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RAFT: A CRISPR-Free RNA Trans-Splicing Platform for MYH7 Correction in Hypertrophic Cardiomyopathy
Unmet Need Hypertrophic cardiomyopathy (HCM) is a common inherited heart disease affecting 1 in 500 individuals, frequently caused by mutations in the MYH7 gene. Current gene therapy approaches, including AAV-based gene replacement, face significant challenges…
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…
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…
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…
Pancreatic Cancer Treatment Targeting the Piezo1/TRPV4 Axis
Unmet Need Pancreatic cancer remains one of the most lethal cancers, with a 5-year survival rate of only 5–10%. In the U.S. alone, an estimated 64,000 people will be diagnosed each year, with over 55,000…
Viral-mediated humanized mouse model for Parkinson’s disease
Unmet Need Parkinson’s disease (PD) is a progressive neurodegenerative disorder characterized by the loss of dopaminergic neurons in the midbrain and the accumulation of α-synuclein aggregates (Lewy bodies), leading to motor and cognitive impairments. PD…
A novel T-cell-based immunotherapy for brain cancer
Unmet Need Cancer immunotherapy represents a new paradigm in cancer treatment with the goal of harnessing the immune system to selectively attack a patient’s tumor. T-cell-based immunotherapies encompass chimeric antigen receptor (CAR)-T cells, tumor-infiltrating lymphocytes,…
Targeted SNCA Downregulation for Parkinson’s and Synucleinopathies
Unmet Need Parkinson’s disease (PD) is the second most common neurodegenerative disorder, affecting approximately 10 million people globally and 1 million in the U.S. The disease is characterized by involuntary movements such as tremors, stiffness,…
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…
TGF-β1-Secreting Bone Marrow Stromal Cell Therapy for Long-Lasting Neuropathic Pain Relief
Unmet Need Neuropathic pain remains a significant clinical challenge, with current treatments providing only temporary relief and failing to address the underlying neuroinflammatory mechanisms. Chronic nerve injury leads to persistent pain hypersensitivity, which is difficult…
PD-L1-Based Therapy for Non-Opioid Treatment of Chronic Pain
Unmet Need Chronic pain, including inflammatory, neuropathic, and cancer-related pain, remains difficult to manage without opioids, which pose risks of addiction and adverse side effects. Existing non-opioid treatments often fail to provide sufficient relief or…
GPR37 and GPR37L1 Agonists for Non-Addictive Pain Resolution in Inflammatory and Neuropathic Pain
Unmet Need Chronic pain remains a widespread clinical issue, with existing treatments primarily focused on symptom suppression rather than addressing the biological mechanisms driving pain persistence. Opioids and NSAIDs provide temporary relief but fail to…