Aeglea BioTherapeutics announced on April 11 promising Phase III results for patients diagnosed with arginase 1 deficiency (ARG1-D), a rare hereditary metabolic disorder. Aeglea said on April 12 the company had submitted its first-ever Biologics License Application (BLA) to the U.S. Food and Drug Administration for the drug.

Investors Hand Disc Medicine $90 Million to Control Hematological Diseases Published: Sept. 2, 2021 By Heather McKenzie BioSpace   A two-year-old biopharma company tackling rare blood diseases has received a […]

AstraZeneca

AstraZeneca said on Aug. 26 a late-stage trial showed the company’s medicine for a rare disease that causes accumulation of copper in the body was three times more successful in helping remove deposits from tissues compared with standard care.

Pivoting from conflicting and controversial press over the company’s approved Alzheimer’s disease treatment, Biogen announced that its gene therapy drug timrepigene emparvovec failed to show a clinically meaningful benefit for a rare inherited eye disease in a Phase III trial.

Find out which biopharma companies are raking in the cash this week, as companies from around the globe provide updates on their financing rounds and IPOs.

The U.S. Food and Drug Administration approved Clinuvel Pharmaceuticals Ltd.’s treatment for a rare inherited disorder that causes skin damage from exposure to light, sending the Australian drugmaker’s shares to a record high.

Deep Genomics’ proprietary artificial intelligence-based drug discovery platform identified a novel treatment target and corresponding drug candidate for the rare and potentially life-threatening genetic disorder Wilson disease.

Pfizer acquired a 15 percent stake in Vivet Therapeutics as well as an exclusive option to fully acquire the business, a deal that will give the New York-based pharma giant access to the French company’s experimental gene therapy for a rare genetic disorder.