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Rationally designed chimeric AAV capsids for enhanced immune evasion, cellular uptake, and transduction efficiency

Rationally designed chimeric AAV capsids for enhanced immune evasion, cellular uptake, and transduction efficiency

Unmet Need Adeno-associated virus (AAV) vectors are a leading platform for in vivo gene therapy, yet their clinical potential is limited by key biological barriers. Pre-existing immunity to common AAV serotypes diminishes vector efficacy and…

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Chimeric AAV vectors enabling antibody evasion and redosing for cardiac gene therapy

Chimeric AAV vectors enabling antibody evasion and redosing for cardiac gene therapy

Unmet Need Gene therapy using adeno-associated virus (AAV) vectors is severely limited by pre-existing immunity in 30–80% of adults and by the development of neutralizing antibodies after initial treatment, preventing redosing. There is a critical…

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RNA switch enabling stress-responsive regulation of protein expression for safer, more consistent gene and mRNA therapies

RNA switch enabling stress-responsive regulation of protein expression for safer, more consistent gene and mRNA therapies

Unmet Need Current gene and mRNA therapies lack mechanisms to regulate protein expression in response to intracellular stress. Overexpression of therapeutic proteins can induce ER stress leading to unfolded proteins that do more harm than…

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Novel AAV capsid variants for CNS-targeted gene therapies

Novel AAV capsid variants for CNS-targeted gene therapies

Unmet Need The development of effective gene therapies for central nervous system (CNS) diseases faces a critical bottleneck: current adeno-associated virus (AAV) vectors lack sufficient transduction efficiency and specificity for human brain cells. Standard AAV…

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Methods and compositions for enhancing viral and mRNA gene therapy with engineered RNA elements

Methods and compositions for enhancing viral and mRNA gene therapy with engineered RNA elements

Unmet Need An outstanding challenge in creating effective gene therapies, particularly those based on adeno-associated virus (AAV) and mRNA delivery platforms, is overcoming sub-optimal transgene expression. Inefficiencies in translation and stability of therapeutic gene products…

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Muscle-targeted gene therapy for SLC25A4 mitochondrial deficiency

Muscle-targeted gene therapy for SLC25A4 mitochondrial deficiency

Unmet Need There is a significant unmet need for disease-modifying therapies that address mitochondrial dysfunction in inherited cardiomyopathies and myopathies such as SLC25A4 deficiency. Existing treatments are limited to supportive care and heart transplantation, with…

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A novel all-in-one AAV system for efficient epigenome editing in neurodegenerative diseases

A novel all-in-one AAV system for efficient epigenome editing in neurodegenerative diseases

Unmet Need Neurodegenerative diseases such as Alzheimer’s and Parkinson’s affect over 55 million people worldwide, with prevalence expected to double by 2050 due to aging populations. These conditions are progressive, debilitating, and currently have no…

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Permanent blood pressure control through liver-directed gene editing

Permanent blood pressure control through liver-directed gene editing

Unmet Need The World Health Organization estimates that high blood pressure (hypertension) results in approximately 9 million annual deaths worldwide. While current medications can lower blood pressure, they are limited by the need for lifelong…

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Gene therapy for ATP1A3-Related Neurologic Disorders

Gene therapy for ATP1A3-Related Neurologic Disorders

Unmet Need Alternating Hemiplegia of Childhood (AHC) is a rare neurologic disorder affecting children, causing episodes of temporary paralysis, dystonia, and severe behavioral issues, with prevalence estimates ranging from 1 in 1 million to 1…

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Novel epigenome editors for multiplexed gene regulation

Novel epigenome editors for multiplexed gene regulation

Unmet Need Epigenome editors activate or inhibit gene expression without modifying the underlying DNA sequence. Consequently, these editors can reversibly fine-tune gene expression without the risk of permanently altering the genome or off-target editing. Epigenome…

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Splice switching oligonucleotides to restore PHKG2 expression in glycogen storage disease IX

Splice switching oligonucleotides to restore PHKG2 expression in glycogen storage disease IX

Unmet Need Glycogen Storage Disease IX (GSD IX) is a rare inherited disorder primarily caused by mutations in the PHKG2 gene, leading to significant deficiencies in glycogen metabolism. Patients typically present with a variety of…

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Novel Method for Creating and Modifying Stable Cell Lines for Enhanced Proteomic Analysis

Novel Method for Creating and Modifying Stable Cell Lines for Enhanced Proteomic Analysis

Unmet Need Characterizing the composition and function of protein complexes can facilitate the understanding of key cellular processes. Indeed, the global proteomics market was estimated to be USD 25.9 billion in 2021. However, the analysis…

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