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A Small Study Raises Big Hopes That Gene-Editing Can Treat Blood Diseases

November 19, 2019, 10:18 PM UTC

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Sometimes, a small study can raise big expectations—especially in the nascent industry of gene-editing to treat serious diseases.

Such is the case for CTX001, a closely-watched therapy from upstart biotech CRISPR Therapeutics and its much larger partner, rare disease specialist Vertex Pharmaceuticals, that was developed using CRISPR gene-editing technology.

The early-stage trial in question treated just two—yes, two—patients with the blood disorders sickle cell disease and beta-thalassemia, respectively. But the companies found the initial results so compelling that, even at this preliminary juncture, they decided to share some of the information.

“The data we announced today are remarkable and demonstrate that CTX001 has the potential to be a curative Crispr/Cas9-based gene-editing therapy for people with sickle-cell disease and beta-thalassemia,” said Vertex CEO Jeffrey Leiden in a statement.

Just what did the data show? For one, the patient with beta-thalassemia (which can cause severe anemia) went from requiring more than 16 blood transfusions per year before treatment to none nine months after treatment.

The patient with sickle cell disease had zero “vaso-occlusive crises” (incidents where the warped blood cells caused by sickle cell can lead to severe pain and obstructions) four months after treatment. That individual had been experiencing about a half dozen incidents every year prior to receiving CTX001.

It’s always prudent to be cautious about these early-stage results, no matter how impressive they sound. But several analysts, including from RBC Capital Markets, sounded cautious optimism.

Investors cheered, too. Shares of Vertex (a $50 billion-plus market value company) rose more than 2% in Tuesday trading, while shares of CRISPR Therapeutics spiked nearly 17%.

Read on for the day’s news.

Sy Mukherjee
sayak.mukherjee@fortune.com
@the_sy_guy

DIGITAL HEALTH

The Fortune Businessperson of the Year who embraces the robots. Fortune released its latest Businessperson of the Year list this morning (you can peruse the entire thing here, especially if you want to find out who took the number one spot). But of the 20 extraordinary businesspeople who made this year's cut, one may stand out to Brainstorm Health readers: Gary Guthart, CEO of Intuitive Surgical, the robotic surgery giant that doesn't appear to show any signs of diminished growth despite a growing cadre of competitors. Here's why he made the list. (Fortune)

INDICATIONS

China's drug regulation reforms prompt shift in Novartis' strategy. Recent regulatory reforms in China have sped up drug approvals in the country so rapidly that pharmaceutical giant Novartis is shifting its focus there from early-stage discovery to later-stage development. "We have decided to focus our R&D activities in Shanghai on expanding the scale and scope of our early clinical development and later stage clinical trial operations to accelerate the development of new medicines," said Novartis in a statement. "As a result, we will exit early discovery research at the site." (Reuters)

THE BIG PICTURE

Philippines latest nation to plan crackdown on e-cigs (and a potentially harsh one). The Philippines are joining India, China, and others in cracking down on vaping and e-cigarette use. President Rodrigo Duterte announced he would be proposing a ban after a case of lung disease related to e-cig use. He also went a step further, warning the country may arrest people who vape in the future. (NPR)

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