Mar 30, 2017

Startup raises $50 million to fight Duchenne muscular dystrophy

Charles Williams / Flickr Creative Commons

Solid Biosciences, a Cambridge, Mass.-based drug developer focused on Duchenne muscular dystrophy, has raised $50 million in Series C funding co-led by RA Capital Management and Bain Capital Life Sciences. The company was founded by a former J.P. Morgan banker, after his son was diagnosed with DMD.

Why it's a big deal: DMD is a fairly rare genetic disorder, reportedly affecting around 1 out of every 3,500 young boys in the U.S., but that hasn't slowed down the pharma interest. In addition to Solid Biosciences, there has been work by both Sarepta Therapeutics (Nasdaq: SRPT) and Marathon Pharma. If that last one sounds familiar, that's because Marathon is a private equity-backed company accused of price-gouging on its DMD drug (we discussed it here). Within the past few weeks, Marathon agreed to sell its treatment to PTC Therapeutics (Nasdaq: PTCT) for a reported $140 million in cash and stock.

Bottom line: "Gene therapy—a method of using a virus to shepherd genetic instructions into the body—has been tried for Duchenne before, though never successfully. But Solid's program is one of a new group of emerging gene therapy and gene editing programs being advanced by academic groups, startups, and larger companies." ― Ben Fidler

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Mass shooting in Milwaukee: What we know

Milwaukee Mayor Tom Barrett in 2012. Photo: John Gress/Corbis via Getty Images

Six people died in a shooting at the Molson Coors Brewing Company in Milwaukee on Wednesday, including the gunman, Mayor Tom Barrett told reporters at a Wednesday evening press conference with local police.

Details: All of the victims worked at the brewery complex, as did the shooter who died of "an apparent self-inflicted gunshot wound," police confirmed in a statement late Wednesday.

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Coronavirus updates: South Korea case count tops 2,000

Data: The Center for Systems Science and Engineering at Johns Hopkins, the CDC, and China's Health Ministry. Note: China numbers are for the mainland only and U.S. numbers include repatriated citizens.

33 people in California have tested positive for the coronavirus, and health officials are monitoring 8,400 people who have recently returned from "points of concern," Gov. Gavin Newsom said Thursday.

The big picture: COVID-19 has killed more than 2,850 people and infected over 83,000 others in some 50 countries and territories. The novel coronavirus is now affecting every continent but Antarctica, and the WHO said Wednesday the number of new cases reported outside China has exceeded those inside the country for the first time.

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Syria's darkest chapter

Family room without a family, in Idlib. Photo: Muhammed Said/Anadolu Agency via Getty Images

The worst humanitarian crisis of Syria’s brutal civil war is colliding today with what could be the war’s most dangerous geopolitical showdown, after at least 29 Turkish troops were killed in an airstrike.

The big picture: The fighting is taking place in Idlib in northwest Syria, where a ferocious Syrian and Russian offensive has displaced 1 million civilians and infuriated Turkey, which borders the region.

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