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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

Go deeper

5 mins ago - World

U.S. envoy to visit Sudan as "most dangerous" crisis intensifies

The sit-in in Khartoum. Photo: Mahmoud Hjaj/Anadolu Agency via Getty

U.S. envoy for the Horn of Africa Jeffrey Feltman will visit Khartoum this week amid what Prime Minister Abdalla Hamdok has called the “worst and most dangerous" crisis of Sudan’s transition to democracy, two sources with direct knowledge tell Axios.

Driving the news: Roughly 2,000-3,000 people had joined a sit-in in Khartoum as of this afternoon, per Reuters, after protesters massed over the weekend to call on the military to bring down the government. The protests came just four weeks after a failed military coup.

Trump sues National Archives, Jan. 6 committee to block records request

Photo: Chip Somodevilla/Getty Images

President Trump filed a lawsuit Monday seeking to block the National Archives from releasing White House records to the House select committee investigating the Jan. 6 Capitol insurrection, citing executive privilege.

Why it matters: It's the latest escalation in Trump's campaign to disrupt the committee's sweeping probe into the circumstances surrounding Jan. 6, including his actions and communications leading up to the Capitol attack.

Obama says Powell exemplified what America "can and should be"

Then-President Obama speaks alongside former Secretary of State Colin Powell during a meeting in the Oval Office in 2010. Photo: Saul Loeb/AFP via Getty Images

Former President Obama called Colin Powell an "exemplary soldier and an exemplary patriot" in a statement honoring the former general following his death from COVID-19 complications on Monday.

Why it matters: Powell, the first Black U.S. secretary of state, was known as a Republican but played a critical role in helping Obama get elected in 2008.

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