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Technology alone won't get you where you want to go. And that's why we won't ever just offer technology.

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Primary Literature

Cell Therapy

CRISPR/Gene Editing

HSPCs

Preclinical evaluation for engraftment of CD34+ cells gene-edited at the sickle cell disease locus in xenograft mouse and non-human primate models

Sickle cell disease (SCD) is caused by a point mutation in the b-globin gene and can be cured by the replacement of hematopoietic stem cells (HSCs).

Primary Literature

Cell Therapy

CRISPR/Gene Editing

T Cells

Mesothelin-Specific Chimeric Antigen Receptor mRNA-Engineered T Cells Induce Anti-Tumor Activity in Solid Malignancies

Off-target toxicity due to the expression of target antigens in normal tissue represents a major obstacle to the use of chimeric antigen receptor (CAR)-engineered T cells for treatment of solid malignancies. To circumvent this issue, we established a clinical platform for engineering T cells with transient CAR expression by using in vitro transcribed mRNA encoding a CAR that includes both the CD3-ζ and 4-1BB co-stimulatory domains.

Primary Literature

Cell Therapy

CRISPR/Gene Editing

HSPCs

MSCs

Modifying Stem Cells for Clinical Regenerative Applications

Various stem cells types are being evaluated for the potential regeneration of heart, bone, cartilage, nerve and other tissues.

Primary Literature

Cell Therapy

CRISPR/Gene Editing

T Cells

Multiple Injections of Electroporated Autologous T Cells Expressing a Chimeric Antigen Receptor Mediate Regression of Human Disseminated Tumor

Redirecting T lymphocyte antigen specificity by gene transfer can provide large numbers of tumor-reactive T lymphocytes for adoptive immunotherapy. However, safety concerns associated with viral vector production have limited clinical application of T cells expressing chimeric antigen receptors (CAR). T lymphocytes can be gene modified by RNA electroporation without integration-associated safety concerns.

Primary Literature

Cell Therapy

CRISPR/Gene Editing

T Cells

Next-generation DNA vectors: is the nS/MARt platform a viable alternative to viruses for autologous T-cell immunotherapy?

Next-generation non-viral RNA and DNA vectors have emerged as an attractive alternative for introducing CARs or TCRs into immune cells; while maintaining a high efficiency of delivery, they are more versatile and are simpler and quicker to manufacture at scale, thus increasing the number of patients who can be treated while significantly reducing the vein-to-vein time of the treatment process.

Primary Literature

Protein Production

CHO

Potent immunomodulatory effects of an anti-CEA-IL-2 immunocytokine on tumor therapy and effects of stereotactic radiation

While anti-CEA antibodies have no direct effect on CEA-positive tumors, they can be used to direct potent antitumor effects as an antibody-IL-2 fusion protein (immunocytokine, ICK), and at the same time reduce the toxicity of IL-2 as a single agent.

Primary Literature

CRISPR/Gene Editing

Gene Therapy

B Cells

Cancer Cell Lines

Rapid and efficient nonviral gene delivery of CD154 to primary chronic lymphocytic leukemia cells

Interactions between CD40 and CD40 ligand (CD154) are essential in the regulation of both humoral and cellular immune responses. Forced expression of human CD154 in B chronic lymphocytic leukemia (B-CLL) cells can upregulate costimulatory and adhesion molecules and restore antigen-presenting capacity.

Primary Literature

Protein Production

CHO

Rapid and sensitive detection of SARS-CoV-2 antibodies by biolayer interferometry

Here, we describe a novel application of biolayer interferometry for the rapid detection of antigen-specific antibody levels in plasma samples, and demonstrate its utility for quantification of SARS-CoV-2 antibodies.

Primary Literature

Cell Therapy

CRISPR/Gene Editing

NK Cells

Large Volume Flow Electroporation of mRNA: Clinical Scale Process

Genetic modification for enhancing cellular function has been continuously pursued for fighting diseases. Messenger RNA (mRNA) transfection is found to be a promising solution in modifying hematopoietic and immune cells for therapeutic purpose. We have developed a flow electroporation-based system for large volume electroporation of cells with various molecules, including mRNA.