Endogena Therapeutics, Inc.’s Post

Everyone wants to know which pathway to target in Thyroid Eye Disease (TED). We (with Raymond Douglas MD) performed an unbiased analysis of gene expression in patients with TED and found the IGF-1R pathway plays a central role in all the phenotypes we see in TED. This groundbreaking research was presented at the American Academy of Ophthalmology. #TED #ThyroidEyeDisease #Research #GeneExpression #IGF1RPathway #AAO #Ophthalmology #MedicalResearch #AMGEN

Today’s Medical Science & Tech Digest delivers a group of absorbing articles covering three distinct topics. Please note: These articles are shared for informational purposes only. The first piece discusses the importance of anesthetics and recent efforts toward improving how much is administered for surgery. A team of researchers conducted a number of trials which partly involved a device that combines brain-monitoring medical equipment with a computer that uses algorithms to determine how the body processes propofol. https://lnkd.in/efscm389 The second article revolves around sickle cell disease, an inherited blood disorder that causes the body’s red blood cells to become misshapen and not last as long as healthy cells. Recently, the United Kingdom became the first country to approve the use of the world’s first gene therapy for treating sickle cell disease and a specific type of sickle cell disease called beta-thalassemia. https://lnkd.in/gxHcTy4H? The third article is concerned with new developments by researchers regarding the human nervous system and its relation to cancer. As mentioned, these researchers believe understanding this interplay and how certain anti-depressant medications factor in may strengthen efforts against some types of cancer. https://lnkd.in/gArVrhZU #manhattanlife #medicalscience #medicaltechnology #medtech

🔬 MINI-RETINAS: TRANSFORMING THE STUDY OF RETINAL DISEASES 👀 Retinal diseases like age-related macular degeneration (AMD) and retinitis pigmentosa are major contributors to global vision loss. Conventional research models have limitations, hindering our progress in understanding these conditions and developing treatments. Researchers have made a groundbreaking advancement by cultivating mini-retinas in the lab. These "retinas in a dish" replicate the intricate structure and functionality of human retinas, opening doors to various transformative applications: 🔍 Disease Mechanism Studies: Gain real-time insights into the development and progression of retinal diseases, uncovering their underlying mechanisms. 💊 Drug Testing and Development: Enhance drug screening accuracy and efficiency, fast-tracking the discovery of effective therapies while reducing reliance on animal models. 🩺 Personalized Medicine: Develop patient-specific mini-retinas from stem cells, facilitating tailored treatment approaches based on an individual's genetic composition. 🧬 Gene Therapy Research: Evaluate gene therapies targeting genetic defects causing retinal diseases using these innovative models. ☠️ Toxicology Studies: Evaluate retinal toxicity of new drugs, ensuring safety before advancing to clinical trials. This innovation not only helps advance vision science understanding but also expedites the journey to effective treatments. #Biotech #DrugDiscovery #VisionScience #Innovation #MedicalResearch #PersonalizedMedicine #GeneTherapy

Ep 126 - What Are the Latest Breakthroughs in Stem Cell Therapy for Treating Refractory Diseases? Uncover the latest breakthroughs in veterinary stem cell research dedicated to refractory cases, promising new hope and solutions for chronic conditions. Quick Summary: In this episode of the Vet Dental Show, we delve into exciting updates on stem cell research for refractory cases, particularly in treating feline stomatitis. Hosted by Brett Beckman, this session highlights groundbreaking work by Dr. Boaz Arzee and his team at UC Davis, exploring how recent advancements could revolutionize treatment options. https://lnkd.in/gsQ9C6dz

Ocular Gene Therapy in a Patient with Dystrophic Epidermolysis Bullosa - #NEJM Dystrophic epidermolysis bullosa is a rare genetic disease caused by damaging variants in COL7A1, which encodes type VII collagen. Blistering and scarring of the ocular surface develop, potentially leading to blindness. Beremagene geperpavec (B-VEC) is a replication-deficient herpes simplex virus type 1–based gene therapy engineered to deliver functional human type VII collagen. Here, we report the case of a patient with cicatrizing conjunctivitis in both eyes caused by dystrophic epidermolysis bullosa who received ophthalmic administration of B-VEC, which was associated with improved visual acuity after surgery. https://lnkd.in/egsP5KFn

Expanded early consumer genetic screening, coupled with early genetic intervention (e.g. Cell and gene therapy, #AAV, #Lentivirus, #CRISPR, #mRNA, #siRNA...) to "circumvent" chronic, "sooner or later" but irreversible, inheritable degenerative diseases? Here's a link to Mayo Clinic 's article to add on: https://lnkd.in/gcX44h-j Thanks to Frank Nocken for the sharing. 😊 #cgt #populationhealth #geneticscreening

Are you using validated retina tissue models of the retina to study efficacy and safety of innovative ophthalmology therapies? Using functionally validated retina tissue models that accurately mimic the physiology of the human retina removes any donor variation seen in ex vivo models and leads to shorter timelines to results ⏳ Whether it is disease modelling, retinal toxicity evaluation or gene therapy viral vector testing, our team has unrivalled expertise in providing robust data from human iPSC derived models of the retina 👁 Customised studies on the human retina models ensure an output of robust and comparative data, predictive of the human retina for confident key decision making in your drug discovery. After an extensive development phase, Newcells have completed numerous studies for customers. We can work with you to design the right study to provide data on large and small molecule effects on the retina that will assist you in lead candidate progression. Find out more: https://lnkd.in/esFBQV4V #RPE #oculardisease #retinaltherapies #genetherapy #ophthalmology

🎓 Scientific Highlight of the Week 🎓 We are thrilled to share insights from a groundbreaking study titled "Magnetically guided adeno-associated virus delivery for the spatially targeted transduction of retina in eyes." Adeno-associated viruses (AAV) are extensively researched for gene therapy applications, particularly in treating retinal diseases. A significant challenge in developing retinal gene therapies lies in controlling the specificity and delivery of AAV to selected areas of the retina. This study aimed to demonstrate that magnetic nanoparticles can be used to transport different AAVs across the retina and modulate the selective transduction of specific retinal layers or photoreceptor cells in ex vivo porcine retinal explants and whole eyes. Researchers brought the viruses close to the target cell layer and controlled their interaction time to trigger transduction. They showed that this magnetically guided approach for transporting AAV to selected areas and layers of the retina does not require specific system optimization. This innovative approach allows for controlled AAV transport and selective transduction of cellular systems. Researchers believe this technology can be applied to various other tissues or organs to selectively deliver genes of interest. Our product, FluoMag-V, played a pivotal role in this research. These fluorescently labeled magnetic nanoparticles are designed for magnetofection applications. This technology uses magnetic fields to magnetize nanoparticles in solution, forming a strong gradient to attract them and cover the entire plate surface (e.g., in vitro use). FluoMag-V is a formulation of magnetic nanoparticles aimed at enhancing the infection and transduction capabilities of viruses. 📚 Explore the full article ➡ https://bit.ly/4foSDgv 🔍 Explore more publications in our Citation Database ➡ https://bit.ly/49qRYah 🛒 Learn more about our FluoMag-V ➡ https://bit.ly/3A99NyE Congratulations to the researchers for this remarkable achievement! 🧬🔬 Seungkuk Ahn Oliver Siontas Janis Koester Jacek Krol Sascha Fauser Daniel J. Müller #ScientificResearch #Biotechnology #Innovation #GeneTherapy #Magnetofection

Leukoencephalopathy with Vanishing White Matter Disease (VWM) is an incurable white matter disease that results in rapid neurological degeneration, seizures, ataxia, and premature death in children. VWM is caused by mutation of a gene critical for mRNA translation and regulation of cellular stress response, eIF2B5, and disease pathology is driven mainly by astrocyte dysfunction. Allison Bradbury, PhD, and her team of experts at Nationwide Children's Hospital, in collaboration with the University of Utah, have developed a novel gene therapy that provides a wildtype copy of eIF2B5 via AAV9 delivery targeting astrocytes to compensate for loss of function mutations. Preclinical investigations (conducted in mouse models) show promising safety and efficacy of this therapy. Click here for more exciting details via Pediatrics Nationwide: https://bit.ly/3zfbuKf We are delighted to share that this technology (2020-072: Development of AAV Gene Therapy for eIF2B5 Related Vanishing White Matter Disease) is currently available for licensing! Please contact senior licensing associate Kyle Murrah, PhD, for more information. #GuidingIdeasToSolutions #InnovateWithUs #GeneTherapy #Research

Starting off on the right foot! Our national, multicenter cohort of #syndromicRP has just been published #openaccess in Graefe's Archive for Clinical and Experimental Ophthalmology (Springer Nature Group)👇 https://lnkd.in/d6tz2fGc This #collaborative effort involved 6 HCPs in Portugal and 122 patients from 100 Portuguese families. A total of 81 genetic variants were identified in 25 different #genes, 22 of which are novel. This has major implications in determining disease-related prognosis and providing targeted genetic counseling for #syndromicRP patients in Portugal. Telmo Cortinhal Cristina Moreira dos Santos Sara Vaz-Pereira Ana Marta Lilianne Duarte Vítor Miranda José F. Dias da Costa Ana Berta Sousa Virginie G. Peter Karolina Kamińska Carlo Rivolta Ana Luísa Carvalho Jorge Saraiva Célia Azevedo Soares Rufino Silva Joaquim Murta Maria Luisa Coutinho Santos