This study shows that #AAV8-mediated FVIII #GeneTherapy with #Tacrolimus–#Glucocorticoid prophylaxis achieves sustained FVIII activity and reduces bleeding in #HemophiliaA, with favorable safety outcomes.
#STTT #OpenAccess: doi.org/10.1038/s413...

Deafness reversed: One injection restores hearing in just weeks A new gene therapy is giving people born deaf the chance to hear, often within just weeks. In a small but groundbreaking study, researchers delivered a working copy of a key hearing gene directly into...

"In a small [10 patients] but groundbreaking study, researchers delivered a working copy of a key hearing gene [OTOF] directly into the inner ear using a single injection." www.sciencedaily.com/releases/202... @ki.se #genetherapy #hearing #keeplistening

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/TRttVM
#GeneTherapy #CellTherapy #Biotech #DueDiligence #InvestmentInsights

Hemophilia Therapy Trends 2026: Market & Innovation Explore the latest hemophilia treatment trends, including adoption of gene therapy and non-factor therapies, key challenges, and US vs EU market insights.

What’s shaping the future of hemophilia treatment?

Innovation, cost, and patient adherence are key drivers.
Get the full insights in our latest case study 👇
www.sperresearch.com/Case-Studies...

#HemophiliaTreatment #GeneTherapy #HealthcareInnovation #PharmaTrends #BiotechIndustry #RareDiseases

Trial at @ki.se
👂 10 pts 1-24yo
👂 deaf due to mutant OTOF -> otoferlin protein
👂 AAV delivered OTOF gene into inner ear thru "round window" of cochlea

Study: AAV gene therapy for autosomal recessive deafness 9: a single-arm trial www.nature.com/articles/s41... ($)

#deafness #hearing #geneTherapy

All 3 Langenhop kids were born with a rare, often-fatal immune disorder called LAD-I. After joining a UCLA #GeneTherapy trial, they're now "normal, happy kids" with functional immune systems.
Their trial participation helped make last week’s FDA accelerated approval possible.
💙 bit.ly/4sUCztg

Positioning Gene Therapy in SMA: Insights From 64-Week STEER Study Outcomes | NeurologyLive - Clinical Neurology News and Neurology Expert Insights Nazem Atassi, MD, SVP and Global Development Head of Neurology & Gene Therapy at Novartis, discussed 64-week STEER data and the evolving role of one-time gene therapy in SMA care.

🧬 New 64-week data from the STEER study highlight the potential durability of one-time gene therapy in #SMA

📈 Key outcomes
• +2.75 HFMSE improvement
• +2.93 RULM improvement
• 🚫 new safety signals

Expert insights from Nazem Atassi, MD ⬇️
www.neurologylive.com/view/positio...
#Neurology #GeneTherapy

Deafness reversed: One injection restores hearing in just weeks sciencedaily.com · Karolinska Institutet A new gene therapy is giving people born deaf the chance to hear, often within just weeks. In a small but groundbreaking study, researchers delivered a working copy of a key hearing gene directly into the inner ear using a single injection. All ten patients, ranging from young children to adults, experienced improved hearing, with some showing rapid gains in just one month.

sciencedaily.com · Karolinska Institutet

A new gene therapy is giving people born deaf the chance to hear, often within just weeks. In a small but groundbreaking study, researchers delivered …

#science #deaf #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/TRrHSn
#CVC #InvestingInInnovation #VentureCapital #GeneTherapy #AI

Interested in learning about the next wave of medicines? Register for this seminar as we discuss the promise of #mRNATherapy as alternative to #ViralBased #GeneTherapy. We will cover a wide range of applicable therapy areas and #translationalstrategies.#raredisease #Cancer #LNP #PrecisionMedicine

Programmable macromolecule delivery via engineered trogocytosis Nature Cell Biology - Chen et al. engineer TRANSFER (trogocytosis-inspired receptor transfer and functional effector release), a method to deliver functional cargos from donor to recipient cells in...

Ever heard of 'cellular nibbling'?
In our @NatureCellBio paper, we’ve turned trogocytosis into a delivery tool for programmable medicine. Congrats to @xinyi-chen.bsky.social and team!
Link: rdcu.be/fa90i
@StanfordBioE @StanfordChEMH @CZBiohub
#GeneTherapy #SyntheticBiology

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

Is Gene Therapy Overhyped as a Medical Paradigm? | Niche Gene therapy has transformed care for rare diseases — but is it a scalable future for medicine? A critical look at the costs, complexity and limits of gene-centric approaches.

Breakthrough or blind spot? Gene therapies promise cures—but at what cost to equity and real-world impact? A recent discussion made me rethink the story we’re telling: niche.org.uk/gene-therapy... #GeneTherapy #HealthPolicy

Submit your research and present alongside global leaders in cell and gene therapy.
Submission Deadline: June 10, 2026. buff.ly/FbepnIc
#ISCTAsia2026 #CellTherapy #GeneTherapy #CGTTranslation #Singapore

Gene Therapy to Be Studied for Treating Type 1 Diabetes A phase 1/2 trial of a novel viral vector gene therapy in adults with type 1 diabetes is scheduled to begin this year.

A clinical trial of #genetherapy for #type1diabetes is set to begin this year. For real. www.medscape.com/viewarticle/...

⏳ LAST CALL!
Applications for the #GeneTherapyAccelerator are closing soon. Only 10 teams will be selected to accelerate their innovation.
📅 Program starts April 22nd.
Don’t miss out 🔗 forms.office.com/e/eRWZ2kdz3q
Learn more: tinyurl.com/genehpt
#GeneH #GeneTherapy #HorizonEurope

Committee advances CAR‑T advisory council bill amid concerns about duplication and conflicts Senate Bill 451 to create an advisory council advising the Department of Public Health on CAR‑T and gene‑therapy delivery, training, and referrals was moved JFS after debate; opponents said it duplicates federal oversight and accreditation while supporters said a state council can improve access and coordinate grants for rare diseases such as sickle cell.

A heated debate in Connecticut sees Senate Bill 451 advance, sparking concerns over duplication of federal oversight while supporters argue it could enhance access to life-saving gene therapies.

Get the details!

#CT #CitizenPortal #GeneTherapy #PatientCare #HealthAccess

🚀 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/TRpD9k #GeneTherapy #SocialListening #LucidQuest
@ASGCTherapy @ESGCT

A photograph of UCLA's Dr. Donald Kohn wearing a blue shirt and tie with a white lab coat. There is a quote overwriting the image. The quote reads, "I saw a marked improvement across all patients in terms of restored immune function. Witnessing that these patients no longer battle life-threatening infections has been incredibly meaningful." Attribution is: Distinguished professor of microbiology, immunology and molecular genetics Dr. Donald Kohn

How does the FDA-approved LAD-I gene therapy work?

Doctors take the patient's OWN stem cells, add a healthy copy of the mutated gene, and return them. No donor needed. No transplant rejection risk.

All 9 trial patients: restored immune systems.

🔗 lnkd.in/gDJzjgvx

#GeneTherapy #StemCells

Implications of the FDA’s new plausible mechanism framework for the development of a personalized in vivo prime editing platform The authors report initial proof-of-concept studies supporting a customizable prime editing platform geared to the treatment of 7 urea cycle disorders and other liver-centered disorders, as well as th...

Read the full publication (open-access) here: www.cell.com/ajhg/fulltex... (4/4)

#GeneTherapy #PersonalizedMedicine #RareDisease

Academic institutions tackle gene therapy costs and accessibility Academics innovate gene therapy manufacturing to cut costs and improve access, addressing commercialization challenges and saving lives worldwide

What happens to gene therapies that are proven to save lives, but that are not commercially viable? Academic institutions are picking up the slack in a for-profit industry.

Read my full article below:
#genetherapy #access #nonprofit

www.bioprocessintl.com/upstream-dow...

FDA demands sham arm for Huntington’s gene therapy The US agency’s U-turn would postpone treatment for participants in the placebo group.

The FDA asked pharma company UniQure for a “double-blind, sham surgery-controlled study” of its mutation-rescuing Huntington’s disease therapy. Study leader Edward Wild has expressed ethical concerns.

Read my full article below:
#FDA #Huntington #genetherapy

www.bioxconomy.com/modalities/f...

Drug Screen Found Way to Keep Dying Vision Cells Alive Key Takeaways Central vision loss results from the death of cone photoreceptors, which traditional treatments cannot halt. A team at the Institute of

Drug Screen Found Way to Keep Dying Vision Cells Alive #Science #HealthandMedicine #GeneticsandGenomics #VisionResearch #GeneTherapy #HealthInnovation

scienceblog.com/drug-screen-found-way-to...

The first look at our #ISCTAsia2026 speaker lineup is here! buff.ly/a6dVmE2
📢 View our Keynote, plenary, and concurrent speakers heading to Singapore this Sept 2–5. #ISCTAsia2026 #CellTherapy #GeneTherapy #CGTTranslation #Singapore

CRISPR/CasRx-mediated dual knockdown of Ctnnb1 and Smo effectively suppresses Wnt/β-catenin and Ihh signaling to reduce #osteoarthritis progression, highlighting a promising RNA-targeting #GeneTherapy strategy for OA treatment.

#OpenAccess: doi.org/10.1016/j.ge...

Got questions? 🤔
🔹 Format: Blended (approx. 8h/week). 🔹 Teams: Max 10 teams. 🔹 Focus: Dedicated to #GeneTherapy translation.
Ready to take the next step? Learn more:https://tinyurl.com/genehpt
#GeneH #GeneTherapyAccelerator #HorizonEurope
@kemijski.bsky.social
@ctgct.bsky.social

A midbrain–cortical circuit mediated by claustrum neuronal ensemble orchestrates drug-paired context memory processing 👉 https://doi.org/10.1172/jci196944 Sex-specific Effects of Acute Stress on Emergence from Isoflurane Anesthesia via Brainstem Circuitry in Mice 👉 https://doi.org/10.1097/aln.0000000000005826 Prefrontal cortex-to-hypothalamic outputs orchestrate cuepotentiated palatable food consumption via AMPKβ2 signaling 👉 https://www.nature.com/articles/s41421-025-00857-2 A bone-derived hormone permits rapid visual escape via GPR37 receptor in a subpopulation of VTA GABAergic neurons 👉 https://www.cell.com/neuron/abstract/S0896-6273(25)01004-9 The axon initial segment-associated microglia regulate neuronal activity and visual perception 👉 https://doi.org/10.1038/s41422-026-01218-8 Glioblastoma-secreted C1QL1 orchestrates tumor microtube expansion and neural synaptic pruning to drive malignant synapse formation and recurrence 👉 https://doi.org/10.1158/2159-8290.cd-25-1123 Sleep disturbance triggers aberrant activation of vagus circuitry and induces intestinal stem cell dysfunction 👉 https://www.cell.com/cell-stem-cell/fulltext/S1934-5909(26)00025-1 Hypothalamic clock governs circadian pain 👉 https://www.science.org/doi/10.1126/science.ady6455 A sympathetic-eosinophil axis orchestrates psychological stress to exacerbate skin inflammation 👉 https://www.science.org/doi/10.1126/science.adv5974

🌟 A collection of recent client articles. 🧬
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If you have any product inquiries or research needs, feel free to reach out to us at bd@ebraincase.com
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#BrainCaseBiotech #Neuroscience #GeneTherapy

Looking forward to contributing to the development of this evolving curriculum and to helping bring cutting-edge science closer to everyday clinical care 💊

#Rheumatology #MedicalEducation #EULAR #CellTherapy #GeneTherapy #Autoimmunity #Lupus

Watch today's Century Report podcast here:

https://www.youtube.com/watch?v=735bE_-NW4k

#AISafety #GeneTherapy #SolarEnergy

Every xAI cofounder is gone. Claude subscribers doubled during the Pentagon confrontation. Solar efficiency crossed the Shockley-Queisser limit. Gene therapy decoupled pain from addiction. #AISafety #GeneTherapy #SolarEnergy sharedsapience.com/century-report/the-centu...