Modified Arrestin-1 to Enhance Photoreceptor Survival in Retinal Disease
Modified Arrestin-1 delivered to retina with AAV Modified Arrestin-1 binds enolcase-1 without reducing inhibitory effect of enolase-1, an advantage over native protein Slows retinal degeneration, slows loss of photoreceptor ERG function, improves photoreceptor survival Murine model of retinitis pigmentosa
A Novel Therapeutic for the Treatment of Lewy Body Diseases (Parkinson’s, Alzheimer’s)
Adoptive cellular therapy vaccine Activates immune system to reduce misfolded proteins Early-stage use prevents development of severe disease No auto-immune response
Personalized Slow-Cycling Tumor RNA-Based Nanoparticle Vaccine to Treat Cancer
For the treatment of resistant cancers Universal vaccine for various tumors Engaging the patient’s immune system Overcoming intra-tumoral and systemic immune suppression
Re-Establishing Immune Tolerance Using for Multiple Sclerosis
Re-establishes immune tolerance, reducing need for long-term therapies and increasing patient comfort and ease of treatment Enables the patient’s own immune system to generate the unique immune regulatory T-Cells specifically needed, permitting a universally applicable treatment AAV gene therapy to have the body make the specific regulatory T-cells needed to regulate immune tolerance MS Mouse […]
Micro-Utrophin/Dystrophin Chimeric Constructs
DMD AAV gene therapy Different mutations and deletions in dystrophin can cause a patient to have an immune response against expressed micro-dystrophin These dystrophin chimeras are designed to avoid neoantigen presentation for a wide range of known dystrophin mutations, reducing potential immune response
AAV Capsid Variants for Gene Therapy
Novel AAV variants that alter the tropisms and improve the transduction efficiency Targeting of skeletal and cardiac muscle over liver Transfection efficiency improved; lower dose required NHP study beginning soon
Countering Obesity in Companion Animals and People with Gene Therapy
Combines administration of GDF15 and a myostatin inhibitor, treating obesity while preventing loss of muscle mass Mice lose adipose tissue while maintaining lean mass, making it a potential therapy for companion animals and humans Different vectors can serve as delivery vehicles, including adeno-associated viral (AAV) vectors
Compositions, Methods and Expertise in Designing and Improving Capsid Variants Targeting Specific Therapeutic Targets
Methods of capsid design and directed evolution Capsid libraries to AAV3B and AAV5 Immune Evasion Capsid variants for AAV3B, AAV9, and AAVrh.10 Capsid variant technologies that targeting human glioblastoma cells and improve cellular transduction
Engineering AAV Vectors With Improved CNS Targeting
AAV capsids expressing protein that attaches to neuronal cell adhesion molecule-bound proteins In vitro data available for AAV1 serotype; technology applicable to other serotypes