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Gene therapy for Crb1 retinopathies
Value Proposition Current treatments for retinal disease include laser surgeries, injectable corticosteroids, and other medications which may slow the process of retinal degeneration but are not able to stop it. The Crb1 protein has been…
Type I CRISPR-Cascade: precise gene modulation platform for human cell applications
Unmet Need The gene editing sector has experienced a transformative surge in technological advancements with the emergence of CRISPR-Cas systems. These systems offer unprecedented capability to modify and modulate genetic sequences, proving instrumental from fundamental…
Bacterial voltage gated ion channels for cardiac reprogramming
Unmet Need Untreated cardiovascular disease (CVD) can lead to irreversible heart damage. This occurs when ischemia — inadequate blood flow — leads to the death of heart muscle fibers that conduct electrical impulses. Consequently, scar…
Type I CRISPR-Cas expands the human genome engineering toolkit
Unmet Need Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-Associated Systems (Cas) provide immune protection to bacteria and archaea. CRISPR-Cas systems can be mono-subunit (Class 2) or multi-subunit (Class 1). Class 2 CRISPR-Cas systems, especially Type…
Methods and compositions to sense m6A RNA modifications using a genetically encoded sensor
Unmet Need Gene expression is highly regulated through the addition of chemical modifications to RNA molecules. One such modification is methylation of adenosine residues to form m6A, a modified nucleotide which is found in thousands…
Anti-LYPD3 CAR T-cell therapy for the treatment of squamous cell carcinoma
Unmet Need Chimeric Antigen Receptor (CAR) T-cell therapy, a form of immunotherapy, has proven highly effective against blood cancers such as leukemias and lymphomas. This therapy uses T-cells that are genetically engineering ex vivo to…
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…
High-stringency method for the isolation and proteomic identification of RNA binding proteins
Unmet Need RNA binding proteins (RBPs) play a critical role in maintaining stable gene expression, and they are involved in several related functions throughout eukaryotic cells, including splicing and translation. RBPs interact with RNA in…
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…
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…