#genetherapy search results
Today, @DesaiMilindY shared new interim clinical data on MyPEAK™-1 Phase 1b/2 clinical trial for TN-201 at #AHA25. Missed it? Tune into our webcast on Monday (11/10) at 8 am EST. Details in our press release: bit.ly/4nIOfMg #GeneTherapy #HeartDisease
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.
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.
$QURE: this might a way out for #FDA and start restoring agency reputation: #geneTherapy #GeneEditing $XBI $IBB $XLV
🧠🔬 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
#KlothoNeurosciences is honored to receive the @BioTech_Awards' Cell Therapy Innovation of the Year Award! This award recognizes #KLTO's work advancing cell and #genetherapy for neurodegenerative disorders. Full release: bit.ly/47J9WWn #BiotechAwards $KLTO
Huge milestone for #UBA5 #genetherapy! The pre-IND package for the UBA5 gene therapy program has been submitted to the FDA. A major step toward the first-ever UBA5 treatment. Thank you to the incredible team @UMassChan and everyone who has supported us along the way.
Simultaneous Publication from Cardiovascular Research @CVR_TomaszGuzik First-in-human study of TN-201 gene therapy for hypertrophic cardiomyopathy shows promising safety & imaging results. Explore the future of cardiac care: oxford.ly/483xI0M #GeneTherapy #AHA2025
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
One dose. Deep brain delivery. 75% disease slowing. What’s next for gene therapy in HD? Dr. Victor Sung (@UABNews) breaks down the AMT-130 Huntington’s trial in a @medcentralmd interview: bit.ly/43tZOzB #Neurology #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
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.
MR-imaging shows complete bilateral putaminal perfusion after convection-enhanced gene therapy using an ‘infuse-as-you-go’ technique. This allows for precise cannula placement, dynamic repositioning and full target coverage for optimal therapeutic benefit. #GeneTherapy…
📢 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
$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
First American Society of Nephrology✔️ At Nionyx, we are building next-generation kidney AAV therapies to help make a meaningful impact for patients. We are privileged and excited to be here. Onwards. #asn #kidneytherapy #genetherapy
Longevity Gene Offers Hope for Aging Disease famouswriters.io/longevity-gene… #aging #geneTherapy #heartHealth
Tenaya Therapeutics’ TN-201 gene therapy shows early success in treating MYBPC3 hypertrophic cardiomyopathy business-news-today.com/tenaya-therape… #TenayaTherapeutics #geneTherapy #HCM #MYBPC3 #cardiology
Some of the larger population geared #geneTherapy #CRISPR #transformative curable treatments can be expanded if available in 2 flavors: 1&DONE, and lower 2 dose course to limit excessive toxicity effects🧬🩺 $NTLA $CRSP $NTLA #AHA2025 $IBB $XBI $arkG
Merck’s $MRK pill to reduce cholesterol on top of statins matches results from injectables statnews.com/2025/11/08/mer… via @cooney_liz #AHA25
CRISPR Cures 97% CRISPR therapy cures 97% sickle cell patients, FDA approval November 16, 2025; one-time edit, Vertex $2.2M, global launch 2026, $3B market. #CRISPRSickleCure #GeneTherapy #IB #InvestorBytes investorbytes.com/crispr-cures-9…
The implications of CRISPR gene editing for treating genetic diseases. #CRISPR #GeneTherapy
Promising early data for TN-201 gene therapy in MYBPC3-associated HCM 👏 ✅ Well tolerated ✅ Increases in MyBP-C protein ✅ Improvements in LV mass, wall thickness & biomarkers Next steps: longer follow-up & broader enrollment. #AHA25 #HCM #GeneTherapy
Today, @DesaiMilindY shared new interim clinical data on MyPEAK™-1 Phase 1b/2 clinical trial for TN-201 at #AHA25. Missed it? Tune into our webcast on Monday (11/10) at 8 am EST. Details in our press release: bit.ly/4nIOfMg #GeneTherapy #HeartDisease
Tired of feeling sluggish? Implant the cheetah's ACTN3 gene for explosive speed bursts! Sprint like the wind and leave traffic behind. #GeneTherapy #SuperSpeed
Gene therapy dreams on hold? Inside the FDA move that rattled Tenaya Therapeutics business-news-today.com/gene-therapy-d… #TenayaTherapeutics #FDA #GeneTherapy #BiotechStocks #TN201
Simultaneous Publication from Cardiovascular Research @CVR_TomaszGuzik First-in-human study of TN-201 gene therapy for hypertrophic cardiomyopathy shows promising safety & imaging results. Explore the future of cardiac care: oxford.ly/483xI0M #GeneTherapy #AHA2025
This #NationalSTEMDay, we’re celebrating the four fellows at #Rocket who are advancing science, technology, engineering & mathematics in the #genetherapy field. We asked them to share what inspired their passion for #STEM & what continues to inspire them today. Read more⬇️
एक साल के Miles Mahota ने अपनी विरासत में मिली बहरेपन को जेनेटिक थेरेपी से मात दी! 🥰❤️Boston Children's Hospital में हुई ओटोफेरिन जीन थेरेपी से उन्होंने पहली बार अपने माता-पिता की आवाज़ सुनी। यह वैज्ञानिक जीत भविष्य में बहुत से बच्चों के लिए आशा की किरण है। #GeneTherapy
Development of an AAV-Based Gene Therapy for the Ocular Phenotype of Friedreich's Ataxia #CureFA @MolTherapy #GeneTherapy cell.com/molecular-ther…
News from biotech dlvr.it/TP8RNr #genetherapy #celltherapy
New patent application #US20250339560A1 by #Akouos explores innovative #GeneTherapy for hearing loss. It details constructs with coding sequences linked to promoters, encoding therapeutic polypeptides. Includes AAV constructs for treating hearing loss/deafness.…
Longevity Gene Offers Hope for Aging Disease famouswriters.io/longevity-gene… #aging #geneTherapy #heartHealth
🔬 Systematic multi-trait AAV capsid engineering for efficient gene delivery. Nature communications(2024) doi.org/10.1038/s41467… @grok summary⬇️ #GeneTherapy #AAV
#FDA watch around 1:00, despite scare of scrutinizing #geneTherapy regulatory process - they seem to actually prefer #curative cost effective/lower stress on healthcare treatments. $CRSP $BEAM $CRBU $EDIT Watch #InPlay, as $NTLA nex-z trial is put on hold and tests regulators⏱️
🔊 Running through the latest batch of National Priority Voucher recipients with Dr. Mundkur. More to come!
I think Professor Angus Dalgleish has considerably more experience & ability than you to express an opinion on the safety of a medical intervention mRNA #Covid #GeneTherapy that has no long term safety data Not to mention the research carried out by the McCullough foundation
Today, @DesaiMilindY shared new interim clinical data on MyPEAK™-1 Phase 1b/2 clinical trial for TN-201 at #AHA25. Missed it? Tune into our webcast on Monday (11/10) at 8 am EST. Details in our press release: bit.ly/4nIOfMg #GeneTherapy #HeartDisease
Smiles all around at @EuroHuntington’s excellent conference in Bucharest 🇷🇴. Pictured here are Astri, President of the EHA and Sophia, HD patient advocate from Germany. Stop by our booth and speak to Dan to learn more about #genetherapy and our #AMT130 program.
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
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
$QURE: this might a way out for #FDA and start restoring agency reputation: #geneTherapy #GeneEditing $XBI $IBB $XLV
🧬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
🧠🔬 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
Busy final day at @EuroHuntington’s conf in Bucharest 🇷🇴. Our Medical Safety Dir Alejandra Padovani participated in a panel on clinical trials and our Exec Dir of Pt Adv Dan Leonard presented updates on our AMT-130 program to an eager audience. #genetherapy #Huntingtonsdisease
Huge milestone for #UBA5 #genetherapy! The pre-IND package for the UBA5 gene therapy program has been submitted to the FDA. A major step toward the first-ever UBA5 treatment. Thank you to the incredible team @UMassChan and everyone who has supported us along the way.
Simultaneous Publication from Cardiovascular Research @CVR_TomaszGuzik First-in-human study of TN-201 gene therapy for hypertrophic cardiomyopathy shows promising safety & imaging results. Explore the future of cardiac care: oxford.ly/483xI0M #GeneTherapy #AHA2025
🚨 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
एक साल के Miles Mahota ने अपनी विरासत में मिली बहरेपन को जेनेटिक थेरेपी से मात दी! 🥰❤️Boston Children's Hospital में हुई ओटोफेरिन जीन थेरेपी से उन्होंने पहली बार अपने माता-पिता की आवाज़ सुनी। यह वैज्ञानिक जीत भविष्य में बहुत से बच्चों के लिए आशा की किरण है। #GeneTherapy
#KlothoNeurosciences is honored to receive the @BioTech_Awards' Cell Therapy Innovation of the Year Award! This award recognizes #KLTO's work advancing cell and #genetherapy for neurodegenerative disorders. Full release: bit.ly/47J9WWn #BiotechAwards $KLTO
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
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
📢 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
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" 🧬
MR-imaging shows complete bilateral putaminal perfusion after convection-enhanced gene therapy using an ‘infuse-as-you-go’ technique. This allows for precise cannula placement, dynamic repositioning and full target coverage for optimal therapeutic benefit. #GeneTherapy…
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