Tag: Editing

Categories
Health

CRISPR gene enhancing may attain sufferers ‘very quickly’: Intellia CEO

CRISPR gene editing could reach patients ‘very soon’: Intellia CEO

Following a breakthrough trial where gene-editing technology CRISPR completed its first systematic delivery as medicine to a human body, Intellia Therapeutics CEO John Leonard said he hopes the gene therapy could be made available to patients “very, very soon.”

“These approaches are subjected to the standard sorts of clinical trials that any drug or gene therapy would be studied under, so we’re in the earlier stages of that,” Leonard said on CNBC’s “Closing Bell” on Thursday afternoon.

He added that over the next few years, the company expect the medical technology to be subjected to standard reviews, “but our hope is that this will be available to patients very, very soon.”

CRISPR, or clustered regularly interspaced short palindromic repeats, effectively cuts genomes and slices DNA to treat genetic diseases.

The latest development, the result of a trial between Intellia and biotech company Regeneron, treated a rare disease after being given as an IV infusion. Previously, other applications of the CRISPR technology had been limited to ex vivo therapy, or where cells are removed from the body for genetic manipulation in a laboratory and then reintroduced to the body.

“What’s particularly exciting about that is we were able to completely inactivate that gene and see that in the clinical effects of the patient, so a major advance in the gene editing space,” Leonard said.

Heart, diabetes and broad disease implications

CRISPR has broad applications, and Leonard said there is a lot of work being done to target some of the most common diseases and causes of death, such as heart disease and diabetes.

“The challenge is getting into those particular genes that cause disease, so we started in the liver, which is an area where there are many problems with disease-causing genes, and it’s been shown that we can reach that very, very successfully,” Leonard said. “There’s other tissues after that that we’re pursuing, especially the bone marrow, where a long list of blood-borne-type diseases can be addressed.”

A key for CRISPR is targeting diseases that are monogenic, or caused by one particular gene, allowing this type of gene-editing therapy to be successful, Leonard said. Other diseases that are polygenic, such a cancers or autoimmune diseases, will be “more difficult to tackle,” he added.

A researcher watches the CRISPR/Cas9 process through a stereomicroscope at the Max-Delbrueck-Centre for Molecular Medicine.

picture alliance | picture alliance | Getty Images

The new treatment is still in the early stages and it has not been priced yet, but as it develops, Leonard said he believes it will be “very valuable for patients and probably resource sparing for the health care system overall.”

“It really comes down to the some of the advantages with single application where literally it’s a one-and-done therapy,” Leonard said. “We expect over time this will be generally very, very favorable in the economics of this entire field.”

Jennifer Doudna, who was awarded the 2020 Nobel Prize in chemistry for her work on CRISPR gene editing and is the co-founder of Intellia, recently told the CNBC Evolve Global Summit that cost is a significant challenge, and in the case of sickle cell anemia, where CRISPR has had early success, treatment can still be $2 million.

“That is clearly not a price point that will make this available to most people that can benefit from it,” she said. Innovations in delivery of CRISPR may help lower cost, but Doudna also said that the medical field needs to figure out how to “scale the molecule production so that we reduce costs.”

She told CNBC the evolution of the technology from the publication of her early work to clinical trials showing it to be effective in treating diseases in less than 10 years represents, “One of the fastest rollouts I think of technology from the fundamental, initial science to an actual application.”

“It’s largely because the technology comes at a moment when there’s enormous demand for genome editing, as well as a lot of knowledge about genomes,” Doudna said.

Categories
Health

How CRISPR gene enhancing will deal with illness: Intellia founder Doudna

How CRISPR gene editing will treat disease: Intellia founder Doudna

The gene editing technology CRISPR reached an important milestone last weekend and completed its first systemic drug delivery to the human body.

CRISPR, or clustered, regularly spaced short palindromic repeats, cuts genomes and cuts DNA effectively to treat genetic diseases.

The latest breakthrough, the result of a study between biotech company Regeneron and Boston startup Intellia Therapeutics, treats a rare disease after it has been given as an IV infusion. Previously, other uses of CRISPR technology have been limited to ex vivo therapy, or where cells are removed from the body for genetic manipulation in a laboratory and then returned to the body.

Jennifer Doudna, who was awarded the Nobel Prize in Chemistry in 2020 for her work on CRISPR gene editing and is the co-founder of Intellia, recently told CNBC about the development of the technology from the publication of her early work to clinical studies showing its effectiveness at treating disease in less than 10 years means “one of the fastest rollouts in my opinion of technology from basic, initial science to actual application.”

“This is mainly because the technology comes at a time when there is a huge demand for genome editing and a lot of knowledge about genomes,” said Doudna at the recent CNBC Global Evolve Summit in mid-June.

As for next, Doudna highlighted several challenges and opportunities that CRISPR has on the horizon.

Deploying CRISPR remains a major challenge

As technology continues to advance, the task of getting the processed molecules in the body to the cells in the areas where they are needed remains a challenge.

“This is a particular issue in clinical medicine where the ability to manipulate brain cells, heart cells or muscle cells has incredible potential, but right now we don’t really have the tools to introduce the editors to those cells,” said Doudna. “We have the editors; we just don’t know how to get them where they need to go.”

Sickle cell anemia was an early focus

Much of the success of CRISPR’s previous applications has been in ex vivo therapy, in which extracted cells are manipulated in a laboratory and then returned to a patient.

Sickle cell anemia, which is genetically inherited and affects approximately 100,000 Americans, according to the CDC, was a particularly good target for the technology because blood stem cells “can be harvested, processed, and then returned to patients,” Doudna said.

Genetic eye diseases were also a focus for CRISPR applications, as Doudna said, “It is certainly easier to get into the eye than other parts of the body.”

The delivery of the processed cells to the liver has also proven to be easier so far. “A liver is an organ that naturally accepts molecules in the body,” she said.

Any advance in eradicating the 100+ liver diseases could have a huge impact on the lives of Americans. According to the American Liver Foundation, at least 30 million people, or one in ten Americans, have liver disease.

Next, let’s focus on the brain, heart, muscles

The next step for innovation around CRISPR will be to move these cells to other parts of the body like the brain, heart and muscles, Doudna said.

“There are already some technologies that make some of this possible, for example with different types of viruses or virus-like particles, and I look forward to the innovations that will come in this regard over the next few years,” she said.

Treatment costs are a problem

But as technology improves and scientists become able to fight disease throughout the body, Doudna said that CRISPR technology needs to be cheaper in order for it to have “widespread effects.”

Treating sickle cell anemia with CRISPR therapy costs about $ 2 million per patient, according to Doudna.

“That is clearly not a price point that makes this available to most of the people who can benefit from it,” she said.

While addressing delivery challenges can also help reduce costs, the medical community needs to figure out how “to scale molecule production so that we can cut costs,” said Doudna.

Applying CRISPR to Agriculture

The advancement of CRISPR technology may have an impact on other industries as well, with agriculture being one of the first to benefit.

Rather than addressing genetic problems through breeding, which can take months to years, or through current methods of genetically modifying crops that have seen a boom over the past few decades but that incorporate biological material from other species, CRISPR technology can address the Genes from plants “without touch” manipulate everything else, “said Doudna.

“This opens the door to a lot of things that can be done now to both address the challenges of climate change, manage drought, and introduce properties into the plants that will protect them from pests,” she said.

Categories
Business

New York Occasions Names Cliff Levy to a Prime Modifying Position

New York Times Names Cliff Levy to a Top Editing Role

The New York Times announced on Wednesday a return to its leadership team in the newsroom with the appointment of its Subway editor, Clifford J. Levy.

Levy, 53, a two-time Pulitzer Prize winner, has been running the subway counter since 2018. Previously, he was deputy editor-in-chief of the Times’ online platforms and worked as head of the Moscow office and investigative reporter.

In a message to the newsroom on Wednesday, Dean Baquet, the editor-in-chief, and Joseph Kahn, the editor-in-chief said Mr. Levy would temporarily advise the audio division, home of the podcast “The Daily,” before moving on to a broader role. The audio division is overseen by Sam Dolnick, a deputy editor-in-chief and member of the Sulzberger family who control The Times, and Lisa Tobin.

Mr. Levy’s promotion comes a month after The Times released a correction for “Caliphate,” a 12-part audio series designed to shed light on the Islamic State. In an editor’s note, The Times said the podcast had too much faith in the misrepresentation or exaggeration of one of its main topics, Shehroze Chaudhry, a Canadian who claimed to have participated in atrocities by the Islamic State. On the day the note was published, Mr. Baquet described the problems with “Caliphate” as “institutional failure” and said his mistakes should not be blamed on “a reporter”.

“I or someone else should have done the same type of test because it was a big, ambitious piece of journalism,” Baquet said in a December interview with Michael Barbaro, the host of “The Daily”. “And I did not do this type of test, nor did my senior officers have extensive experience reviewing investigative reports.”

In their note on Wednesday, Mr. Baquet and Mr. Kahn said, “Cliff will spend the coming weeks learning the rhythms of ‘The Daily’ and the wider audio team, then helping Sam, Lisa and the Masthead better integrate with the daily Operation of the audio department in the wider newsroom. “

Business & Economy

Updated

Jan. 27, 2021, 11:46 ET

“One of his priorities is the development of new procedures for checking ambitious audio series,” the communication continues.

“The Daily” has become a central part of The Times, with four million listeners every weekday.

Times editors who hold the title of assistant editor-in-chief or assistant editor-in-chief are at the top of the editorial board, referred to by the editorial staff as senior masthead editors because their names appear along with the publisher at the top of page A2 of the print edition. AG Sulzberger and Mr. Baquet.

The number of names on Page 2 has increased in the last few months as 64-year-old Baquet approaches retirement age. Traditionally, Top Times editors have made high-profile posts before they are 66.

Carolyn Ryan, who heads the newsroom’s recruiting, strategy and high-profile journalism, became deputy editor-in-chief in October. The promotion followed her stations in charge of the newspaper’s political coverage, the subway division, and the Washington office.

With the return of Mr Levy to the crew, the newspaper has five assistant senior editors. The others are Rebecca Blumenstein, Steve Duenes and Matthew Purdy.

Mr. Kahn, the managing editor, ranks second after Mr. Baquet in the Times imprint. In December, national editor Marc Lacey was promoted to deputy editor-in-chief and one of seven journalists to hold the title. In the new role, Mr. Lacey is responsible for the live reporting.

While Mr. Levy was in charge of subway coverage, The Times won a Pulitzer Prize for a series by Brian M. Rosenthal that exposed predatory loans and other problems in the New York taxi industry. Mr Baquet and Mr Kahn said in their note on Wednesday that the search for a new subway editor was underway.