The latest data from a Phase I/II study shows that pegtibatinase can yield a rapid, sustained reduction in homocysteine, the protein behind the underlying cause of the rare disease.

Almost 80% of novel agent submissions from the biggest companies were approved in recent years, compared with a 60% approval rate for the rest of the industry, according to a Pink Sheet analysis of FDA approval decisions since 2021.

Public Company Edition: With the XBI up more than 9% in April, a few follow-on offerings –$23m to support Gamida Cell’s small product launch and $500m to fund Vaxcyte’s large clinical trial program – have made it to market. Also, China’s CytoMed launched the sixth biopharma IPO in the US this year.

Aspect says its bioprinted tissue therapeutics include immune-resistant encapsulation, which Novo Nordisk hopes will offer a safer approach to type 1 diabetes cell therapy.