Lexeo Therapeutics’ Post

Check out this on-demand webinar on long-term follow-up (LFTU) considerations for cell and gene therapy studies featuring our colleagues from Thermo Fisher Scientific as well as industry experts.

#ICYMI: Last month, LEXEO received IND clearance from the U.S. FDA to proceed testing our investigational AAVrh10 gene therapy, LX2020, in clinical trials. LX2020 is designed to address arrhythmogenic cardiomyopathy due to plakophilin 2 pathogenic variants (PKP2-ACM).    Learn more about the condition and LEXEO’s approach: https://bit.ly/3ruqT5z  

Today we announced 12-month safety and efficacy data from the ongoing Phase I/II clinical trial of ATSN-101, our investigational #GeneTherapy for GUCY2D-associated #LeberCongenitalAmaurosis (#LCA1). ATSN-101 continues to demonstrate clinically meaningful improvements in vision at the highest dose and is well-tolerated 12 months post-treatment.   We are pleased that the 12-month data have been accepted for presentation at the 47th Annual Macula Society Meeting, which will be held February 7-10, 2024, in Palm Springs, CA.   These encouraging data and the recent #RMAT designation underscore the potential of our subretinal gene therapy to fill a significant unmet need within the LCA community.   Read more: https://lnkd.in/e5rNk-AA  

BioMarin's gene therapy for #Hemophilia A has at last landed in Europe. However, the concerning gap between EMA's green light and the first patient's treatment initiation is sending shockwaves. While the Phase 3 success was commendable, the slow start after approval raises an urgent query about access to innovative therapies. Time is of the essence in medical progress, particularly for those in critical need. Can we afford to dawdle in delivering hope? To the payers we ask, in a world where a year can redefine lives, how will you redefine speed in patient access? Time ticks, and lives await. #genetherapy #marketaccess #accesstocare BioMarin Pharmaceutical Inc.

Neurogene Expands Low-Dose Cohort of Rett Syndrome Gene Therapy Trial, Adds High-Dose Cohort. The first and second cohorts of the Phase I/II trial of the experimental therapy NGN-401 will enroll patients in parallel with one another. http://ow.ly/NxHL105kCvs

Check out this on-demand webinar on long-term follow-up (LFTU) considerations for cell and gene therapy studies featuring our colleagues from Thermo Fisher Scientific as well as industry experts.

💫 ONCOnews 💫 🌟 Update on Phase 3 Study of Investigational Gene Therapy for Ambulatory Boys with Duchenne Muscular Dystrophy 👉 The overall safety profile of fordadistrogene movaparvovec in the CIFFREO trial was manageable, with mostly mild to moderate adverse events, and treatment-related serious adverse events generally responding to clinical management 🔗 https://buff.ly/3Vp9Zk4 #OncEd #ONCOnews #OncoAlert

Join us at ISCT 2024 in Vancouver for a pivotal roundtable discussion from May 29 to June 1: 'Debate, Deliberate & Drive Solutions - Preparing for AI: Anticipating Regulatory Challenges'. Dive deep into the future of AIs with industry leaders and innovators.

#podcast Nolan Townsend, CEO of Lexeo Therapeutics, and Jen Farmer, CEO of FARA, the Friedreich's Ataxia Research Alliance, are working together to better understand and treat Friedreich's Ataxia. This rare genetic condition includes cardiomyopathy and scoliosis. Lexeo Therapeutics is studying gene therapy as a potential treatment for the cardiovascular component of the disease. The Friedreich's Ataxia Research Alliance (FARA) plays a role in funding research, understanding the natural history of the disease, and advocating for treatments. #FriedreichsAtaxia #CureFA #FAAwarenessMonth #FAAwarenessDay #RareDiseases  LexeoTX.com CureFA.org https://lnkd.in/g94V2-Pz

Less than a week to go for #EUCROF24! CROs are telling us sponsor study protocols are becoming increasingly complex and sample-intensive. What we often hear: 🏨 More sites, more patients, and strict timelines... ..... Will the kits be customized for study needs? ..... Will sites have the kits in time for FPI? ..... Are the kits easy to use? What if the site makes collection errors? 🔍 More endpoints and specialized testing. It's hard to find the right labs... ..... Is there a lab near the site to process short-stability samples in<24h? ..... Can the lab develop and validate this biomarker assay on-time? ..... Can the lab do the testing according to protocol? ✈ Thousands of samples scattered across sites, labs, and with couriers... ..... Where is sample X? Is it collected? ..... Do I have sample visibility? How will I keep the sponsor informed? ..... Can I ensure sample & data integrity? 📊 Millions of data points generated - from disparate data sources... ..... When and how will data be transferred? What about ad hoc transfers? ..... Will I have a unified dataset in time for submission? ..... Is the data accurate, consistent and usable? Sounds familiar? Stop by our booth #4 if you're open to learning more about how we help CROs and sponsors generate endpoint data for their complex trials - while preventing delays and reducing costs. #clinicalresearch #clinicaltrials #clinicaloperations