The drug, meant to complement other treatments for paroxysmal nocturnal hemoglobinuria, comes via Alexion, which had acquired it from Achillion in 2019.

Preliminary study results show the biotech’s gene editing treatment could reduce markers and symptoms of hereditary angioedema, though the data come from few patients and follow-up remains limited.

The company is planning an FDA submission as it works to expand its vaccine business beyond top-selling shots for COVID-19 and pneumonia.

A gene discovery four years ago led to a partnership between the companies, which plan to start a Phase 2 study later this year after encouraging early data. 

The public-private partnership plans to bring together scientific experts, private entities and patient groups to better understand certain neurodegenerative disorders and find new treatments. 

The startup’s $185 million initial public offering is the largest for the sector since early May and potentially a sign of renewed investor interest in young biotechs.

Capstan Therapeutics was formed by pioneering CAR-T and messenger RNA researchers, and has secured funding from five large drugmakers.

The trial, expected to enroll 25,000 adults, is the second late-stage test of an mRNA flu shot to begin this year, following the June start of a Moderna study.

The joint venture with Radiopharm Theranostics is pursuing a field of research that’s seen a string of recent deals and investments by large drugmakers. 

The well-funded startup is planning Phase 3 trials for a drug it claims could be superior to top-selling medicines from Novartis and others.

The biotech will reduce staff by 75% and end development of two drugs, the latest setback for a company that was worth nearly $2 billion when it went public in 2018.

The development comes three weeks after Roctavian was approved in Europe and ahead of a planned regulatory submission in the U.S. Drug regulators have not ordered a trial hold, however.