Amino acid supplementation enhances in vivo efficacy of lipid nanoparticle–mediated mRNA delivery in preclinical models Coinjection of an amino acid cocktail with LNPs enhanced in vivo delivery efficacy and improved preclinical mRNA therapy outcomes.

Amino acid supplementation enhances the uptake of lipid nanoparticles carrying #mRNA therapies in vitro and in mice, suggesting this simple approach could overcome delivery efficacy roadblocks in mRNA #GeneTherapy.

Learn more in #ScienceTranslationalMedicine: https://scim.ag/4ul12ZL

Recent gene therapy updates from industry:

Precision BioSciences Receives FDA Fast Track Designation for PBGENE-DMD and Announces Duchenne Muscular Dystrophy Investor Event (1/5)

investor.precisionbiosciences.com/news-release...

#GeneTherapy #IndustryNews #RareDisease

Learn more about the current status of AAV gene therapy as a potential treatment for GM1 gangliosidosis.

READ: curegm1.org/aav-gene-therapy-for-gm1

#curegm1 #raredisease #aav #genetherapy #research

📢 Meet us at Advanced Therapies Conference UK – March 17, 14:55 (Track 7)
🎤 Angela Columbano will share our latest innovations in gene therapy platforms.
🤝 Meet Angela & Imen Mestiri in London to explore partnerships shaping the future of advanced therapies.
#GeneTherapy

Ovarian Cancer’s Silent Killer: A New Hope Emerges Ovarian cancer's silent threat may finally be met with a revolutionary gene therapy. Discover the breakthrough offering new hope.

Ovarian Cancer’s Silent Killer: A New Hope Emerges #OvarianCancer #CancerAwareness #GeneTherapy #HealthCare #MedicalBreakthrough

BioPharma (+ Google) Corporate Venture Capital Investment Trends: Who is leading in 2025? Get instant access to a concise analysis of 22 BioPharma (+ Google) CVCs and their portfolios. In minutes, you’ll see where strategic capital is flowing across Technology and Therapy Areas—and how 2025 trends compare to 2023. What’s inside: Clear mapping of CVC focus by Technology and Therapy Areas Trend shifts vs. 2023 to validate your thesis Actionable insights to spot momentum early How it works: Fill out the form to receive your report immediately. Questions or feedback? info@lqventures.com LucidQuest empowers BioPharma executives and Life Sciences investors with up-to-date analysis. Stay tuned for 2025 deep dives on Cell & Gene Therapy, Digital Health, Oncology & Hematology, and more.

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...

Board directs technical advisory committee to evaluate newborn screening for MLD after federal RUSP listing After the federal RUSP added metachromatic leukodystrophy (MLD), the board declined direct rulemaking and directed staff to convene a technical advisory committee to assess screening feasibility, treatment access, costs, and equity implications in Washington.

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

Regenxbio, Solid report data with Duchenne gene therapies Regenxbio and Solid Bio say their gene therapies for Duchenne muscular dystrophy show no sign so far of the liver issues seen with Sarepta's Elevidys.

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.

Cincinnati Children’s Chosen to Prepare Novel CAR‑T Therapy for Further U.S. Clinical Development - Research Horizons

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 co-authors New England Journal of Medicine article First year MD/PhD student Connor J. Lewis performed neuroimaging for the study on GM1 gangliosidosis.

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

Manufacturing contaminants linked with toxicity in gene therapies A research team from London examined the relationship between adenovirus-associated virus (AAV)-based vectors and liver toxicity in gene therapy patients.

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 and Rare Disease Intelligence - LucidQuest Strategic insights for gene and cell therapy from R&D through commercialization. Primary KOL research, competitive analysis, and investment diligence in rare diseases.

🧬 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 The conference provides an interactive environment where researchers, clinicians, and innovators can collaborate to accelerate progress in targeted

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

BioPharma (+ Google) Corporate Venture Capital Investment Trends: Who is leading in 2025? Get instant access to a concise analysis of 22 BioPharma (+ Google) CVCs and their portfolios. In minutes, you’ll see where strategic capital is flowing across Technology and Therapy Areas—and how 2025 trends compare to 2023. What’s inside: Clear mapping of CVC focus by Technology and Therapy Areas Trend shifts vs. 2023 to validate your thesis Actionable insights to spot momentum early How it works: Fill out the form to receive your report immediately. Questions or feedback? info@lqventures.com LucidQuest empowers BioPharma executives and Life Sciences investors with up-to-date analysis. Stay tuned for 2025 deep dives on Cell & Gene Therapy, Digital Health, Oncology & Hematology, and more.

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