Shoaib Ugradar, MD’s Post

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

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

The recent free access publication "Gene and cell therapy for age-related macular degeneration: A review" offers a comprehensive overview of the progress in treating AMD, from the introduction of intravitreal anti-VEGF therapy to the recent approval of pegcetacoplan for geographic atrophy. The next frontier? Gene and cell therapies for age-related macular edema. A very good overview of current approaches published in the journal "Survey of Ophthalmology". https://lnkd.in/dBDj4Nt4 #Retinology #AMD #Ophthalmology #MedicalAdvancements"

The Division of Ophthalmology at Lurie Children's is the first in Illinois to become a surgical site for LUXTURNA®, the first gene therapy approved in the U.S. to treat patients with a form of vision loss that can result in blindness. The treatment, which improves vision for patients with a hereditary retina degeneration, was administered by Dr. Safa Rahmani and an all-female team of pediatric eye experts. https://lnkd.in/gHm-_Mc5 #ophthalmology #womeninmedicine #genetherapy #pediatricmedicine

Retina World Congress Recap! Earlier this month, Dr. Carl Regillo, Director of Wills Eye Hospital Retina Service, presented preliminary data from the Phase 2 LUNA Study and three-year results from the Phase 1 OPTIC Extension Trial of Ixo-vec (ixoberogene soroparvovec) intravitreal gene therapy for #nAMD. According to Dr. Regillo, the OPTIC long-term extension study demonstrated that a single IVT injection of Ixo-vec provides a durable clinical benefit and substantially reduces treatment burden for at least three years. The study showed an 84%-95% reduction in annualized anti-VEGF injections, with 53%-73% of participants remaining supplemental injection-free in year three. The LUNA Phase 2 study indicated that BCVA was maintained and CST was reduced and remained stable at both Ixo-vec doses through week 26. There was a ≥90% reduction in annualized anti-VEGF injections in both dose groups, and 85%-68% of patients remained free of injections at six months at the 2x10^11 and 6x10^10 doses, respectively. A prespecified interim analysis will be conducted when all participants complete their 26-week study visit. #retinaworldcongress #genetherapy #ixovec #retina Healio | Ocular Surgery News #lunastudy Retina World Congress Financial Disclosures: Dr. Regillo is a consultant for Adverum Biotechnologies References:  Regillo, C. ADVM-022 Intravitreal Gene Therapy for nAMD: Preliminary Data from the Phase 2 LUNA Study and 3-Year Results from the Phase 1 OPTIC Extension Trial. Presented at: Retina World Congress; May 9-12, 2024; Fort Lauderdale, Florida.

Ophthalmology researcher Joseph Arboleda-Velasquez, MD, PhD, is working toward a future without Alzheimer's. His recent discovery of a mutation in the reelin gene, a protein that may protect those at risk for the disease, is offering new hope. Learn more about Dr. Arboleda-Velasquez's groundbreaking research and how it's shaping the fight to #ENDALZ.

The future of back pain treatment is here - Nanocarriers Deliver Gene Therapy Scientists have made a big step forward in treating chronic back pain. They used tiny, engineered particles called extracellular vesicles (EVs) carrying the gene FOXF1 to repair damaged spinal discs in mice. This new, minimally invasive approach could not only help with back pain but also other joint problems. More research is needed, but the results so far are very promising. Imagine a future where back pain can be healed from within, without surgery. For more information on new nanocarriers and spinal disc regeneration, click on this link: https://lnkd.in/gezSq8_i #PaddaInstitute #PainMD #NewNanocarriers #SpinalDiscRegeneration #Nanocarriers

Exploring preclinical ophthalmology research: Your FAQs Answered! (Episode 1)🎬 Traditional treatments for ophthalmic diseases have limitations, making gene therapy a promising avenue. Yet, challenges persist in preclinical research. Cyagen's Ophthalmic Gene Therapy Platform is here to help. We tackle your FAQs on mouse models in our FAQ series. Stay tuned for insights on retinitis pigmentosa, congenital nystagmus, AMD, and total color blindness. Our TurboKnockout and CRISPR-Pro models support basic research and drug development, with personalized customization options. 👁️Check out our preclinical ophthalmology research solutions here: https://lnkd.in/g7gcqsU2 Let's illuminate the path to innovative solutions in ophthalmology! 🚀 #ophthalmology #genetherapy #MouseModels #HUGOGT #genetherapies #preclinical #preclinicalresearch #ophtalmologie #ophthalmic #science #researchimpact #biotechnology #biotech

🌟Join us at ARVO 2024 to Advance Your Ophthalmology Pipelines!🌟 We're gearing up for ARVO 2024, happening in Seattle from May 5th to 9th. Mark your calendars and make sure to swing by our booth #1243. Meet our experts, Elisabet Mateu, Commercial Director, and Vincenzo Di Donato, Scientific Director, who will be on hand to showcase how the zebrafish model can accelerate your pipeline in ophthalmology. Here are a few reasons to stop by and explore the zebrafish model: ✅ Readily assess visual function in a live organism. ✅ Swiftly validate gene therapy constructs. ✅ De-risk clinical trials with comprehensive efficacy data. ✅ Empower your findings with our High-Throughput Screenings, adhering to the principles of the 3Rs. ⏱ Access disease models promptly and undergo thorough phenotypic screening, including molecular profiling, histology, and visually driven behavior. Don't let this opportunity slip by! Discover how zebrafish can revolutionize your ophthalmology research at #ARVO2024. We can't wait to see you there! Are you planning to attend the Congress? Contact us to arrange a personal meeting 👉 https://lnkd.in/dpQGHRz #zeclinics #zebrafish #ophthalmologyresearch

Check out this insightful article from Eyes On Eyecare’s At a Glance, featuring highlights from Octane’s Ophthalmology Tech Forum 2024. @Sergio Rossi, Managing Director and Partner at Boston Consulting Group (BCG), provides a detailed analysis of the current state of the ophthalmic market, emphasizing the transformative potential of genetic engineering and cell transplantation. These cutting-edge advancements are set to revolutionize patient care and pave the way for new innovations in eye health. The discussion explores the fast-evolving landscape of gene therapies, CRISPR technology, and the crucial impact these innovations will have on meeting unmet needs in ophthalmology. As we anticipate OTF 2025, now is the perfect time to secure your spot with early pricing and be part of the future of eye care. Read more here: https://bit.ly/3Aw7mGw Registration is now available for 2025! #OTF2024 #InvestmentOpportunities #EyesonEyecare