Skip to main content
Gene therapy for long-term treatment of GSDIa

Gene therapy for long-term treatment of GSDIa

Unmet Need Glycogen storage disease type Ia (GSDIa), or von Gierke disease, is a rare heritable metabolic disorder that impacts the liver and kidneys due to an inability to break down glycogen. An estimated 1…

Read More

Methods and compositions for treating protein aggregation diseases

Methods and compositions for treating protein aggregation diseases

Unmet Need Neurodegenerative disease affects more than 50 million people per year. Certain neurodegenerative diseases, like Alzheimer’s disease, result from the accumulation of amyloid-forming proteins in the brain, which results in cognitive impairment and has…

Read More

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…

Read More

Method for rapid regeneration of modifiable stable cell lines

Method for rapid regeneration of modifiable stable cell lines

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…

Read More

Blood pressure control by gene editing

Blood pressure control by gene editing

Unmet Need The World Health Organization estimates that high blood pressure (hypertension) results in approximately 9 million annual deaths worldwide. Currently available drugs used to control blood pressure can be effective but are limited by…

Read More

Gene therapy for ATP1A3-Related Neurologic Disorders

Gene therapy for ATP1A3-Related Neurologic Disorders

Unmet Need Children with Alternating Hemiplegia of Childhood can have episodes of temporary paralysis, dystonia, and severe behavioral problems. The prevalence of AHC is estimated to be 1 in one million to 1 out of…

Read More

Method for increasing gene expression in mammalian cells from transfected plasmids

Method for increasing gene expression in mammalian cells from transfected plasmids

Unmet Need Mammalian cell-based protein expression systems enable the production of mammalian proteins in the most native structure and activity. Thus, they have been commonly used for producing antibodies and therapeutic proteins, and more recently,…

Read More

Gene therapy for long-term decrease of intraocular pressure

Gene therapy for long-term decrease of intraocular pressure

Unmet Need It is estimated that in the US alone, approximately 3 million individuals are living with glaucoma, making it the leading cause of blindness in the US. There currently is no cure for glaucoma,…

Read More

Gene therapy and small molecule treatment of chronic pain without addiction

Gene therapy and small molecule treatment of chronic pain without addiction

Unmet Need Chronic pain is an escalating issue worldwide – in the U.S., an estimated one of five adults are affected. Chronic pain is difficult to treat, often requiring powerful opioids to provide relief. These…

Read More

CRISPR-based antiviral treatment of coronavirus infections

CRISPR-based antiviral treatment of coronavirus infections

Unmet Need The global market for antiviral drugs is a large one, topping $60B, and is getting larger due to the ongoing COVID-19 pandemic. Spurred on by a $3B investment from the U.S. government into…

Read More

A method of treating neurodegenerative diseases by preventing mitochondrial dysfunction

A method of treating neurodegenerative diseases by preventing mitochondrial dysfunction

Unmet Need Alzheimer's Disease (AD) is a devastating neurological disorder that results in significant cognitive impairment and memory loss. A common feature in late onset AD patients is the accumulation of amyloid plaques in the…

Read More

A method for treating critical limb ischemia and other ischemic injuries

A method for treating critical limb ischemia and other ischemic injuries

Unmet Need The most severe stage of peripheral artery disease, a chronic vascular disease characterized by impaired circulation to patients’ lower extremities, is critical limb ischemia (CLI), and it affects approximately two million people in…

Read More

Have Questions?

Please contact us or subscribe for more opportunities

Stay in Touch with Us