Precision BioSciences의 유전자 편집 기술이 CRISPR의 대항마 될까?

 

 

1. 내용 Key Highlights

 

an opportunity to showcase the company’s platform, which was derived from I-CreI, a naturally occurring gene editing enzyme that comes from algae.

 

According to Precision Chief Scientific Officer Derek Jantz, Ph.D., Arcus' X-factor is its ability to make insertions to the genome as opposed to only eliminating gene function. 

 

When it comes to leveraging gene editing for rare genetic disease, this ability to make insertions is significant, because "the vast majority of genetic disorders are caused by the absence of something that is supposed to be there,” Jantz told Fierce Biotech. 

 

One of the two early programs Precision highlighted detailed this exact mechanism among infant nonhuman primates to treat ornithine transcarbamylase deficiency (OTCD).

 

Once it settled on PCSK9, the company used two adeno-associated viral (AAV) vectors to actually tweak the genes: one that knocked out PCSK9 and another that delivered an ornithine transcarbamylase minigene. 

 

Precision is also working on a lipid nanoparticle to deliver gene therapy that it touts as potentially curative for chronic hepatitis B. Whereas most treatments look to stop the hepatitis virus from replicating, Precision’s intends to kill off the viral DNA entirely.

 

To test it, the company used both a mouse and nonhuman primate model, which found a "significant decrease" in total AAV copy number as well as high indel frequency, a measure of gene editing precision. Treatment in the mouse model specifically resulted in a 96% reduction in circulating surface antigen of the virus. Study author Cassandra Gorsuch, Ph.D., director of gene therapy discovery at Precision, said this was promising. 

 

Evidently, the hype that Precision has accrued through its early development has been noticed by others. The OTCD program has been picked up by iECURE, one of the multiple companies that spun out of the lab of Jim Wilson, M.D., Ph.D., at the University of Pennsylvania. The chronic hepatitis B program, on the other hand, was first snagged by Gilead Sciences in 2018 before being axed in 2020. That’s no problem for Jantz, who said the company is happy to have it back. 

 

https://www.fiercebiotech.com/research/precision-biosciences-boasts-preclinical-data-two-gene-editing-programs-making-its-case

Precision BioSciences makes case for 2 gene editing programs over CRISPR with preclinical data

 

2. 내 생각

 

1) Time to market is the key to preoccupying the gene editing market.

 

There are several types of gene editing technology that are currently under development. There are some differences among those techs, for example,mechanism of action, difficulties of engineering and delivery tech and so on. If we do a head-to-head comparison of those techs, I am sure we can find the most superior one. However, in this stage where any tech. is approved and marketed, comparing them is meaningless.

 

The first one which shows clincal effcicay and comes to the market will dominate the market. For this new type of tech., time to market is the key for a success and preoccupation of the market.

 

2) ARCUS from Precision BioSciences are required to proceed clinical trials to show human POC (proof of concept).

 

Those two preclincal results in Hepa B. and OTCD look promising. Similar preclinical data will not be surprising for investors. Precision needs to go through the next stage which is clinical trial.

 

3) CRISPR is being tested under clinical trials in diverse indications, ahead of ARCUS.

 

CRISPR seems ahead of ARCUS when it comes to the development stage. 

 

 

3. 추가 리서치 / 참고 자료

 

 

 

https://www.google.com/search?q=crispr+vs+arcus&sxsrf=ALiCzsYA2NjXxJp7zjPK-viiAberFkzGMQ:1653600300816&source=lnms&tbm=isch&sa=X&ved=2ahUKEwirgZ-Ujf73AhX1zYsBHconD8sQ_AUoAXoECAEQAw&biw=1991&bih=837&dpr=0.94#imgrc=xO7cHWcpDHZyrM 

crispr vs arcus - Google 검색

 

https://innovativegenomics.org/news/crispr-clinical-trials-2022/

CRISPR Clinical Trials: A 2022 Update