Phoenix-Micron, Inc. 的动态

News alert ?? Researchers have developed a highly efficient gene editing therapy that could potentially treat Stargardt disease, the most common form of inherited macular degeneration. This groundbreaking study,?published in Nature Medicine today, demonstrates the therapeutic use of a precision gene editing technology called base editing. #womeninoptometry #womeninmedicine #optometry #eyecare

#SpeakingofScience: Groundbreaking studies in the New England Journal of Medicine reveal the potential of exa-cel #genetherapy for #betathalassemia and #sicklecelldisease. According to recent #medicalresearch, 90% of beta thalassemia patients stopped needing transfusions with exa-cel gene therapy, and 97% of sickle cell patients were free from pain crises. These results mark a potential cure, significantly improving quality of life. The next challenge is ensuring equitable access to these costly treatments!???????? #RareDiseases #BioTech #HealthcareInnovation #MedTech #GeneticResearch #ClinicalResearch #EqualAccess #HealthAccess #HealthEquity https://lnkd.in/eRvGXq5A

Axonis Therapeutics is making strides in neurological treatments by leveraging the microgravity environment of the International Space Station to develop 3D brain organoids. Co-founder Shane Hegarty highlights how these groundbreaking advancements have helped de-risk the company's gene therapy and attract investors, contributing to their $115M oversubscribed Series A financing. West Orlando News https://lnkd.in/eSBff8B6 #lifesciences

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.

Cardiac gene therapy is offering new possibilities in treating genetic heart diseases, bridging molecular innovation and clinical translation. A transformative step for precision medicine. #GeneTherapy #Cardiology #Innovation European Society of Cardiology https://lnkd.in/eKWbnEn8

Familial dysautonomia (FD) is a rare neurodevelopmental and neurodegenerative disease stemming from a mutation in the ELP1 gene, causing visual impairment due to retinal ganglion cell (RGC) death. Two therapeutic approaches, gene replacement therapy and small molecule splicing modifiers, show promise in rescuing RGCs in mouse models, potentially paving the way for future treatments in FD patients. #FamilialDysautonomia #Neurodevelopmental #Neurodegenerative #GeneticDisorder #MedicalResearch #RareDisease #GeneTherapy #PreclinicalResearch #SplicingModifiers #ELP1Gene https://lnkd.in/eaMDVAJd

Current gene therapies for restoring progranulin (PGRN) in the CNS face limitations due to poor brain exposure and distribution. To address this, scientists developed an adeno-associated virus (AAV) targeting the liver to achieve sustained peripheral expression of a brain-penetrant PGRN variant (AAV(L)) in two mouse models of frontotemporal lobar degeneration with TDP-43 pathology (FTLD-GRN). Using this method, they avoided the issues associated with CNS-administered AAVs and maintained PGRN levels in the brain. Their results demonstrated that AAV(L) treatment reduced multiple FTLD-GRN pathologies, including motor deficits, TDP-43 phosphorylation, protein degradation issues, lipid metabolism problems, gliosis, and neurodegeneration. In vitro models using human induced pluripotent stem cell (hiPSC)–derived microglia and neurons showed similar improvements. These findings provide preclinical evidence of the concept of using an AAV platform to treat FTLD-GRN and other CNS disorders. Visit us at https://treventis.com/ #genetherapy #adenoassociatedvirus #TDP43 #stemcell #microglia https://lnkd.in/e59Kxp-C

#glaucoma #genetherapy #neuropathy The concept of gene therapy for glaucoma: the dream that has not come true yet https://lnkd.in/g6M4z5Dm Adrian Smedowski MD, PhD, FEBO Medical University of Silesia Gene therapies, despite of being a relatively new therapeutic approach, have a potential to become an important alternative to current treatment strategies in glaucoma.??Since glaucoma is not considered a single gene disease, the identified goals of gene therapy would be rather to provide neuroprotection of retinal ganglion cells, especially, in intraocular-pressure-independent manner.??The goal of this review is to summarize the current state-of-art and the direction of development of gene therapy strategies for glaucomatous neuropathy.

??Wondering why #mRNA delivery to solid tumors via #LNPs is so difficult (apart from dosing issues)? ?Gong et al. led by Drew Weissman, Wei Guo and Michael J. Mitchell from University of Pennsylvania in Nature Materials (pub. 2nd Sep, Link in comments) identified another hurdle for nanoparticles targeting solid tumor tissue. ?Content Gong et al. show that tumour cell-derived small extracellular vesicles (sEVs) hamper nanoparticle delivery to tumours. These bind to nanoparticles entering tumour tissue and direct them to liver Kupffer cells for degradation. Knockdown of a gene that controls sEV secretion (Rab27a) decreased sEV levels and enhances nanoparticle accumulation in tumour tissue. Co-encapsulation of mRNA encoding tumour suppressing/proinflammatory proteins together with Rab27a targeting siRNA enhanced therapeutic efficacy. ? Takeaway Tumour cell-derived sEVs and associated systems could be a potential target for improving nanoparticle-based tumour therapies. Follow me for more breaking content in the Gene Delivery and Cell & Gene Therapy field.

#innovativetreatment #research #nanomedicine #health #LNPs ???? Precision medicine is now turning to thio-lipid #nanoparticles (LNPs) against cystic fibrosis and vision loss. Thanks to NANO Magazine for this excellent article that highlights research on #thiolipids that can encapsulate genetic drugs, including messenger RNA (mRNA) and CRISPR-Cas9 gene editors. This encapsulation is a crucial step in delivering these therapies directly to affected cells, providing the opportunity to treat and potentially cure rare genetic diseases.