Ultragenyx’s Dojolvi wins FDA approval for rare disease FDA approved Dojolvi triheptanoin (UX007) from Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) to treat long-chain fatty acid oxidation disorders, making it the first drug approved for the indication. The...

The latest in a string of Qiming portfolio companies to go public, Gan & Lee saw its stock gain the maximum allowed by the Shanghai exchange in its first day of trading to bring its...

DISEASE CATEGORY: Musculoskeletal INDICATION: Myotonic dystrophy type 1 (DM1) Inhibiting the transcription elongation regulator CDK12 could treat DM1, which is caused by CTG repeat expansions in the DMPK gene that result in cellular accumulation of...

CSL gets global rights to uniQure gene therapy  CSL Ltd. (ASX:CSL) is paying $450 million up front for an exclusive, worldwide license to commercialize etranacogene dezaparvovec (AMT-061) from uniQure N.V. (NASDAQ:QURE), which is also eligible...

ArcherDX picks deal with Invitae over IPO  Genetic testing company Invitae (NYSE:NVTA) is acquiring ArcherDX Inc., adding a cancer diagnostics platform to its precision oncology business. ArcherDX’s shareholders will receive $325 million in cash plus...

With a first-day stock move that nearly tripled its share price, SinocellTech became the highest-valued biotech traded on Shanghai’s STAR market since the launch of its biotech chapter last year. SinocellTech Ltd. (Shanghai:688520) climbed 185%...

Prosit Sole is improving upon protein and cytokine therapies against validated targets for rare diseases using its chimeric protein engineering platform. The idea is to combine desirable structural features from different protein family members to...

Novavax Inc. (NASDAQ:NVAX), which expects Phase I data for its COVID-19 vaccine in July, hired Filip Dubovsky as SVP and CMO. He succeeds CMO Louis Fries, who has retired. Dubovsky was head of clinical engagement...

A first from Ultragenyx in a rare disease  FDA approved Crysvita burosumab-twza from Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) to treat tumor-induced osteomalacia in patients age two and older. The anti-FGF23 mAb is the first to be...

In response to a court decision overturning mandatory disclosures of drug pricing in television ads, Sens. Chuck Grassley and Dick Durbin are pushing legislation that would achieve similar goals. On Tuesday, the U.S. Court of...