River Pugh, a three-year-old boy from Indiana, is fighting to survive Pompe Disease, a rare genetic disease that causes ...

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

The Oxford-Harrington Rare Disease Centre ('OHC') today announced the recipients of the 2025 Rare Disease Scholar Award, which advances promising discoveries from academic labs into clinical practice.

Ten scientists selected for second annual award to pursue innovations in gene, RNA, small molecule and cell therapies to treat rare and ultra-rare diseases OXFORD, England ...

Moderna is currently in the middle of an aggressive cost-cutting campaign. In September last year, the company announced that ...

Beam's broad early-stage pipeline and recent study progress take center stage as investors weigh its potential against rival NTLA's prospects.

Third quarter total revenue of $160 million, Crysvita® revenue of $112 million and Dojolvi® revenue of $24 millionReaffirm 2025 Revenue Guidance: ...

With much of Moderna’s rise in this decade tied to its COVID-19 vaccine, the company has struggled in recent years to match ...

After revoking Sarepta’s award in July and awarding one to Krystal last month, the FDA’s platform technology designation program appears to be back on track. These six biotechs could be on the ...

Moderna’s nearly $20 billion cash hoard has dwindled to a single-digit stash following a series of setbacks for the biotech.

Intellia Therapeutics NTLA has made good progress with the advancement of its in vivo candidate, lonvo-z (or, NTLA-2002), ...