NeurologyLive’s Post

Ocugen Inc has announced that first patient has been dosed in the ArMaDa Phase 1/2 clinical trial of OCU410 (AAV-RORA), a modifier gene therapy product candidate being developed for dry AMD (dAMD). According to the company, the Phase 1/2 trial will assess the safety and efficacy of OCU410 for geographic atrophy (GA) secondary to dAMD and will be conducted in two phases. The first phase is a multicenter, open-label, dose-ranging study, while phase 2 is a randomly assigned expansion phase in which subjects will be randomized in a 1:1:1 ratio to either 1 of 2 OCU410 dose groups or to an untreated control group. #news #dAMD #AMD #dryAMD #GeographicAtrophy #GA

Following #ECFS2024, Jennifer Taylor-Cousar, M.D., #ClinicalTrial Principal Investigator, discussed the progress of the AEROW trial investigating 4DMT’s aerosolized 4D-710 gene therapy candidate for CFTR modulator-ineligible/-intolerant #cysticfibrosis (CF) lung disease.   Listen to Dr. Taylor-Cousar as she dives into the data she presented at the conference and shares an update on the trial’s next steps with CGTLive. https://lnkd.in/ePRDPtsc

Our latest research article, “Endothelial to mesenchymal transition enhances permissiveness to AAV vectors in cardiac endothelial cells” is out in Molecular Therapy! This work explores a novel approach to improving gene therapy for cardiovascular disease by enhancing the efficiency of gene transfer to cardiac endothelial cells. We found that inducing endothelial to mesenchymal transition (EndMT) significantly boosts AAV transduction, offering new insights into therapeutic angiogenesis in the heart. One of the biggest lessons I’ve learned through this process is the importance of perseverance and attention to detail. Even when faced with unencouraging results, it’s crucial to never give up. The devil truly is in the details and staying focused on them can lead to significant breakthroughs. A sincere thank you to all my co-authors and collaborators—each of you made an invaluable contribution to this work. I want to extend a special thanks to our #CardioVascularBiology Group Leader, Serena Zacchigna, for her expert guidance, encouragement, and constant support throughout this journey. Last but not least, I wish to acknowledge the co-first author, Roman Vuerich. What started as my PhD project would never have seen the light of the day without your equal dedication and hard work. Thank you for being part of this achievement! You can read the full article on: https://lnkd.in/dhBGMWCd ICGEB #EndothelialCells #GeneTherapy #AdenoAssociatedVirusVectors #EndMT

Here’s my interview for VarSome about #NGS dectection of gene #fusions in #liquidbiopsy of advanced #NSCLC patients diagnosed with #ALK #ROS #RET fusions and #METex14 in tissue biopsy. And how we used #Varsome Premium for variant interpretation.

New findings from part 1 of the phase 3 EMBARK study assessing Sarepta Inc Therapeutics’ gene therapy delandistrogene moxeparvovec (SRP-9001) showed that the agent failed to meet its primary end point in change among patients with Duchenne muscular dystrophy (DMD); however, did demonstrate several benefits on other secondary analyses. https://lnkd.in/ethTq7b3

📑 𝗔𝘁𝘀𝗲𝗻𝗮 𝗧𝗵𝗲𝗿𝗮𝗽𝗲𝘂𝘁𝗶𝗰𝘀 𝗣𝘂𝗯𝗹𝗶𝘀𝗵𝗲𝘀 𝟭𝟮-𝗠𝗼𝗻𝘁𝗵 𝗗𝗮𝘁𝗮 𝗼𝗻 𝗔𝗧𝗦𝗡-𝟭𝟬𝟭 𝗳𝗼𝗿 𝗟𝗖𝗔𝟭 Atsena Therapeutics has shared positive 12-month results from their Phase I/II trial of ATSN-101, a gene therapy for Leber congenital amaurosis (LCA1). Kenji Fujita, MD, Chief Medical Officer, said, “The preliminary efficacy and robust safety data from our ongoing Phase I/II trial underscore the potential of ATSN-101 to be a first-in-class gene therapy for LCA1." 𝗟𝗲𝗮𝗿𝗻 𝗺𝗼𝗿𝗲 𝗮𝗯𝗼𝘂𝘁 𝘁𝗵𝗶𝘀 𝗽𝗿𝗼𝗴𝗿𝗲𝘀𝘀 𝗶𝗻 𝗴𝗲𝗻𝗲 𝘁𝗵𝗲𝗿𝗮𝗽𝘆 𝗳𝗼𝗿 𝘃𝗶𝘀𝗶𝗼𝗻 𝗿𝗲𝘀𝘁𝗼𝗿𝗮𝘁𝗶𝗼𝗻: https://lnkd.in/dyfeV3eh #GeneTherapy #ClinicalTrial #VisionLoss #LCA1 #EyeHealth

Missed last week’s webinar focused on services supporting cell and gene therapies? Watch the webinar recording to learn how our services help predict safety, toxicity, and efficacy of cutting-edge therapies. We also introduced our animal models including immunodeficient (B-NDG mice https://lnkd.in/epzJmBds) and humanized models. Don't miss this opportunity to stay ahead in the field of preclinical research: https://lnkd.in/exC7XnW7 #CellTherapies #GeneTherapies #PreclinicalResearch #WebinarRecording #bndg #immunodeficient

Navigating the Complex Landscape of Cell and Gene Therapy: Challenges and Solutions: There are numerous challenges with cell and gene therapies in oncology, such as accessibility, cost, and prior authorizations, as well as the need for improved education and training within the oncology community. #finance #pharmacy #lifesciences

#Gene therapy Gene Therapy #LX2006 Positively Impacts Cardiac Biomarkers of #Friedreich Ataxia, Clinical Data Show LX2006 shows safety, tolerance, and significant improvements in cardiac biomarkers in #FA cardiomyopathy, progressing to higher dose cohorts.

Today, we’ll likely see Pfizer’s Beqvez (or another commercial name) get approved for Hemophilia B. + Recall, Hemgenix from CSL is already approved in Hemophilia B as of November 2022. + The addition of Pfizer’s gene therapy will make for the third rare disease category with more than one gene therapy available – the others being Sickle Cell Disease (Lyfgenia and Casgevy) and Transfusion Dependent Beta Thalassemia (Zynteglo and Casgevy). + Competition within the same indication already exists in cell therapy in oncology. + Unlike both of the above-named categories where a CRISPR-based therapy is competing with an AAV-based therapy, both Heme B products will be AAV-based (virally delivered). More to come from Pfizer.... #zolgensma #cgt #genetherapy #hemophilia #hemgenix #csl