Latest posts tagged with #Genetherapy on Bluesky
What’s shaping CVC investments for 2025? Key trends: AI/ML, Gene Therapy, and a focus on Immune Disorders & Neurodegenerative Diseases. Curious for more? 📥 Download the full report for insights! http://dlvr.it/TRVbh8
#CVC #InvestingInInnovation #VentureCapital #GeneTherapy #AI
This review summarizes the molecular drivers of #Osteoarthritis and highlights innovative therapeutic strategies, including #GeneTherapy and stem cell interventions, to enhance joint regeneration and improve disease outcomes.
#STTT #OpenAccess: doi.org/10.1038/s413...
Washington State is taking a crucial step towards newborn screening for metachromatic leukodystrophy (MLD), emphasizing the importance of early detection and equitable access to life-saving gene therapy.
Learn more here
#WA #PublicHealth #CitizenPortal #GeneTherapy #HealthEquity
Learn how gene therapy could transform the future of hereditary angioedema treatment: https://bit.ly/4mxg8aV
#GeneTherapy #HAETreatment #InnovationInMedicine #RareDiseaseTreatment #MedicalAdvances #AngioedemaNews #Bionews
#MDAConference: Barry Byrne, MD, PhD, Gene therapy expert and Board Member of the @mda.org, speaking at the MDA’s 2026 Clinical & Scientific Conference in Orlando, FL.
Watch the interview here 🎥
#RareDisease #MuscularDystrophy #GeneTherapy
📢 𝐂𝐚𝐥𝐥 𝐟𝐨𝐫 𝐏𝐚𝐩𝐞𝐫𝐬: 𝐂𝐮𝐫𝐫𝐞𝐧𝐭 𝐆𝐞𝐧𝐞 𝐓𝐡𝐞𝐫𝐚𝐩𝐲 🧪
Publish in a high-impact platform for molecular medicine & genetic therapies. 🧬
📈 Impact Factor: 3.3
📌 Editor: Dr Fanglin Guan
🔗 https://bit.ly/4b7BTdx
#Genetics #Biotech #GeneTherapy #ScienceSky #MedSky
🧬 Genetic Therapies for Rare Diseases: Developing Ethical Regulatory Policies
🔗https://bit.ly/4sIQl1A
#Bioethics #GeneTherapy #RareDiseases #GeneticMedicine #HealthPolicy
11-Mar-2026
Researchers develop new way to safely insert #gene-sized #DNA into the #genome
https://www.eurekalert.org/news-releases/1119454 #science #geneTherapy
Stop by exhibition stall #3 to find out more about the Hub, and how @edinburgh-uni.bsky.social is supporting innovation in advanced therapies.
Attending the Congress? Let us know; we look forward to connecting with you there.
#advancedtherapies #genetherapy #engineeringbiology
This week saw both #Regenxbio and #SolidBio report new #data with their #genetherapy candidates for #Duchenne muscular dystrophy (DMD), vying to join Sarepta's Elevidys on the market.
Our newly expanded Applied Gene and Cell Therapy Center was selected by Tempest Therapeutics to advance a dual-targeting CAR‑T therapy for relapsed/refractory multiple myeloma. Proud of our team advancing next-gen cell therapies! #CellTherapy #GeneTherapy @cincychildrens.bsky.social
Read more:
First-year MD/PhD student Connor Lewis used computational methods he developed for a study on the rare disease GM1 gangliosidosis, published in @nejm.org: direc.to/o6zM
The average lifespan for Type II GM1 gangliosidosis patients is 20 to 40 years.
#GeneTherapy #RareDisease @uofmass.bsky.social
🚀 Big buzz at #ASGCT25: #GeneEditing & #CRISPR headlines, 20% data-driven talk, 85% positive vibes on breakthrough therapies for #DMD & #rarediseases!🧬 Download our full #socialmedia report! http://dlvr.it/TRSr0P #GeneTherapy #SocialListening #LucidQuest
@ASGCTherapy @ESGCT
Advanced Therapeutics Postdoctoral Research Associates. Kings Buildings, University of Edinburgh. Closing date 10th April 2026. Image shows King's Buildings Campus with the Swann Building in the foreground.
📣 Calling talented and passionate researchers to apply for two exciting postdoctoral research associate roles with us at @edinburgh-uni.bsky.social. Both roles report to Prof. Susan Rosser, Hub academic director.
Role details below 👇
#genetherapy #engineeringbiology
🧬 Intrathecal gene therapy for SMA may offer outcomes comparable to existing treatments.
An indirect comparison presented at #MDA2026 suggests Itvisma shows similar motor function improvements to nusinersen and risdiplam.
Visit NeurologyLive to read more‼️
#Neurology #SMA #GeneTherapy #RareDisease
🧬 We are launching the #GeneTherapyAccelerator, a #GeneH initiative to translate breakthrough research into market-ready therapies.
Save the dates: 📅 April 22 & 29 📅 May 6 & 13
🔗 Apply now: forms.office.com/e/eRWZ2kdz3q
#GeneTherapy #HorizonEurope
@kemijski.bsky.social
@ctgct.bsky.social
Trump's divisive FDA vaccine regulator self-destructs, will exit agency (again) https://arstechni.ca #genetherapy #vaccines #Health #Prasad #fda
Live webinar: How to Build a Measurable Evidence Engine for Cell, Gene and RNA Therapies. Thursday, March 12 at 8am PDT/11am EDT/3pm GMT/4pm CET. Speaker: Todd Oakland, SVP & GM of Biopharma at DNAnexus, Bruce Church, Ph.D., EVP, Research & Early Development, Aitia Bio.
This Thursday: turn multimodal data into an evidence engine for drug development. xtalks.com/webinars/how...
Learn how to replace disconnected workflows and support clinical decision-making at scale.
@xtalks.com
#Pharma #Multiomics #DrugDevelopment #AI #GeneTherapy #CellTherapy
AAV-associated toxicity in gene therapies could be caused by contaminants in the manufacturing process, a research team based out of London claim. Liver biopsies tested positive for viral fragments.
Read my full article below:
#genetherapy #AAV #news
www.bioprocessintl.com/therapeutic-...
🧬 Two phase 3 trials are underway to evaluate salanersen in presymptomatic newborns with spinal muscular atrophy.
The STELLAR-1 and STELLAR-2 studies will assess outcomes with salanersen alone or following gene therapy.
Presented at the 2026 #MDAconference
#SMA #Neurology #GeneTherapy #RareDisease
🧬 Gene & Cell Therapy | #RareDiseases 🚀
#LucidQuest delivers sharp insights to help biotechs, investors & BD teams stay ahead in fast-moving markets.
📩 info@lqventures.com or http://dlvr.it/TRNgmx
#GeneTherapy #CellTherapy #Biotech #DueDiligence #InvestmentInsights
🧬 Real-world data suggest motor improvements in children with #SMA after switching to #genetherapy.
A Polish study found patients transitioning from nusinersen or risdiplam to onasemnogene abeparvovec showed gains on motor scales within 6 months. Read more on NeurologyLive!
#Neurology #RareDisease
2nd International Conference on Cancer Research & Immuno-Oncology
April 3,4,5 2026
Atlantis the Royal, Palm Jumeirah - Crescent Road - Dubai
www.vydya.com/events/2nd-i...
#CancerResearch #ImmunoOncology #GenitourinaryCancer #GeneTherapy #PrecisionOncology
#OncologyConference #MedicalConference
Do you know how bioinformatics analyses support cell and gene therapy research? 🤔Read our blog to find out! 💡
www.fiosgenomics.com/bioinformatics-analyses-...
#cellandgenetherapy #celltherapy #celltherapymanufacturing #genetherapy
What’s shaping CVC investments for 2025? Key trends: AI/ML, Gene Therapy, and a focus on Immune Disorders & Neurodegenerative Diseases. Curious for more? 📥 Download the full report for insights! http://dlvr.it/TRKfql
#CVC #InvestingInInnovation #VentureCapital #GeneTherapy #AI
`These data set the stage for a potential therapeutic strategy using antisense oligonucleotides to promote isoform switching in patients with RTT who carry partially functioning alleles of MECP2.`
www.science.org/doi/10.1126/scitranslmed...
#Rett #RettSyndrome #geneTherapy #antisense
Pleased to be sharing our latest data with the DMD community at MDA 2026 highlighting RIPPLE™ as an efficient, scalable, nonviral platform for full-length dystrophin delivery in DMD. Get more details: www.sonothera.com
#MDA #SonoThera #DMD #neuromusculardiseases #nonviral #ultrasound #genetherapy
Pleased to be sharing our latest data with the DMD community at MDA 2026 highlighting RIPPLE™ as an efficient, scalable, nonviral platform for full-length dystrophin delivery in DMD. Get more details: www.sonothera.com
#neuromusculardiseases #MDA #genetherapy #nonviral #ultrasound #DMD #SonoThera
A proof-of-concept trial in a single patient has shown that genetically engineered pancreas cells can survive transplantation without immunosuppression.
#chemsky #genetherapy 🧪
Text which says "Scaling AAV Manufacturing for Neuromuscular Gene Therapy" alongside an image on an AAV
AAV gene therapies can only progress if we can manufacture high‑quality vectors at scale.
MAGIC and DINAMIQS have been working to make AAV production more robust and development‑ready, helping ensure emerging neuromuscular therapies have a practical path toward the clinic.
#GeneTherapy #AAV
