The IPO train continues to barrel down the tracks as more life sciences companies announced and closed their initial public offerings, bringing millions more to their coffers.
Intellia Therapeutics and Regeneron Pharmaceuticals shook the biotech and CRISPR world on June 26, announcing positive early data from the first-ever patients to have their DNA edited with an in vivo CRISPR/Cas9 therapy delivered systemically.
The European Commission approved Roche’s injectible drug Enspryng, which can be administered at home to treat neuromyelitis optica spectrum disorder.
Shares of Vertex Pharmaceuticals fell in trading after the company announced it will not advance a novel small molecule corrector of the Z-AAT protein into a Phase III study for a rare disease that can lead to complications of the liver and lungs.
It’s hard to address something that you cannot define. Rare disease management is a high priority for payers. In a recent rare disease trend report, 90% of surveyed payer respondents ranked rare disease management as a high or moderate priority. This is due primarily to the significant budget impact of therapies that may be in the hundreds of thousands of dollars
The U.S. Food and Drug Administration granted marketing clearance for Ryplazim as the first FDA-approved therapy for the treatment of patients with the plasminogen deficiency type 1 (hypoplasminogenia), a rare genetic disorder.
Several biopharmaceutical companies submitted New Drug Applications (NDAs) to the U.S. Food and Drug Administration in recent weeks, covering treatment indications ranging from an opioid overdose to bipolar disorder and rare disease.
A first-of-its-kind organization launched on May 13 unifying the voices of life science companies committed to delivering desperately needed treatments to rare disease patients. The Rare Disease Company Coalition’s 10 founding members have brought 22 treatments to market and have more than 160 rare disease programs in the works.
Amryt inked an all-stock acquisition deal with Chiasma. The combined company will focus on rare and orphan diseases with three marketed products and a strong development pipeline.
On the back of a $135 million public offering in February, rare disease gene therapy company Krystal Biotech Inc. launched subsidiary Jeune Inc. to promote gene therapies for aesthetic skin conditions.
