Broad Institute of MIT and Harvard’s Post

𝐇𝐢𝐭-𝐚𝐧𝐝-𝐑𝐮𝐧 𝐄𝐩𝐢𝐠𝐞𝐧𝐨𝐦𝐞 𝐄𝐝𝐢𝐭𝐢𝐧𝐠 𝐏𝐫𝐨𝐯𝐢𝐝𝐞𝐬 𝐄𝐟𝐟𝐢𝐜𝐢𝐞𝐧𝐭 𝐚𝐧𝐝 𝐃𝐮𝐫𝐚𝐛𝐥𝐞 𝐄𝐩𝐢𝐠𝐞𝐧𝐞𝐭𝐢𝐜 𝐒𝐢𝐥𝐞𝐧𝐜𝐢𝐧𝐠 𝐈𝐧 𝐕𝐢𝐯𝐨, 𝐍𝐞𝐰 𝐒𝐭𝐮𝐝𝐲 𝐒𝐡𝐨𝐰𝐬 In a recent study, researchers at San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Italy addressed the critical need for experimental validation of durable epigenetic silencing after transient delivery of editors in vivo. Using lipid nanoparticles loaded with editor mRNAs, the team showed that a single administration led to a significant reduction in circulating Pcsk9 levels in mice for nearly a year. This proof-of-concept study paves the way for the development of advanced gene therapies based on epigenetic silencing, without altering the DNA sequence. Read more in our latest interview with Martino Alfredo Cappelluti, PhD. Interview by: Christos Evangelou, PhD https://lnkd.in/eEcTF2Y2 #crisprmedicinenews #crisprmedicine #epigeneticediting #crispr #geneediting

A study examined the potential for gene therapy in Friedreich’s ataxia, a disorder caused by mutations in the FXN gene leading to mitochondrial dysfunction. Researchers administered recombinant adeno-associated virus encoding human frataxin (AAVrh.10hFXN) in mice, inducing dose-dependent expression of mature human frataxin (hFXN) in the heart and liver, approaching endogenous mouse frataxin (mFXN) levels. While hFXN was found to be truncated, it was at different positions than mFXN, suggesting AAVrh.10hFXN as a promising approach for inducing therapeutic hFXN expression. #FriedreichsAtaxia #GeneTherapy #MitochondrialDysfunction #Frataxin #AAVrh10hFXN #NeurodegenerativeDisorder #RareDisease #ResearchUpdate #MedicalScience #TherapeuticIntervention https://lnkd.in/euXFhSXw

We are thrilled to share that the prestigious, peer-reviewed journal Gene Therapy has published our article on non-replicative HSV-1 vectors.   The publication, “Non-replicative herpes simplex virus genomic and amplicon vectors for gene therapy – an update,” explores the two main types of non-replicative HSV-1 vectors, highlighting their features, advancements, and potential for clinical applications.   “This review on non-replicative HSV vectors, published in Gene Therapy, illustrates the tremendous potential that these have to bring the benefits of DNA medicine to the field of chronic, highly prevalent diseases, particularly in neurology,” says Philippe Chambon MD, PhD, CEO of EG 427. “Clinical trials with nrHSV-1 vectors, including ours at EG 427, are progressing and demonstrate consistently the low immunogenicity, allowing for multiple re-administrations, and enhanced safety profiles of these vectors.”   Key points from the article include: ·     nrHSV-1 vectors show low immunogenicity, high safety, unrivaled DNA payload capacity and are adaptable for neuron-targeted therapies. ·     Significant breakthroughs in vector design have paved the way for clinical trials and approval. ·     Amplicons, although not yet in clinical trials, demonstrate exceptional potential as next-generation nrHSV therapeutics.   We’re driven by the mission to address neurological diseases that impact nearly 3 billion people globally. Our proprietary HERMES (HERpes based Modular Expression system) platform allows us to develop a broad range of therapies for these diseases, to provide these patients with safe and long-lasting drugs.   Our lead candidate, EG110A, is advancing in a phase 1b/2a study for Neurogenic Detrusor Overactivity (NDO) in spinal cord injury patients, with trials underway at top US institutions.   Stay tuned as we continue to make strides in this critical field – and click here for the full article https://lnkd.in/e8rvjAHz https://lnkd.in/eRxvNFdy   #genetherapy #vectors #spinalcordinjury #medicine #neurology

Researchers at the Harvard Medical School have developed a universal gene therapy for Diamond-Blackfan Anemia (#DBA), a rare genetic disorder characterized by impaired #erythropoiesis caused by #ribosomal protein mutations. the approach entails controlling the expression of #GATA1, a crucial #transcriptionfactor in erythropoiesis, only in developing erythroid cells using an erythroid-specific enhancer called #hG1E

We're excited to share this Cell & Gene article authored by Atsena co-founder Sanford Boye that highlights our pioneering work to address a critical challenge in gene therapy: achieving effective transduction across the retina.   This innovative approach to treating the #InheritedRetinalDisease X-linked retinoschisis (#XLRS) leverages a unique AAV capsid engineered for robust subretinal spread. This breakthrough enables efficient delivery of the therapeutic gene to target cells across the retina, offering hope to patients impacted by this vision-debilitating condition.   Read more about how our science is shaping the future of ocular #GeneTherapy: https://lnkd.in/ewThiuYy

Biomedicine Breakthrough: Complete Gene Insertion Now Possible in Human Cells

Researchers have developed a gene therapy approach using an advanced prime editing system to correct the F508del mutation, which causes cystic fibrosis. This technique allows precise editing of DNA to repair the mutation in patient-derived lung cells. The restored function of the edited cells matched current CF drug therapies, indicating the potential of gene therapy as a one-time treatment. However, the challenge remains in effectively delivering the gene-editing system to patient cells.

New Gene Therapy Reverses Heart Failure in a Large Animal Model with a Single IV Injection🌟 A groundbreaking gene therapy has demonstrated the potential to reverse heart failure in large animal models, offering hope for a condition that is typically irreversible without a transplant. Researchers at the University of Utah Health used a gene therapy approach to increase levels of cardiac bridging integrator 1 (cBIN1), a key protein often depleted in heart failure patients. Delivered via an adeno-associated virus (AAV9), the therapy introduced the cBIN1 gene directly into the heart cells of pigs with heart failure. The results were remarkable: the treated pigs showed significant improvements in heart function and survived six months beyond the typical fatal progression of the disease. This therapy not only halted the progression of heart failure but also reversed some of the damage by enhancing heart pumping efficiency and reducing structural deterioration. These changes represent a "reverse remodeling" process, restoring the heart closer to a normal state. Researchers noted improvements of up to 30% in heart function, far exceeding previous therapies that achieved only 5-10% improvements. This breakthrough is now being prepared for human clinical trials, with FDA approval anticipated in 2025. While more testing is needed, the results could redefine heart failure treatment by directly repairing heart tissue, a significant departure from current symptom-management approaches.. https://lnkd.in/gvpgQjVn

A recent study has demonstrated a promising gene therapy that reverses heart failure in a large animal model by restoring cardiac function. This therapy targets the cardiac bridging integrator 1 (cBIN1) protein, crucial for heart health, and has shown a 30% improvement in heart function. Unlike current treatments that only slow disease progression, this approach suggests potential for actual heart repair. The therapy is being adapted for human trials, with FDA approval sought for 2025. While further testing is needed, these findings could pave the way for transformative heart failure treatments.