"Gene Magic Scissors" CRISPR announced the results of in vivo clinical trials, and many gene editing stocks soared!

Thanks to the success of NTLA-2001, Beam stock rose 16% that day
.

CRISPR Therapeutics-up 7% that day

CRISPR Therapeutics is developing a variety of CRISPR therapies in vivo and in vitro
.
The company currently has four drug candidates: CTX001, CTX110, CTX120 and CTX130
.

Among them, CTX001 has the fastest clinical progress.
It works by cutting a BCL11A gene that inhibits the production of fetal hemoglobin (HbF)
.
In vitro, CTX001 introduces the patient’s autologous hematopoietic stem cells through electroporation, and then returns them to the patient’s body to produce red blood cells containing high levels of HbF for the treatment of genetic diseases with impaired oxygen transport in the blood, such as β-thalassemia and Sickle cell disease
.

In the phase I/II clinical trial for the treatment of patients with severe sickle cell disease (SCD), patients with severe SCD who received a single dose of CTX001 quickly achieved strong, long-lasting, and high-level expression of pan-cellular HbF, while reducing pain and A debilitating sickle crisis
.

Thanks to the success of NTLA-2001, CRISPR Therapeutics stock rose by 7% that day
.

Editas Medicine-6%

The core product of Editas Medicine is AGN-151587 (EDIT-101), a gene editing therapy based on CRISPR technology.
It loads the gene encoding Cas9 and two guide RNAs (gRNA) into the AAV5 viral vector
.
AGN-151587 is currently undergoing clinical trials to treat congenital amaurosis (LCA) type 10 by delivering the CRISPR gene editing system directly to the eye to delete genetic mutations that cause hereditary blindness
.

Congenital amaurosis is a type of hereditary retinal degenerative disease.
Genetic mutations in at least 18 genes can cause this disease
.
It is the most common cause of hereditary blindness in children
.
Editas Medicine's product pipeline mostly focuses on the treatment of rare ocular diseases
.

Thanks to the success of NTLA-2001, Editas stock rose 6% that day
.

In recent years, gene editing technology represented by CRISPR/Cas9 has brought revolutionary changes to life medicine.
People have high hopes for new technologies such as gene editing and gene therapy, and they look forward to showing success in clinical treatment and overcoming chronic diseases
.
In addition, China's policy releases in the field of precision medicine are also very intensive, and a lot of support is given to technologies such as gene editing
.
For example, precision medicine will be upgraded to a national strategy, and 60 billion yuan will be invested in 2030
.
In April 2017, the "Thirteenth Five-Year" Biotechnology Innovation Special Plan was issued, pointing out the development of "a new generation of genetic manipulation technology"
.

As a new field of medicine, gene editing therapy is profoundly changing the face of the pharmaceutical industry, but it has not yet matured.
Humans need to explore and discover for a longer period of time, such as off-target effects and human application.
ethical and safety issues
.

Of course, supervision, approval, pricing, payment, etc.
are also problems that will inevitably be encountered in the commercialization of gene therapy, and long-term exploration and discovery by all parties are required
.
It is believed that in the future, with in-depth research and technological advancement, regulatory policies and payment systems are gradually improved, gene therapy will shine
.

reference:

1.
Intellia and Regeneron Announce Landmark Clinical data Showing Deep Reduction in Disease-Causing Protein After Single Infusion of NTLA-2001, an Investigational CRISPR Therapy for Transthyretin (ATTR) Amyloidosis.
Retrieved June 26, 2021, from https://ir.
intelliatx.
com/news-releases/news-release-details/intellia-and-regeneron-announce-landmark-clinical-data-showing;

2.
Creating transformative gene-based medicines for serious diseases;

3.
The official website of each company
.