Genome Editing – Simplified solution to modify target gene

New York, NY, 2019-Feb-27 — /EPR Network/ — Genome editing is a scientific way of modifying the DNA of many organisms such as plants, bacteria, and animals. By making changes in DNA it leads to change in physical traits, like eye color, and disease risk. Scientists use different technologies to carry out to remove, add, or replace the DNA where it was edited

The genome editing is used in cell line engineering, plant genetic engineering and animal genetic engineering of which cell line engineering dominates the market owing to new cell lines techniques, such as cell line engineering, plant genetic engineering, and animal genetic engineering In June 2018, Synthego, one of a leading genome engineering solutions company, introduced its Engineered Cells Portfolio with immediate access to Knockout Clone and Pool, and Advanced Cells. The Knockout Clone and Pool products enable single-click online ordering of any human cell line with a guaranteed CRISPR knockout. Strategic collaboration between Oxford Genetics signed an agreement with global e-commerce providers on supply and licensing agreements, where Oxford Genetics leveraged its high throughput automated genomic engineering platform for CRISPR modification of mammalian cell lines.  

The CRISPR technology is the most powerful tool for editing genomes which allows researchers to easily alter DNA sequences and modify gene function. CRISPR technology is used for many applications include correcting genetic defects, treating and preventing the spread of diseases and improving crops. Additionally, increasing clinical trials on CRISPR is one of the key factor fueling the global genome editing market.

For instance, in September 2018, Boston-based Vertex Pharmaceuticals and CRISPR Therapeutics, jointly introduced a trial of an experimental CRISPR-Cas9 therapy for the blood disorder β-thalassemia.

In August 2018, CRISPR Therapeutics and Vertex Pharmaceuticals have sponsored the first human trial of gene editing technology. The trial has taken place in Germany. CTX001 is being developed for the blood disorders sickle cell disease and beta thalassemia. CTX001 uses CRISPR gene-editing ex vivo which is, outside the body.

The global genome editing market was valued at US$ 3,213.2 million in 2018 and is expected to witness a CAGR of 14.1% over the forecast period (2019–2026).

 

Government Initiatives for Precision Medicine and Potential opportunities in orphan diseases and oncology

Increasing government initiatives to enhance precision medicine as a new and advanced technology for precise cure and treatment is expected to boost the global precision medicine and further boosts the global genome editing market

For instance, in February 2018, the California government has introduced the bipartisan Advancing Access to Precision Medicine Act, to push forward use of genetic and genomic testing to improve and save lives. The Advancing Access to Precision Medicine Act would direct the Department of Health and Human Services to enter into an agreement with the National Academy of Medicine to develop recommendations on how the federal government may reduce barriers to the utilization of genetic and genomic testing.

In January 2018, the governor of California has funded US$ 30 million to establish the California Institute to Advance Precision Health and Medicine.

In October 2016, The collaboration between the University of Auckland, Ministry of Business, local health board and software company Orion Health have invested US$ 26.25 million for multidisciplinary research projects in precision medicines.

CRISPR/Cas9, ZNF, and TALEN are widely used in gene therapy to cure genetic diseases and lethal diseases through gene modification. Pharma and biotech companies are adopting these technologies for better disease management as well as the development of personalized medicine.

CRISPR/Cas9 system is one of the best innovative technologies that have the potential to reshape a larger gene therapy landscape to treat a broad range of disorders including cancer, viral infection and genetic diseases. Many vendors are using the CRISPR/Cas9 system to enter into the niche genome engineering market.

TALEN can be used to treat X-linked severe combined immunodeficiency, lymphoblastic leukemia. The technology has generated custom T-cells that are useful to treat cancer, and ZNF produces an HIV-resistant T-cell population that is expected to treat HIV in individuals.

Research Funding for genome editing

As genome editing has gained trust among most of the major players,  there has been a significant contribution and funding in the year 2018, for instance, in  October 2018, Synthego, one of a leading genome engineering innovation company, has received US$110 million funds to accelerate CRISPR capabilities for rapid and precise genome engineering in applications such as cell and gene therapies.

In February 2018, Inscripta, a key gene-editing technology company, has received US$ 55.5 million funds for its first CRISPR enzyme (MAD7), and will accelerate Inscripta’s development and commercialization of gene-editing tools, including instruments, reagents, and software.

In October 2018, Gladstone Institutes have received than $3.6 million funds to introduce the Somatic Cell Genome Editing program, the fund is available for the next 5 years to assess the safety of genome editing in human cells and tissues. The funding is also available to expand the current toolkit for human genome editing by exploring new CRISPR-Cas proteins and enzymes that can repair DNA.

In May 2018, Beam Therapeutics has received US$ 87 million in funding for technological development in order to target genome editing. Beam’s research will focus on multiple DNA base editor platforms developed in the lab, aiming to use these technologies to generate a broad pipeline of precision genetic medicines that repair disease-causing point mutations, write in protective genetic variations, or modulate the expression or function of disease-causing genes.

More market insights about Genome Editing Market https://www.datamintelligence.com/research-report/genome-editing-market/

 

Recent Product launches and key developments

The product launches, application grants, and other key developments are the dominating strategies adopted by the market players to enhance their product portfolio in the genome editing market.

For instance, on January 25, 2019, Vertex regained limited rights of two DNA-dependent protein kinase (DNA-PK) inhibitors from Merck. Merck licensed the drugs and other assets for $230 million upfront earlier and has now granted Vertex the right to use them in certain gene-editing applications. With such developments, researchers used DNA-PK inhibitors to modulate mechanisms that repair DNA breaks and thereby improve CRISPR/Cas9 genome editing.

On January 21, 2019, Horizon Discovery extended its CRISPR Screening Service to include ex vivo T lymphocytes. The service extension would meet the requirements of immunology-based research in drug discovery, enabling new gene targets to be identified in biologically and potentially therapeutically relevant settings.

On January 22, 2019, CRISPR Therapeutics and ProBioGen, a premier German service and technology provider, collaborated for multi-year research and development of the novel in vivo delivery modalities for CRISPR/Cas9.

On January 04, 2019, CRISPR Therapeutics and Vertex Pharmaceuticals Incorporated were granted Fast Track Designation by FDA, for CTX001 for the treatment of sickle cell disease (SCD). CTX001 is an investigational, autologous, gene-edited hematopoietic stem cell therapy for patients suffering from severe hemoglobinopathies.

Such recent key developments were also seen by the other prominent players such as Thermo Fisher Scientific Inc., New England Biolabs, Inc., GenScript USA Inc., Integrated DNA Technologies Inc., Horizon Discovery Group plc, Origene Technologies Inc., Sangamo Biosciences, Inc., Transposagen Biopharmaceuticals, Inc., and CRISPR Therapeutics, among others.