Biohaven Pharmaceutical said on May 23 the company’s experimental drug for patients with spinocerebellar ataxia, a genetic disease that affects the nervous system, failed to meet the main goal of a late-stage study.

The U.S. Food and Drug Administration granted full approval to Orphalan’s Cuvrior (trientine tetrahydrochloride) for the treatment of Wilson’s disease, a rare genetic disorder. The approval announcement marks the first new treatment available for patients in over five decades.

Following the ongoing trend in the pharmaceutical industry, bluebird bio orchestrated a restructuring that includes saving financially and cutting staff. The restructuring is designed to prolong the company’s budget as bluebird bio awaits decisions from the U.S. Food and Drug Administration (FDA).

Shares of Taysha Gene Therapies were down more than 10 percent after a patient’s death marred positive interim data for the company’s experimental gene therapy treatment for Sandhoff and Tay-Sachs diseases, which are two forms of GM2 gangliosidosis.

ProQR Therapeutics is collaborating with Indianapolis-based Eli Lilly to bolster the Dutch biotechnology company’s Axiomer RNA editing platform and create new medicines that could treat a range of genetic disorders. 

The U.S. Food and Drug Administration placed a clinical hold on BioMarin Pharmaceutical’s BMN 307 Phearless Phase I/II study.

Toronto-based biotech Deep Genomics landed $180 million in the company’s oversubscribed Series C.

Shape Therapeutics generated $112 million from a recent Series B financing round the Seattle biotechnology firm co-led with Decheng Capital and Breton Capital.

The U.S. Food and Drug Administration rejected Orphazyme’s key drug candidate for Niemann-Pick disease type C (NPC), causing the Denmark-based biopharmaceutical company’s “meme stock” to decline in trading. 

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.