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Gene editing company: Precision BioSciences (DTIL)

Precision BioSciences, Inc. (NASDAQ:DTIL) was founded in January 2006 and is headquartered in Durham, North Carolina, USA, with 127 full-time employees (12/31/2018). It is a company dedicated to pioneering The proprietary genome editing platform “ARCUS” is a genome editing company that improves lives.

Precision BioSciences

Precision BioSciences (DTIL) US stock:

Precision BioSciences uses its proprietary ARCUS® platform to develop product candidates aimed at treating human diseases and creating healthy and sustainable food and agricultural solutions. The company believes that the versatility and breadth of ARCUS® supports the company’s ability to develop various biotechnology products.

Precision BioSciences is actively developing candidate products in three innovative and high-value fields. The company believes that its technology has the potential to overcome the limitations of other genome editing technologies: allogeneic CAR T immunotherapy, in vivo gene correction and food.

The US Food and Drug Administration (FDA) recently accepted Precision BioSciences’ research new drug (IND) application-the company’s first gene-edited allogeneic CAR T cell candidate targeting CD19.

Precision BioSciences is currently screening patients with relapsed or refractory (R/R) B-cell precursor acute lymphoblastic leukemia and R/R non-Hodgkin’s lymphoma in a planned phase I/IIa clinical trial, and Expect to treat the first batch of patients in April 2019. The company believes that this trial will be the first clinical study of allogeneic CAR T in the treatment of non-Hodgkin’s lymphoma.

Precision BioSciences believes that the company’s proprietary one-stop engineering process can produce allogeneic CAR T cells in a cost-effective manner on a large scale, which will enable the company to overcome the basic challenges that have limited the development of the CAR T field so far.

Precision BioSciences’ gene editing platform:

Genome editing is a biotechnology process that can remove, insert, or repair a part of DNA at a specific location in the cell’s genome. The company’s proprietary genome editing platform ARCUS is a new type of genome editing technology that uses sequence-specific DNA cutting enzymes or nucleases to modify the DNA of living cells and organisms.

ARCUS is not CRISPR/Cas9 technology. ARCUS is a collection of protein engineering methods. The company specifically develops DNA recognition features for reprogramming the natural genome editing enzyme I-CreI. In nature, I-CreI is an endonuclease found in the genome of Chlamydomonas reinhardtii, which has evolved to perform complex gene insertion editing.

In order to apply I-CreI to genome editing in other cells or organisms, Precision BioSciences must modify it to identify and cut the different DNA sequences for each new application the company pursues. Since I-CreI endonuclease has evolved to highly selectively recognize its target sequence in the algae genome, with the support of scientific literature, it is necessary for companies to develop complex protein engineering methods to redesign I-CreI to combine and cut different DNA sequence. Use ARCU Precision BioSciences to create custom endonucleases for specific applications. Precision BioSciences refers to these customized endonucleases as “ARCUS nucleases.”

Precision Biosciences believes that its gene editing platform has : high specificity, high efficiency, easy delivery, programmable and cutting characteristics.

Cooperation with Gilead Sciences:

In September 2018, Gilead Sciences and Precision BioSciences jointly announced that the two parties have reached a strategic cooperation agreement to use Precision’s unique ARCUS gene editing technology platform to jointly develop innovative therapies to eliminate hepatitis B virus (HBV) in the body .

There are approximately 257 million hepatitis B patients in the world. Current treatments for HBV can inhibit the replication of the virus, but cannot completely eliminate the virus. Once the medication is stopped, the covalently closed circular DNA (cccDNA) present in the human body can allow HBV to resume replication. Therefore, how to target the cccDNA of HBV is the key to the complete elimination of HBV from the body.

Precision’s ARCUS genome editing system is based on a nuclease called ARC, which is a completely artificially synthesized nuclease and has similar properties to the homing endonuclease that occurs naturally in the human body. The unique ability of homing endonuclease is the ability to accurately identify long DNA sequences (12-40 base pairs) that may only appear once in a complex genome. Therefore, they can cause only one gene editing in a complex genome without random off-target effects.

According to the agreement, Precision is mainly responsible for the research and development, formulation, and preclinical research of the nuclease under investigation. Gilead Sciences will be responsible for clinical development and promotion. Based on the milestones reached, Precision may receive a total of US$445 million in payments and future product sales.

Precision BioSciences (DTIL) financing and cooperation history:

  • In May 2015, in the A round of financing, Precision BioSciences received a lead investment from VenBio, and Fidelity Biosciences, Amgen Ventures, Baxter Ventures, Osage University Partners, the Longevity Fund followed up with a US$25.6 million investment.
  • In the B round of financing in June 2018, Precision BioSciences was led by ArrowMark Partners, and new investors Franklin Templeton Investments, Cowen Healthcare Investments, Brace Pharma Capital, Pontifax AgTech, OCV Partners, Adage Capital Management, Cormorant Asset Management, Gilead Sciences, Vivo Capital, Alexandria Venture Investments, Ridgeback Capital, Agent Capital, Leerink Partners, and existing investors venBio, F-Prime, RA Capital Management, Amgen Ventures, Osage University Partners, DUMAC and Longevity Fund, etc. with a joint investment of 110 million US dollars investment.
  • In September 2018, Gilead Sciences and Precision BioSciences jointly announced that the two parties have reached a strategic cooperation agreement to use Precision’s unique ARCUS gene editing technology platform to jointly develop innovative therapies to eliminate hepatitis B virus (HBV) in the body According to the milestones reached, Precision may receive a total of US$445 million in payments and future product sales. .

Precision BioSciences (DTIL) investment:

Precision BioSciences, Inc. (NASDAQ:DTIL) submitted its IPO prospectus on 3/1/2019, and plans to log on to Nasdaq on 3/28/2019. The issue price is US$16, and 7.9 million shares are issued. Capital of 126.4 million US dollars, JP Morgan/Goldman Sachs/Jefferies/Barclays underwriting.

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