2019: CRISPR genera and the emerging genital gene are age



Now, the first CRISPR treatments make their way to clinical trials. In November, the Scottish Food and Drugs Administration (FDA) agreement requests for Research Drugs (IND) Editas Medicine for a 1/2 CRISPR Allergan-partnered drug EDIT-101 drug test for LCA10, type scarce problem without healing effect.

Green light came after delays around a year caused by manufacturing issues, which allowed another candidate from CRISPR Therapeutics and Vertex Pharma – CTX001 for the rare blood disease of beta thalassemia – to become the first CRISPR drug to start testing in Europe. The FDA added the CTX001 in beta thalasssemia and a test of a cell disease disease on clinical retreat shortly after, but stopped on October last.

Both candidates can both be based on CRISPR, but they have huge differences. CTX001 is an ex-therapy, which means
CRISPR technology is used to make changes to the external cellular cellular cells to express fetal hemoglobin, and then return to the patient. The aim is to make red blood cells that give up the fetal hemoglobin change that will cause; cell tension that causes hemoglobin for adults uninhabited.

Compared to that, Editas is a cure in vivo, with the CRISPR delivered directly under the eye vein that uses a viral vector (AAV) that is attached to the adeno, the only delivery vehicle is determined in Gene therapies (and the technology that caused the FDA to raise issues that delayed the start of the test). The idea is to alter the photoreceptor cells in the eye to eliminate borting in the CEP290 gene that causes LCA10. The company says that this is a # 39; The first test of the CRISPR drug that will be held anywhere in the world.

However, Editas and all generations of generation have recently been demolished with two special problems – retiring as Edita's Head, and the failure of the windows-so zinc-finger rival Sangamo Therapeutics (ZFNs) platform.

However, the first medicine use of CRISPR from the biology team of the US did not. Back in 2016, Chinese scientists were led by the Lu You oncologist at the Sichuan University in Chengdu, CRISPR cells were altered in patients with a type of lung cancer at the West China Hospital. The cells were edited due to the lack of PD-1, the defensible inspection area that was targeted on drugs such as Merck & Keytruda (pembrolizumab) and Bristol-Myers Squibb Opdivo (nivolumab) , and hoped that – without breaking PD-1 – the modified cells would attack and turn it off; eliminating cancer. The journalistic work was published in the journal Nature and, since then, another Chinese group has examined a similar approach called head and mound.

At the same time, CRISPR is not the only generation technology that is used in human theoretics, although its advocates say that it is the simplest, easiest and cheapest way to use.

Ro CRISPR, which includes nucleink zinc-finger (ZFNs) and TALE (TALENs) nuclei in the genome engineering toolbox has already allowed to generate genius in higher organisms such as cells human. These enzymes do not include double DNA breaks and they will be innovative to identify specific DNA targets that they want. The cell is trying to & # 39; breakdown by using another copy of the series as a backup, such as the other unlocking cromosome in the # 39; couple but by installing a new template, the system can be submitted for an order required.

The ZFN approach has been initiated by Sangamo Therapeutics, but company shares have been reduced early in February after interim results have been made; show that the effective cure against its rare genetic disease target. The study of stage 1/2 CHAMPIONS showed that single injection of SB-913 for the type II mucopolysaccharidosis (MPS II) or Hunter syndrome-type illness caused the disease IDS. However, the cure can not affect bio-marketers that would be used to effectively assess MPS II drugs, and The company now proposes to have regard to the second generation of the ZFN design that it hopes to be more powerful.

Sangamo Chief Executive Sandy MacRath (below) tried to assume owners that the results were a step on the way to a living generation curation which is "the most difficult, most recent way technology ", but the stock largely lost a third of the value, including inventories, had expected to be an effective signal.

SAndy MacRath

TALENs work in a similar way to the ZFNan and, as long as they are likely to become larger molecules, they are considered easier and cheaper.

Until now, the TALEN application has not been made largely in ex vivo messages, starting in 2015 when researchers from Great Ormond Street Hospital in London used the technology for its use. first time as a therapy. An 11-month-old child suffering from CD19-positive Droma-energy-leukemia (A ALL) was treated with enhanced authorized cells that were made to attack leukemia cells.

TALEN is now widely used in T-receptor antigen (CAR-T) chemical remedy, which includes the exhaustion of T cells from patients, adapted to attack tumor cells with identifying antigons on the surface, and then increasing the number and its; including them to patients.

Companies like Cellectis and Allogene partners use TALEN to take a slightly different approach, and & # 39; Using a generation toolbar to create a CAR-Ts that does not require the harvest level. At the meeting of the Hematology American Asiatic Society (ASH) this year, Allogene and the Servant participated in stage 1 data for the APPRO19 candidate in ALL who showed very good response rates.

But the method is also proven as an in vivo cure for human papillomavirus (HPV) cervical cancer made by researchers in China, which compares the Plasmid TALEN to CRISPR medicine .

Safety issues

As with the emerging new technology, there is still enough to consider safety as to how; first human exams to start & # 39; Generating results – even before making an unscrupulous application for sending scientific messages.

For example, there is no doubt that CRISPR- Cas9 has made a gene cutting very easy, but the device has been detected more precisely than previously taken. A Dutch team recently revealed that it can encourage a well-known repair tool that can prevent the edited editing; Expectations and also that depressed genes are being implemented, which may be adversely affected – for example if a gene gene has to say.

At the same time, earlier this year, found another organization at the Wellcome Sanger Institute that the method of deleting it can be deleted much longer than expected, Suggestions that could have trouble & # 39; and that there needs to be a detailed study of remedies that are based on the harmful effects approach.

Genealogy drug developers have been identifying the results but they say that antibiotics such as cancer have not been seen in prehistoric checks and animal model, regardless of the technology that was used . It is clear that every test of a generation that is carried out in the west will be rigorous, both before they start and after the results are available.

Everyone knows, what is obvious, although he has a remarkable rehearsal test, with reports that he is now under a home host in China, he is responsible with companies such as Editas, CRISPR, Sangamo, Cellectis / Allogene and their peers to take the slow way to prove that generating can provide effective and effective treatment for patients with depressing diseases.


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