Orphazyme has fresh cash from the sale of shares in the US and Europe, which will support its lead rare disease drug through regulatory review and, if approved, a launch. But the drug fell short in key clinical trial measures, raising questions about its approvability. Now the FDA wants additional data, potentially throwing a wrench […]

Praxis Precision Medicines kept mostly quiet about its research on central nervous system disorder (CNS) drugs until May, when it emerged from stealth and revealed $100 million in cumulative financing. Now the company is aiming to raise about that amount in an IPO that will fund clinical development of its drug pipeline. Cambridge, MA-based Praxis […]

Had things turned out differently, scientists at Galecto might be watching their idiopathic pulmonary fibrosis drug candidate develop in the hands of a big pharmaceutical company. But Bristol Myers Squibb passed on its option to acquire Galecto leaving the biotech to forge ahead on its own. Now the company has $64 million to advance clinical […]

Many drugs work by binding to a protein and blocking it. Technology for eliminating disease-causing proteins altogether remains experimental, but this approach has gained ground in the past year with hundreds of millions of dollars raised and compounds advanced into clinical testing. Monte Rosa is one of the newer companies in this “protein degradation” space […]

Amyotrophic lateral sclerosis (ALS) has no cure and few available treatments but the past decade has revealed new knowledge about the pathology of the disease. Libra Therapeutics is turning that research into potential therapies, and it now has $29 million to advance its work. While the exact causes of many neurological disorders are unknown, Libra […]

Gene therapy product developers are seeking more clarity from the US Food and Drug Administration on when viral vectors from the same class will be considered sufficiently different for purposes of awarding orphan drug designation or exclusivity. In addition, stakeholders want the FDA to better define, and provide examples of, the types of minor differences […]

The 2020 National Xconomy Awards complimentary two-week, 11-part, on-demand Meet the Finalists daily webcast series continues this Tuesday and Wednesday at 11 a.m. ET. Registration is complimentary and includes access to a networking app with already more than 600 life science contacts. The daily series leads up to the Thursday, Sept. 24 free online National Xconomy Awards […]

Gilead Sciences CEO Daniel O’Day has spoken openly about acquisitions as a way to build up the company’s drug pipeline. This week, it made its biggest buy yet. The $21 billion Immunomedics acquisition brings to Gilead a cancer drug that many analysts project will become a blockbuster seller. Some of those analysts think Gilead might […]

Dyne Therapeutics, a company developing genetic medicines for rare muscular disorders, has raised $233 million in an IPO that topped the preclinical-stage biotech’s projections. Late Wednesday, Dyne priced its offering of about 12.3 million shares at $19 each. The Waltham, MA-based biotech had previously planned to sell 10.3 million shares in the range of $16 […]

An Arrowhead Pharmaceuticals drug for a rare liver disease now has early Phase 2 results and the data so far look good. Not only did the therapy reduce levels of a mutant protein associated with the disorder, but patients also showed improvements in several biological measures of liver injury. The results are for just four […]

Cell therapy offers another option for addressing the most difficult blood cancer cases, but such treatments, which are engineered from a patient’s own immune cells, don’t yet work on solid tumors. Neogene Therapeutics is developing technology with the potential to bring cell therapies to solid tumors and it now has $110 million to advance its […]

Mark Rothera has been appointed president and CEO of Silence Therapeutics (NASDAQ: SLN). He comes to London-based Silence from Orchard Therapeutics (NASDAQ: ORTX), where he was chief executive. Rothera’s experience also includes positions at PTC Therapeutics (NASDAQ: PTCT), Aegerion Pharmaceuticals, and Shire. Rothera is starting at Silence one week after the company debuted on the […]