RenovoRx, Inc. (“RenovoRx” or the “Company”) (Nasdaq: RNXT), a life sciences company developing novel targeted oncology therapies and commercializing ...

Given Sobi's cash flow generation from its hemophilia business and mature product Synagis, the firm ended 2024 with a net debt/EBITDA ratio of 1.6 times. We expect the firm can support this level of ...

The US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) and rare paediatric disease designation (RPDD) to Arbor Biotechnologies' gene editing therapeutic, ABO-101, to treat ...

Ultragenyx Pharmaceutical RARE reported new findings from an ongoing, pivotal study of its investigational candidate, UX111 (ABO-102) AAV gene therapy, for Sanfilippo syndrome type A (MPS IIIA). Per ...

On January 7, 2025, the FDA granted priority review to the new drug application for sunvozertinib (DZD9008), an oral EGFR ...

CEO Ken d'Entremont highlighted the successful FDA approval of GRAFAPEX, the branded name for treosulfan for injection in the U.S., which holds Orphan Drug Designation, ensuring at least seven years ...

Ariceum Therapeutics is a private, clinical stage radiopharmaceutical company focused on the diagnosis and precision treatment of certain neuroendocrine and other aggressive, hard-to-treat cancers.

Quince Therapeutics, Inc. (Nasdaq: QNCX) is a late-stage biotechnology company dedicated to unlocking the power of a patient's own biology for the treatment of rare diseases. For more information on ...

Cumberland Pharmaceuticals' Phase 2 trial shows ifetroban improves heart function in Duchenne muscular dystrophy patients.

Despite some market exclusivity concerns, GERN still has many years of sales ahead, unencumbered by generic competition.

A Boston-area biopharmaceutical firm has submitted a biologics license application to the FDA for its investigational, fully ...