gene therapy as a "tour de force of neurosurgery and molecular biology." geneticliteracyproject.org/2025/11/03/tre… #GeneTherapy
Today Dr. Victor Sung, one of the investigators in our AMT-130 #genetherapy trial, gave an oral presentation on the AMT-130 topline pivotal data as part of the #HDCongress2025 HD Clinical Trials Update. #huntingtonsdisease
Gene therapies for rare diseases are advancing in Europe (2017–2023): 93 studies, 300 investigations; 73 therapies for 35 diseases; 12 approved, 15 in phase III. Europe shows strong potential but lags behind US & UK in R&D. 👉 oaepublish.com/articles/rdodj… #GeneTherapy #RareDiseases
$OCGN OcuGen is focusing on gene therapy for rare eye diseases, particularly OCU400 for retinitis pigmentosa. With potential FDA filing around 2026 and OCU410ST for Stargardt disease receiving FDA designation, the future hinges on trial successes. #genetherapy #OcuGen
Longevity Gene Offers Hope for Aging Disease famouswriters.io/longevity-gene… #aging #geneTherapy #heartHealth
🧠🔬 Tweaking a single gene in microglia can transform them from Alzheimer's culprits to protectors, shielding brain connections and slowing disease. Curious how one genetic switch could change the game? news-medical.net/news/20251106/… #Alzheimers #Neuroscience #GeneTherapy @Nature
Longevity Gene Offers Hope for Aging Disease famouswriters.io/longevity-gene… #aging #geneTherapy #heartHealth
We're honored that our #genetherapy treatment for #AADCdeficiency received the Therapeutics Innovation of the Year by BioTech Breakthrough Awards, a milestone in advancing care for patients with a rare disease.
$NTLA gr4 AE patient ☎️📣“complicating commodities being further evaluated” #InPlay small details matter more here #geneTherapy #CRISPR $arkG $IBB $XBI $XLV $BEAM $CRSP $EDIT $PRME $CRBU $QURE $RGNX
$NTLA gr4 adverse effects nex-z patient had “complicating comorbidities”… #InPlay #geneTherapy #CRISPR $arkG $BEAM $PRME $CRBU $CRSP $EDIT
$NTLA gr4 AE patient death - evaluating impact of physician reported “complicating comorbidities” #InPlay small details matter more - not yet over 👀 - unfortunately this will take ⏰ #geneTherapy #CRISPR $arkG $IBB $XBI $XLV $BEAM $CRSP $EDIT $PRME $CRBU $QURE $RGNX
$NTLA gr4 adverse effects nex-z patient had “complicating comorbidities”… #InPlay #geneTherapy #CRISPR $arkG $BEAM $PRME $CRBU $CRSP $EDIT
ICYMI: In October, @US_FDA granted platform technology designation to @KrystalBiotech and selected @AbeonaBio's ABO-503 for its RDEA Pilot Program. #genetherapy #celltherapy #FDA Read more: hubs.li/Q03S5Crg0
We are a family from Türkiye. Both of my sons are fighting brain tumors — my 8-year-old Yusuf and my 4-year-old Furkan. We are desperately seeking a chance for gene therapy or any treatment support from St. Jude. Please help us. 💔 #BrainTumor #GeneTherapy #StJude
$NTLA gr4 adverse effects nex-z patient had “complicating comorbidities”… #InPlay #geneTherapy #CRISPR $arkG $BEAM $PRME $CRBU $CRSP $EDIT
"All the scientific accolades aside, I think that the field also is where it is today in part because industry got involved." -Renier Brentjens, MD, PhD #CART #celltherapy #genetherapy Read more: hubs.li/Q03S4t610
Touchlight expands its mbDNA portfolio Read more here: bit.ly/3LoQkj0 Subscribe to our weekly newsletter for the latest news delivered to your inbox: bit.ly/47sT3kl #science #DNA #genetherapy
RAC1 Directly Phosphorylates Both PKM2 and FBP1 to Promote Radioresistance in Hepatocellular Carcinoma dlvr.it/TP6hPh #genetherapy #celltherapy
What is siRNA? Small interfering RNA = tiny molecules that silence genes. They guide cell machinery to destroy mRNA from target genes and stopping unwanted proteins. Think of siRNA as a gene “off switch.” #siRNA #biotech #genetherapy
The implications of CRISPR gene editing for treating genetic diseases. #CRISPR #GeneTherapy
The implications of CRISPR gene editing for treating genetic diseases. #CRISPR #GeneTherapy
What sets biologics apart from traditional meds? 🧬 Discover how living cells are revolutionizing treatments for diseases once impossible to cure - from gene therapies to precision cancer drugs. Learn more: news-medical.net/whitepaper/202… #Biologics #HealthcareInnovation #GeneTherapy
#Catalent to lay off 350 at Baltimore site due to large customer shift in demand. The reduction affects staff at Catalent's Harmans/BWI #viralvector facility, which manufactures #genetherapy drug substance and finished product. pharmamanufacturing.com/industry-news/… #CDMO #layoffs #NovoHoldings
🧠🔬 Tweaking a single gene in microglia can transform them from Alzheimer's culprits to protectors, shielding brain connections and slowing disease. Curious how one genetic switch could change the game? news-medical.net/news/20251106/… #Alzheimers #Neuroscience #GeneTherapy @Nature
We're excited to sponsor & attend the @HuntingtonSG Clinical Research Congress in Nashville this weekend, where we'll connect with the #huntingtonsdisease research community & share a scientific presentation on our investigational #genetherapy candidate AMT-130. #HDCongress2025
🧬 Rare Disease R&D Is Accelerating Breakthrough designations, gene therapy advances, and billion-dollar deals are fueling hope for patients with unmet needs. Beyond the headlines, the scale of innovation keeps growing 👇 #RareDisease #GeneTherapy #Biopharma #ClinicalTrials
📢 We’re hiring! @NeurosurgeryOSU & the Gene Therapy Institute are recruiting tenure-track faculty at the Associate/Full Professor level. Join our interdisciplinary team advancing novel #GeneTherapy approaches from lab to clinic. Apply by Dec 15 👉 go.osu.edu/C8Qz
Huge congratulations to Complement Therapeutics for receiving FDA clearance to begin their Opti-GAIN Phase I/II clinical trial. #LifeSciences #Innovation #GeneTherapy #HealthTech #WestLondonInnovators
Gene therapy slowed Huntington's disease progression by 75 per cent in trial patients, in what researchers describe as the first effective treatment. 🔗 Full story: buff.ly/f1bnrUt #Huntingtons #NeuroResearch #GeneTherapy #BrainHealth #Neurology
We are looking forward to the GTI speaker series this week where we'll hear from Dr. X. Margaret Liu on "A mitochondrial-targeted #genetherapy (mLumiOpto) for cancer treatment" 🧬
🧬Congratulations to @TomMendelEye on receiving a prestigious K08 award! 🎉 His project, “Insulin-accelerated surgical approach to pre-retinal #genetherapy,” will advance innovative strategies to treat inherited retinal diseases. 👁️@ohiostateeye #OhioStateResearch
🚨 Huntington’s Disease Update 🚨 🧬 uniQure reports positive Ph I/II data for AMT-130 gene therapy! Why it matters: 🔹 Huntington’s has no cure 🔹 Gene tx may slow progression 🔹 Milestones like this fuel hope & biotech innovation #Huntingtons #GeneTherapy #Clinicaltrial
Getting the value equation right for #genetherapy will have a real and tangible impact on patients’ ability to benefit from this game-changing field. gene-therapies.org/value-of-gene-…
Breakthrough in #GeneTherapy: @HopkinsMedicine researchers developed a tumor-targeted viral vector for treating NF1. It suppresses tumor growth in preclinical models while minimizing off-target effects. Honoring Dr. Verena Staedtke’s visionary leadership. buff.ly/5MYqcGc
In a first, a gene therapy seems to slow Huntington disease! 🤯 @uniQure_NV reported that its #genetherapy candidate AMT-130 reduced the progression of Huntington symptoms by 75% over 3 years in 12 people who had received it versus a comparison group. bit.ly/46UmXNE
Gene therapies for rare diseases are advancing in Europe (2017–2023): 93 studies, 300 investigations; 73 therapies for 35 diseases; 12 approved, 15 in phase III. Europe shows strong potential but lags behind US & UK in R&D. 👉 oaepublish.com/articles/rdodj… #GeneTherapy #RareDiseases
Less than 3 weeks before scientists and clinicians convene in Los Angeles, CA for @CellSymposia #CSStemCells2025. Check out the scientific program and register today: hubs.li/Q03Ngy5z0 #GeneTherapy #CellTherapy #Aging #Rejuvenation #StemCells
Fantastic talk today by Dr. Rabi Hanna, chairman of the Division of Pediatric Hematology/Oncology and BMT at Cleveland Clinic Children's, on gene therapy innovations in sickle cell disease and infectious challenges with this evolving field! 🧬🧫 #GeneTherapy #MedEd #IDFellows
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