Neurophth Therapeutics / 纽福斯生物科技有限公司’s Post

Intellia Therapeutics has unveiled strong clinical data showing the feasibility of redosing their CRISPR-based therapy, NTLA-2001, using a LNP system. Administering a second, higher dose resulted in a significant 90% median reduction in serum TTR levels among patients with transthyretin amyloidosis. While redosing isn't currently planned for this program, the findings suggest potential benefits for future therapies. YolTech's proprietary in vivo gene editing therapy, YOLT-201, also utilizes LNP delivery system to achieve efficient TTR gene knockout in patients. In precious investigator-initiated trial (IIT), all patient enrollments have been completed, showcasing excellent clinical data with significant preliminary efficacy and safety. Stay tuned for more good news. #GeneTherapy #CRISPR #LNP #ClinicalData #Biotechnology #HealthcareInnovation #YolTech #IntelliaTherapeutics

Leaving the snow behind in Reno for a few days and heading to Palm Springs to attend the prestigious Macula Society 2024 meeting! Thrilled to be author/co-author on 5 abstracts. I am honored to present the FIRST time interim results from the phase 2 LUNA study: Day and Date: Thursday, February 08, 2024 Session: AMD II (Neovascular Trials) Presentation Title: Ixoberogene soroparvovec (Ixo-vec) Intravitreal Gene Therapy for Neovascular Age-Related Macular Degeneration: Preliminary Results from the Phase 2 LUNA Study Presentation Time: 7:57 AM - 8:04 AM In addition, I am thrilled to be a co-author on 4 other exciting talks: 1. The DAVIO2 Trial: A Phase-2 Multicenter Study of A Single Injection of EYP-1901 vs Aflibercept for Wet Age-Related Macular Degeneration - presented by Rishi Singh 2. Greater Reduction in Pigment Epithelial Detachment Size With Faricimab vs Aflibercept During Head-to-Head Dosing in Patients With nAMD - presented by Robert Avery 3. The Efficacy of Avacincaptad Pegol in Geographic Atrophy: 24-Month Results from GATHER2 - presented by Christina Weng 4. The Safety and Tolerability of Avacincaptad Pegol in Geographic Atrophy: 24-Month Results from GATHER2 - presented by Lejla Vajzovic, MD, FASRS Looking forward to seeing everyone in Palm Springs soon!! Adverum Biotechnologies EyePoint Pharmaceuticals Roche Genentech Iveric Bio, An Astellas Company #clinicaltrials #genetherapy #sustaineddelivery #clinicalresearch

Casgevy, a personalized gene therapy utilizing CRISPR/Cas9, edits a patient's blood stem cells, specifically targeting the BCL11A gene to enhance functional hemoglobin production. This one-time treatment involves mobilizing and infusing modified bone marrow stem cells, showcasing the potential of CRISPR/Cas9 in drug and clinical development. I am very fortunate to have received training in using this novel technology and am moving forward with my research project. #crisprcas9 #geneediting #drugdevelopment #cancerresearch #cancertreatment

#LegendCapital #PortfolioNews Our portfolio Pharmaron, in collaboration with Complement Therapeutics and Cell and Gene Therapy Catapult, has been awarded over £1 million in an Innovate UK Grant to support the "AAV Production Process Intensification using Perfusion Bioreactor Technology" project. This initiative, funded over the next two years, aims to enhance the efficiency and cost-effectiveness of AAV manufacturing, addressing the rising demand for gene therapies. Pharmaron's established expertise in biomanufacturing will play a critical role in advancing this project, which is expected to improve access to innovative treatments for patients. We anticipate the significant contributions this collaboration will make to the pharmaceutical landscape. #GeneTherapy #InnovateUK #PharmaceuticalInnovation #AAV

Heading to the 3rd LNP Development Summit Europe in Berlin? Join us from September 25 – 26 to exchange insights and perspectives on advancing RNA-LNP technology, including these interactive sessions and poster presentations: - Optimizing scale-up to GMP manufacturing of RNA-LNPs - Gene editing in CD34+ hematopoietic stem and progenitor cells using non-viral LNPs - Effect of LNP components on mRNA-mediated protein expression of LNPs in different organs #mRNA #LNPs

Heading to the 3rd LNP Development Summit Europe in Berlin? Join us from September 25 – 26 to exchange insights and perspectives on advancing RNA-LNP technology, including these interactive sessions and poster presentations: - Optimizing scale-up to GMP manufacturing of RNA-LNPs - Gene editing in CD34+ hematopoietic stem and progenitor cells using non-viral LNPs - Effect of LNP components on mRNA-mediated protein expression of LNPs in different organs #mRNA #LNPs

The safety and efficacy of a #CRISPRCas9-based gene editing therapy, EDIT-101, has been evaluated in patients with CEP290-associated inherited retinal degeneration.   Results of the BRILLIANCE study demonstrated that EDIT-101 is safe and showed that patients exhibited improved vision and light sensitivity with 79% showing improvement in at least one efficacy outcome, and 43% improved in two or more outcomes.   Providing proof of concept for the therapeutic potential of in vivo retinal gene editing, the study supports further research into CRISPR/Cas9-mediated therapies for other inherited retinal degenerations as well as other previously incurable #blindness.   Eye diseases and vision disorders are some of the most common conditions in the world. Our family of licensees recently expanded with the addition of StemSight - Vision Unlimited – a biotechnology company using CRISPR/Cas9 to develop off-the-shelf cell therapies for unmet medical needs in corneal blindness.   Read the full results from the BRILLIANCE study at NEJM Group: https://shorturl.at/JuW1e    #EyeDiseases #RetinalDegeneration

Heading to the 3rd LNP Development Summit Europe in Berlin? Join us from September 25 – 26 to exchange insights and perspectives on advancing RNA-LNP technology, including these interactive sessions and poster presentations: - Optimizing scale-up to GMP manufacturing of RNA-LNPs - Gene editing in CD34+ hematopoietic stem and progenitor cells using non-viral LNPs - Effect of LNP components on mRNA-mediated protein expression of LNPs in different organs #mRNA #LNPs

Heading to the 3rd LNP Development Summit Europe in Berlin? Join us from September 25 – 26 to exchange insights and perspectives on advancing RNA-LNP technology, including these interactive sessions and poster presentations: - Optimizing scale-up to GMP manufacturing of RNA-LNPs - Gene editing in CD34+ hematopoietic stem and progenitor cells using non-viral LNPs - Effect of LNP components on mRNA-mediated protein expression of LNPs in different organs #mRNA #LNPs

Heading to the 3rd LNP Development Summit Europe in Berlin? Join us from September 25 – 26 to exchange insights and perspectives on advancing RNA-LNP technology, including these interactive sessions and poster presentations: - Optimizing scale-up to GMP manufacturing of RNA-LNPs - Gene editing in CD34+ hematopoietic stem and progenitor cells using non-viral LNPs - Effect of LNP components on mRNA-mediated protein expression of LNPs in different organs #mRNA #LNPs