AVA-101 / Adverum Biotech - LARVOL DELTA

Subretinal Gene Therapy for Treatment of Retinal and Choroidal Vascular Diseases. (PubMed, Am J Ophthalmol) - "This review article discusses investigational subretinal gene therapies for retinal vascular diseases, including AVA-101, an adeno-associated viral (AAV) 2 vector expressing soluble vascular endothelial growth factor (VEGF) receptor 1, ABBV-RGX-314, an AAV8 vector expressing an anti-VEGF-A antibody fragment, and EXG102-031, an AAV8 vector expressing a recombinant protein that blocks VEGF family members and angiopoietin 2. In contrast, subretinal injection of some doses of ABBV-RGX-314 have shown evidence of controlling exudation and the maintaining vision, as well as safety and tolerability, leading to two ongoing pivotal trials comparing subretinal delivery of two different doses of ABBV-RGX-314 versus intravitreal injections of 0.5mg ranibizumab or 2mg aflibercept. These preliminary results are an encouraging and welcome development in the search for efficacious, long-duration treatments for retinal vascular diseases."

Gene therapy • Journal • Age-related Macular Degeneration • Gene Therapies • Inflammation • Macular Degeneration • Ophthalmology • Retinal Disorders

Intraocular VEGF deprivation induces degeneration and fibrogenic response in retina. (PubMed, FASEB J) - "To study mechanisms for VEGF antagonism-associated adverse effects in visual system, we intravitreally delivered recombinant adeno-associated virus-mediated expression of soluble Fms-related tyrosine kinase-1 (rAAV.sFLT-1), the extracellular domain of VEGF receptor, and analyzed the morphology and functions of retinal tissue...Moreover, high-throughput RNA sequencing analysis suggests that VEGF antagonism activates retinal degeneration, inflammation, and other adverse effects. Taken together, our findings have shed light on pathogenic mechanisms for VEGF antagonism-associated adverse effects and potential therapeutic targets.-Xiao, M., Liu, Y., Wang, L., Liang, J., Wang, T., Zhai, Y., Wang, Y., Liu, S., Liu, W., Luo, X., Wang, F., Sun, X. Intraocular VEGF deprivation induces degeneration and fibrogenic response in retina."

Journal • Fibrosis • Immunology • Ocular Inflammation • Ophthalmology

Three-year follow-up of Phase 1 and 2a rAAV.sFLT-1 subretinal gene therapy trials for exudative age related macular degeneration. (PubMed, Am J Ophthalmol) - "Given the small number of patients this study was unable to unequivocally confirm the existence of a biological efficacy signal, however it confirmed that rAAV.sFLT-1 gene delivery was well tolerated among the elderly."

Journal • P1 data

Avalon GloboCare Provides Updates on its Lead Clinical Programs in CAR-T Therapy and Exosome-Based Regenerative Therapeutics (GlobeNewswire, Avalon GloboCare Corp.) - Next-generation, transposon-engineered, multi-targeted CAR-T candidate, AVA-101, has entered process development and validation phase; expect to launch first-in-human trial in Q1 2020. Accelerate clinical development of exosome-based oncology and regenerative therapeutic programs, AVA-201 and AVA-202. FREEHOLD, N.J., July 15, 2019 (GLOBE NEWSWIRE) -- Avalon GloboCare Corp (NASDAQ: AVCO), a leading global developer of cell-based technologies and therapeutics, today announced an update on its four clinical programs in cellular therapy, including AVA-001 and AVA-101, that leverage individualized CAR (Chimeric Antigen Receptor) T-cell therapy for immuno-oncology, as well as AVA-201 and AVA-202 exploring novel development with stem cell derived exosomes.

Clinical • New trial • Preclinical

Avalon GloboCare Announces Filing of Provisional Patent Applications for AVA-101, a Novel Transposon-Based, Multi-Targeted CAR-T Therapy (GlobeNewswire, Avalon GloboCare Corp.) - "Avalon GloboCare Corp...announced today that the Company and Arbele Limited (joint venture partner) have jointly filed provisional patent applications covering method and composition of matter claims for AVA-101, a novel multi-targeted, transposon-based Chimeric Antigen Receptor (CAR)-T cellular immunotherapy...This set of patent applications primarily involves an innovative, versatile and inducible transposon-based vector system composed of humanized CD19 and CD22 dual and bispecific CARs with a safety switch targeted by Rituximab."

Patent