APPLICATIONS

CRISPR Cancer Research

Cancer affects millions of people worldwide, yet researchers have been working for decades to find effective treatments. New drugs, chemotherapy, and radiation techniques have been used over the years to treat patients, but none of these are devoid of side effects.
The discovery of CRISPR as a powerful gene editing tool ushers hope with its promising potential in cell and gene-based therapies, such as CAR-T therapy. Here we will discuss the basics of cancer and how CRISPR is transforming cancer medicine.

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When the idea of gene therapy was first proposed, it seemed like science fiction - even to medical professionals and researchers. Let's explore the emergence of gene therapy, its promise, and early setbacks.

A major setback

Cancer is a disease in which abnormal cells divide uncontrollably and, if malignant, can invade other tissues of the body. There are several types of cancer, commonly categorized by the affected tissue, including breast cancer, lung cancer, skin cancer, etc., or by the type of cell it originates from: carcinoma, lymphoma, leukemia, etc.

Sarcomas - cancers formed from bone or soft tissue (such as muscle and fat)
Leukemias - cancers that arise from tissue responsible for forming blood cells
Lymphomas - cancers that stem from a key cell type of the immune system i.e. lymphocytes (T cells or B cells)
Multiple myeloma - cancer that begins in plasma cells

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CRISPR Applications

CRISPR Base Editing (TEST)

A significant number of human genetic diseases are caused by single nucleotide variants, mutations that involve the alteration of a single DNA nucleotide.

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We are at an exciting inflection point in the development and delivery of cell and gene therapies, and I see significant opportunities for Synthego to further establish our position as a leading provider of products and services to the many companies globally developing CRISPR-based therapeutics.

Craig Christianson

Chief Executive Officer & President, Synthego

Synthego’s product allows us to quickly evaluate, in a cost-effective manner, the best guide RNA to stimulate genome editing, including by homologous recombination, in therapeutically relevant human stem cells and primary human T cells.

Matthew Porteus, M.D., Ph.D.

Principal Investigator, Stanford School of Medicine

The CRISPR Revolution

Trial	Treatment name	Editing method/strategy	Company or clinician	Status
NCT03745287	CTX001 (Casgevy)	Restored fetal hemoglobin by knocking out BCL11A transcription factor in autologous CD34+ HSPCs via CRISPR-Cas9	Vertex Pharmaceuticals Inc. CRISPR Therapeutics	Complete, successful
NCT03872479	EDIT-301	Enhanced fetal hemoglobin expression via targeted editing	Editas Medicine	Ongoing Phase I/II

The development of CRISPR-Cas9 as an easily customizable gene-editing tool in 2012 was groundbreaking for gene therapy researchers. CRISPR can fix the root cause of genetic conditions, potentially curing disease with a single treatment. Compared to previous gene-editing methods, CRISPR-Cas9 is also much simpler to perform, more precise and efficient at editing, and more cost-effective.

The simplicity of the CRISPR-Cas system and its easily programmable guides means that researchers can easily adapt it to any of the thousands of potential disease targets. It has predictable and consistent outcomes, with precise editing and low off-target activity compared to previous technologies, offering rapid translation from the bench to the clinic and higher levels of safety for patients.

Treatment Options for Cancer Patients

A Deep Dive into CRISPR Applications in Immuno-Oncology with Imran House and Junyun Lai

In this interview, Senior Scientist Imran House and Junyun Lai from oNKo-innate shared their approach at developing cytokine and CAR-T therapies to combat cancer and how they pivot their career path from academics to the biotech industry.

Immunotherapy and Cell-Based Therapies

T7 RNAP transcription cycle. Dousis, A., Ravichandran, K., Hobert, E.M. et al. An engineered T7 RNA polymerase that produces mRNA free of immunostimulatory byproducts. Nat Biotechnol 41, 560–568 (2023). https://doi.org/10.1038/s41587-022-01525-6. Cropped from original image. Licensed under CC BY 4.0: https://creativecommons.org/licenses/by/4.0/

Ready to get started?Synthego's CRISPR Nucleases

With a successful track record in synthesizing and optimizing gRNAs, Synthego is dedicated to equipping you with high-performance nucleases precisely paired with engineered gRNAs to meet your most challenging CRISPR objectives.

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References

Dutta, Devanjali, Inha Heo, and Hans Clevers. "Disease modeling in stem cell-derived 3D organoid systems." Trends in molecular medicine 23.5 (2017): 393-410.
Yang, Yinmeng, Elad Jacoby, and Terry J. Fry. "Challenges and opportunities of allogeneic donor-derived CAR T cells." Current opinion in hematology 22.6 (2015): 509.
Jinek, Martin, et al. "A programmable dual-RNA–guided DNA endonuclease in adaptive bacterial immunity." Science 337.6096 (2012): 816-821.
Eyquem, Justin, et al. "Targeting a CAR to the TRAC locus with CRISPR/Cas9 enhances tumour rejection." Nature 543.7643 (2017): 113.
Fenerty, Kathleen E., et al. "Immunotherapy utilizing the combination of natural killer–and antibody dependent cellular cytotoxicity (ADCC)–mediating agents with poly (ADP-ribose) polymerase (PARP) inhibition." Journal for immunotherapy of cancer 6.1 (2018): 133.
Roth, Theodore L., et al. "Reprogramming human T cell function and specificity with non-viral genome targeting." Nature 559.7714 (2018): 405.
Rogers, Zoë N., et al. "A quantitative and multiplexed approach to uncover the fitness landscape of tumor suppression in vivo." Nature methods 14.7 (2017): 737.

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