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CRISPR/Cas9-based Therapy Development Platform

CRISPR/Cas9 is a commonly used gene-editing tool that can add, remove, or alter genetic material at particular locations in the genome. With a professional technical team and a complete genome editing platform, our company's CRISPR/Cas9-based therapy development service can provide a full set of gene therapy solutions. We customize personalized solutions according to your scientific research needs and provide cost-effective and professional services to accelerate your gene therapy research and development for rare diseases.

Introduction to CRISPR/Cas9

CRISPR/Cas9, also known as clustered regularly interspaced short palindromic repeats or CRISPR-associated nuclease 9, is an RNA-guided targeted genome editing technology adapted from the antiviral immune response of bacteria. The CRISPR/Cas9 system consists of two elements, sgRNA (single-guide RNA) and Cas9 protein.

CRISPR/Cas9-based Therapy Development Platform-1

Fig.1 Applications of CRISPR-Cas9 based gene engineering. (Wang, Yu, et al., 2019)

Usually, sgRNA guides the Cas9 protein to recognize the genome. With the protospacer-adjacent motif (PAM - the sequence NGG) present at the 3' end. Cas9 can cleave specific sites in the genome to form double strand breaks (DSBS). Double-strand breaks are usually repaired through non-homologous end joining, which usually deletes or changes the nucleotide at the site where the joining occurs. If Cas9 has one nuclease site inactivated, it will produce a single-stranded break in the target sequence. In the presence of recombinant donor DNA fragments, homologous DNA recombination sometimes changes the sequence of the break site to match the sequence of the donor DNA.

CRISPR/Cas9-based Therapy for Rare Diseases

CRISPR/Cas9-based therapy can be used to change the DNA of cells or organismsCas9-based be used for therapeutics research of rare diseases, such as cystic fibrosis, hemophilia, β-thalassemia, Huntington's, Parkinson's, tyrosinemia, Alzheimer's, Duchenne muscular dystrophy, Tay-Sachs, and fragile X syndrome disorders. Currently, there are already some cases of using CRISPR/Cas9-based therapy to treat rare diseases:

Table 1 Rare disease product pipelines of leading companies based on
CRISPR/Cas9-based therapy. (Mani, Indra., 2021)

Rare Disease Companies Mechanism Delivery Notes
a-1-Antitrypsin deficiency EDIT NHEJ & HDR In vivo By AAV or LNP
NTLA n/a In vivo By LNP
Amyotrophic lateral sclerosis CRSP n/a In vivo Collaboration with Target ALS and University of Florida
Autoimmune and inflammatory diseases NTLA n/a Ex vivo Disease unspecified
β-Hemoglobinopathies (β-thalassemia & sickle cell disease) CRSP NHEJ Ex vivo CTX001;based on induction of y-globin
NTLA n/a Ex vivo In collaboration with Novartis Institute for Biomedical Research, Inc.
Cystic fibrosis CRSP HDR In vivo Correction/insertion of CFTR gene
EDIT NHE & HDR In vivo By AAV or LNP
Duchenne muscular dystrophy CRSP NHEJ In vivo Functional correction of DMD gene by deletion
EDIT NHEJ In vivo Correction by small and large deletion; by AAV or LNP
Friedreich's ataxia CRSP n/a In vivo Collaboration with Friedreich's Ataxia Alliance & University of Alabama (Birmingham)
Glycogen storage disease Ia CRSP HDR In vivo Correction/insertion of G6PC gene
Hemophilia CRSP HDR In vivo Withdrawn from portfolio Q3/4 2018
Hurler Syndrome CRSP HDR Ex vivo Correction/insertion of IDUA gene
Inborn errors of metabolism NTLA n/a In vivo Disease unspecified; by LNP
Leber congenital amaurosis 10 EDIT NHEJ In vivo Correction by small deletion; by local AAV injection
Liver diseases NTLA n/a Ex vivo Disease unspecified
Primary hyperoxaluria type 1 NTLA na In vivo By LNP
Severe combined immunodeficiency CRSP HDR Ex vivo Withdrawn from portfolio Q3/4 2018
Transthyretin amyloidosis NTLA n/a In vivo By LNP: collaboration with Regeneron Pharmaceuticals, Inc.
Usher syndrome 2a EDIT NHEJ In vivo By local AAV injection

Our Services

With extensive experience in gene therapy development, our company provides you with a CRISPR/Cas9-based therapy development service to edit and treat any DNA mutations associated with rare diseases and provides you with one-stop solutions to accelerate your research and development of rare disease therapies.

CRISPR/Cas9 System Development

According to different disease types and therapeutics requirements, our company designs and develops suitable CRISPR/Cas9 systems, including selecting the appropriate Cas protein, designing and synthesizing gRNA, and providing you with precise gene editing for your rare disease gene therapy research.

CRISPR/Cas9 System Delivery

Based on specific application needs and cell types, our company uses viral vector and non-viral vector delivery strategies to provide you with efficient, low-toxicity, and highly specific CRISPR/Cas9 system delivery services. Support your rare disease gene therapy research by building the cell models needed for gene therapy, or by repairing genetic mutations in vivo through gene editing.

Off-target Effect Analysis

Our company provides comprehensive and accurate CRISPR/Cas9 off-target effect detection and analysis services to improve the safety of CRISPR/Cas9-based therapies. The analysis methods we use include but are not limited to:

  • Whole Genome Sequencing
  • Digenome-Seq

Project Workflow

CRISPR/Cas9-based Therapy Development Platform-5

With a research team with extensive expertise, our company is confident to provide clients with CRISPR/Cas9-based therapy development services. We have the capabilities and resources to provide professional communication and problem-solving support to ensure that we can quickly respond to the changing needs of your rare disease therapy research projects. If you are interested in our services, please feel free to contact us for more details and quotation information of related services.


  • Wang, Yu, et al. "A highly efficient CRISPR-Cas9-based genome engineering platform in Acinetobacter baumannii to understand the H2O2-sensing mechanism of OxyR." Cell Chemical Biology 26.12 (2019): 1732-1742.
  • Mani, Indra. "CRISPR-Cas9 for treating hereditary diseases." Progress in Molecular Biology and Translational Science 181 (2021): 165-183.

All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.

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