Crispr infuses the first human in a landmark study on gene editing


(Bloomberg) – The shares of Crispr Therapeutics AG surged after the company said it had treated the first human being with the same genetic technology and gave its name to a preliminary study.

Crispr Therapeutics and its partner Vertex Pharmaceuticals Inc. said Monday morning that the first patient of a trial using CTX001, a therapy created using Crispr technology, as a treatment for rare blood disease , beta-thalassemia, had received the drug for single use. The couple also announced that the recruitment of the first patient had begun in a parallel study on the treatment of sickle cell disease, whose first dose was on track for the middle of the year.

"Treating the first patient in this study marks an important scientific and medical milestone and the beginning of our efforts to fully realize the promise of CRISPR / Cas9 therapies as a new class of potentially transformative drugs for the treatment of serious diseases," Samarth Kulkarni , chief executive officer of Crispr Therapeutics, said in a statement.

After the announcement, Crispr Therapeutics grew 20% to reach $ 38.10 at 9:56 am in New York. This is the largest intra-day movement recorded for more than a year. Peers Editas Medicine Inc. and Intellia Therapeutics Inc. were up 12% and 7%, respectively, after the update and a pair of biopharmaceuticals.

Technology has a wide range of applications and has captured the imagination of investors for much of the past 18 months, when it was only used on animal models. Crispr led the pack in stock movement and market valuation last year, when the word "Crispr" spread like wildfire in traders' conversations.

However, a series of scientific articles questioning the application of technology, the release of Edites' general manager and a more general verification of the reality that these drugs are years of US approval have weighed on the trio of listed Crispr stocks.

Crispr shares had lost about a quarter of their value in the last year before today's announcement, while Editas and its Intellia counterpart were down about 45% in the same segment. This underperformance contrasts sharply with the Nasdaq Biotechnology Index, a benchmark for the sector, which has hardly changed since the market closed in late 2018.

This news comes a few weeks after Sangamo Therapeutics Inc., a partner gene publisher, uses a different platform to repair the disease-causing DNA, known as Zinc Finger Nuclease, has surprised the community investors with interim results in a benefit study for patients. The results triggered a sell-off in the Crispr-focused stock basket and rekindled some bearish expectations that the drug's effectiveness may not be all that it had been announced.

(Updates lead, add a graph, share the move in the fourth paragraph, add peers and context throughout.)

To contact the reporter on this story: Bailey Lipschultz in New York at [email protected]

To contact the editors responsible for this story: Catherine Larkin at [email protected], Courtney Dentch

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