Immusoft of CA, a clinical-stage biotechnology company pioneering engineered B cell therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to ...

Discover why Denali Therapeutics Inc. earns a Strong Buy with FDA-priority drugs, solid pipeline, and a strong cash position.

India’s rare disease care relies on crowdfunding as costly therapies outstrip public support. Government aid is capped and ...

Completion of the agreement remains contingent on several closing conditions.

Learn more about whether Immunocore Holdings plc or Ultragenyx Pharmaceutical Inc. is a better investment based on AAII's A+ ...

A Downham Market mother whose 11-year-old son died from a rare genetic disorder has spoken out for the first time ahead of a ...

Denali Therapeutics and Royalty Pharma entered a $275 million royalty funding agreement based on future sales of tividenofusp alfa.

Shares of US drug developer Denali Therapeutics closed up 5.8% at $20.11 yesterday, after it announced a $275 million ...

Now Ollie is making history as the world’s first successful gene therapy patient for this cruel condition. Ten months after receiving revolutionary treatment at Royal Manchester Children’s Hospital, ...

In return, Royalty will receive a 9.25% royalty on worldwide sales of tividenofusp alfa from Denali until it reaches three times the amount Royalty has handed to Denali—or, alternatively, 2.5 times ...

A California toddler is the first person in the world to receive gene therapy to treat his devastating disease. Three-year-old Oliver Chu was born with a rare, genetic condition called Hunter syndrome ...

Denali Therapeutics Inc. DNLI has announced a funding agreement with Royalty Pharma plc RPRX for $275 million. The stock gained 5.84% following the announcement. The deal centers on future net sales ...