- Agriculture
- Antibiotic/anti-viral
- Biologics
- Biomarkers
-
By Clinical Application
×
- Anesthesiology
- Blood & Lymphatic Disease
- CNS & Neurosciences
- Dermatology
- Diabetes, Metabolism, Endocrinology & Obesity
- Ear, Nose, & Throat
- Emergency Services
- Gastroenterology & Digestive Disease
- General & Plastic Surgery
- Health Education, Medical Training and Operations
- Heart and Vascular
- Immunology, Autoimmune & Inflammation
- Infectious Diseases
- Mental Health
- Multiple clinical applications
- Musculoskeletal Disorders, Orthopedics/Bone
- Nephrology/Renal
- Oncology
- Ophthalmology
- Orphan Diseases
- Pediatrics
- Physical Medicine & Rehabilitation
- Radiology
- Regenerative Medicine / Tissue Engineering
- Reproductive Health: Obstetrics & Gynecology
- Respiratory & Pulmonary
- Surgery
- Transplantation
- Urology
- Wound Healing
- COVID-19
- Creative Works
- Diagnostics
- Drug Delivery
- Drug screening and discovery
- Energy, Cleantech & Environmental
- Engineering & Physical Sciences
- Gene therapy ×
- Imaging
- Materials
- Medical Devices
- Nutraceuticals
- Other
- Research & Design Tools
- RNAi/siRNA
- Sensors & Controls
- Small molecules
- Software & Information Technology
- Stem Cells
- Vaccines
- Veterinary Medicine
Gene therapy for long-term treatment of Pompe disease
Unmet Need Pompe disease is a rare heritable disorder impacting heart and skeletal muscle function with an estimated incidence rate of 1 in 40,000 births in the US. Of these affected births, a third take…
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…
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…
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…
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…
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…
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,…
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…
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…
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…
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…
An injectable hydrogel scaffold that delivers gene therapies locally
Unmet Need Gene therapy has applications ranging from tissue engineering to vaccine development to infectious disease. Most approaches to deliver nucleic material involve the systemic intravenous delivery of condensed nucleic acid (e.g., within a virus…