Ultragenyx received a Complete Response Letter from the FDA for UX111 AAV gene therapy to treat Sanfilippo Syndrome Type A ...

Solid Biosciences' DMD gene therapy shows promise with SGT-003 and strong cash backing through 2027. Click here to read an ...

Ultragenyx (RARE) witnessed a jump in share price last session on above-average trading volume. The latest trend in earnings ...

The deal gives AstraZeneca’s rare disease unit Alexion access to specialized capsids developed by the Japanese biotech JCR ...

The draft is done. So is development camp. Free agency — never much of a frenzy to begin with — has mostly wrapped up, too.

Investing.com -- Klotho Neurosciences, Inc. (NASDAQ: KLTO) stock surged 22% after the company announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its ...

Fintel reports that on July 11, 2025, B of A Securities initiated coverage of Taysha Gene Therapies (NasdaqGS:TSHA) with a ...

CIBC’s Canadian REITs Monthly report focused on the confusion caused by tariffs, ...

Merck & Co. Inc. has strengthened its cardiopulmonary pipeline with its $10 billion acquisition of London-based-Verona Pharma plc. The deal brings Merck Ohtuvayre (ensifentrine) for chronic ...

The Pittsburgh Penguins make a trade with the Vancouver Canucks, sending Chase Stillman and pick to acquire goaltender Arturs ...

Robbins Geller files suit against Rocket Pharma over undisclosed trial risks tied to RP-A501. Lead plaintiff deadline: August ...