POEM is a groundbreaking 3D bioprinting technology enabling high-resolution, multi-material, and cell-laden structure fabrication with enhanced cell viability.

This technology offers a novel approach to treating epilepsy by preventing the spread of epileptic networks and improving memory deficits through targeted electrical stimulation.

A revolutionary method for improving the efficiency and quality of reprogramming adult cells into stem cells or other therapeutically relevant cell types via adhesome gene manipulation.

This technology represents a groundbreaking approach to treating Primary Open Angle Glaucoma by directly targeting the trabecular meshwork pathology with lipid nanoparticle-mediated delivery of gene editing tools or anti-sense oligos.

A novel approach to significantly enhance vancomycin's effectiveness against drug-resistant pathogens by conjugating it with a minimal teixobactin pharmacophore.

Innovative cell lines enabling precise genetic modifications to advance research in gene function, disease modeling, and potential therapeutic interventions.

A revolutionary drug modality for the selective modification and degradation of intracellular proteins in lysosomes.

A novel method for selectively targeting and modulating brain border-associated myeloid cells for the treatment of neurological disorders.

Precise control of wound healing depends on physician’s evaluation, experience. Physicians provide conditions and time for body to either heal itself, or to accept and heal around direct transplantations, and their practice relies a lot on passive recovery. Slow healing of recalcitrant wounds is a known persistent problem, with incomplete healing, scarring, and abnormal tissue regeneration. 23% of military blast and burn wounds do not close, affecting a patient’s bone, skin, nerves. 64% of military trauma have abnormal bone growth into soft tissue. While newer static approaches have demonstrated enhanced growth of non-regenerative tissue, they do not adapt to the changing state of wound, thus resulting in limited efficacy.

A cutting-edge vaccine delivery platform that enhances tumor treatment by co-delivering MHC class I and II restricted antigens.

A groundbreaking method for the efficient isolation and preservation of high-purity small extracellular vesicles (sEVs - exosomes) from biofluids using a novel EXO-PEG-TR reagent.

Huntingtin disease (HD) is a heritable neurodegenerative disorder affecting up to 1 in 10,000 people, with an average survival duration of 17-20 years post symptom onset. HD patients typically suffer from severe motor/coordination decline and weight loss. There is no cure for HD, and traditional small molecule drugs only address symptom management. Prior approaches to treatment have failed for several reasons. Protein-targeting approaches such as ubiquitin ligase lack specificity, degrading both mutant and wild type huntingtin protein indiscriminately. Other approaches such as antisense oligonucleotides (ASOs) can target mutant RNA but require many doses over the patient’s lifetime. The disorder affects the huntingtin gene (HTT), which is essential in transcription, reactive oxygen species detection, DNA damage repair, and axonal transport. HD is caused by a heterozygous polyglutamine repeat expansion in exon 1 of HTT. Genome editing is an attractive alternative therapy for HD, as it would require a single dose and is permanent. UC Berkeley researchers have developed a system for CRISPR-based genome editing for genetic diseases like HD. Allele specific excision is possible through two different mechanisms: heterozygous SNPs that create/remove a PAM site, and heterozygous SNPs that create a mismatch within the seed region. For patients with these genotypes, the invention allows selective excision of the pathogenic repeats from only that allele. Over 20% of HD patients can be treated with just one of our novel candidate pairs, and about half of all patients could benefit from one of our novel candidate pairs.

This technology introduces a novel class of synthetic genetic polymers, capable of enhancing protein target binding and mimicking antibodies, for therapeutic and diagnostic applications.

Researchers at UCI and Harvard have engineered a new platform for diversifying antibody genes in yeast, eliminating a crucial bottleneck in making effective antibodies. This technology enables the rapid continuous directed evolution of affinity reagents for applications ranging from structural and cellular biology to diagnostics and immunotherapy.

An innovative small molecules therapy that significantly lowers intraocular pressure in glaucoma patients, offering neuroprotection and addressing trabecular meshwork fibrosis.

This technology offers a significant improvement in the therapeutic application of type E botulinum neurotoxin (BoNT/E) by introducing rationally designed mutations into the receptor binding domain.

IS110 family transposons encode a protein component (also referred to as the transposase) and a non coding RNA component (also referred to as the bridgeRNA or bRNA). In its naturally occurring context, a bRNA-bound transposase directs the integration of its cognate transposon (also referred to as the donor) into target DNA sites. The nucleic acid sequence and structure of the bRNA partially determines the sequence identify of the terminal ends of the mobilized donor, and the sequence identify of the target DNA molecule (also referred to as the target or target DNA). UC Berkeley researchers have developed a programmable gene editing technology based on IS110 family transposons that can be used for targeted insertions, deletions, excisions, inversions, replacements, and capture of DNA in vitro and in vivo. Additionally, this technology can be multiplexed to achieve complex assembles of multiple fragments of DNA.

Most tumors are extremely complex, having many oncogene drivers and are, therefore, not as amenable to a CRISPR-mediated therapies. Pediatric low-grade glioma (pLGG) is a type of brain cancer that arises during childhood. Some interventions exist, including surgery and inhibitor drugs, but there is no cure for pLGG. In contrast to most types of cancer (which feature a host of driver oncogenes), pLGG tumors tend to arise due to a single driver oncogene mutation. This aspect makes pLGG a potential target for a genome editing intervention. Because CRISPR enzymes can precisely discriminate between wild-type and mutant sequences in a single cell, enzymes such as Cas9 can target a mutant oncogene site without impacting the corresponding wild-type locus in a non-cancer cell. UC Berkeley researchers have developed a CRISPR-based strategies for anti-cancer genome editing.  The invention consists of a suite of genome editing strategies with the capacity to selectively inactivate the oncogene underlying tumor pathology, for example, mutations in pLGG. Deployed via a delivery strategy with the capacity for broad genome editing of brain cells, our strategy will have the capacity to halt – and potentially reverse – tumor growth.

A novel antimicrobial approach combining pore-forming agents with histones to eradicate bacteria and bypass known resistance mechanisms.

SUMO inhibitors offer a promising new therapy for protecting against cardiac rhythm disturbances and pump failure associated with heart attacks.

Researchers at the University of California, Davis have developed a technology that targets CD147 to significantly improve bone health and treat musculoskeletal diseases without the side effects of current therapies.

Existing RNA-targeting tools for sequence-specific manipulation include anti-sense oligos (ASOs), designer PUF proteins and CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats)/Cas systems. However, there are significant limitations to each of the current tools. ASOs are usually not available for most RNA manipulations other than gene silencing. Designer proteins, such as PUF (Pumilio and FBF homology protein), possess low RNA recognition efficiency and it remains challenging to target RNA sequences >8-nucleotides (nt) in length. The bulky Cas protein (Cas13d: average 930 amino acids) leads to complication for transgene delivery and concerns of its immunogenicity due to its bacterial origin. Mutants of zinc finger(ZnF) proteins in ZRANB2 recognize a single-strand RNA containing a novel GGG motif with micromolar affinity, compared to the original motif GGU. These mutants serve as a foundation for RNA-binding ZnF designer protein engineering for in vivo RNA sequence-specific targeting.ZnFs are generally compact domains (~3kDa each) that have been successfully engineered for DNA recognition as modular arrays. A ZnF-based system has unique advantages, especially in a therapeutic context: (1) Broad application with the possibility to fuse with other effector domains; (2) High efficiency of RNA recognition (3 RNA bases recognized per 30-amino-acid ZnF) with a small size of protein. Only 4 ZnFs (~100 aa) is required for specific targeting in the transcriptome. (3) Humanized components without immunogenic concern.By engineering new sequence specificity of the ZRANB2 ZnF1, researchers from UC San Diego identified 13 mutants that altered their preferred RNA binding motif from GGU to GGG. They are N24R, N24H, N14D/N24R, N14D/N24H, N14R/N24R, N14R/N24H, N14H/N24R, N14H/N24H, N14Q/N24R, N14Q/N24H, N14E/N24R, N14S/N24R, N14E/N24H.

Lipid Nanoparticles (LNPs) are a leading platform for nucleic acid delivery, widely used in therapeutics and vaccine development. However, the process of optimizing new LNP formulations has been significantly hindered by labor-intensive and costly screening methods, which require individual injections into animal models. Given the vast array of potential lipid compositions and formulation variables, these constraints severely impede the efficiency of research and development.To overcome these challenges, UC Berkeley researchers have developed a novel approach for identifying and characterizing functional nucleic acid delivery vehicles. This innovative method leverages circular RNA barcoding technology, enabling a more efficient screening process. Instead of relying on conventional cell sorting techniques, which restrict screening to specific organs and host species, this breakthrough allows direct detection of barcoded nucleic acids within circular RNAs in treated cells. By analyzing the barcodes detected, researchers can accurately determine which lipid compositions and formulations successfully delivered RNA molecules.  This technology represents a significant advancement in LNP research, offering a scalable, cost-effective solution that enhances the precision and scope of nucleic acid delivery screening.

Researchers at UC Irvine have identified genetic polymorphisms associated with disease progression and responsiveness to treatment with Tetracosapentaenoic acid (24:5 n-3) for age-related eye disorders such as age-related macular degeneration (AMD), diabetic retinopathy and glaucoma. These variations found in the ELOVL2 gene are associated with AMD progression and the varying responses individuals have to AMD treatments, including preventative measures. Additionally, these genetic variations have applications in human identification.

Researchers at the University of California, Davis, have developed a process for isolating anti-inflammatory lipids for treating autoimmune and inflammatory diseases.