Sanofi announced today that the U.S. FDA has granted orphan drug designation to rilzabrutinib for the treatment of sickle ...

Rilzabrutinib granted orphan drug designation in the US for sickle cell disease <li /> Fourth orphan drug designation for rilzabrutinib ...

Sickle cell disease is a group of rare ... medicines and vaccines that treat and protect millions of people around the world, with an innovative pipeline that could benefit millions more. Our team is ...

Sanofi SNY announced that the FDA has granted orphan drug designation to its investigational BTK inhibitor, rilzabrutinib, for sickle cell disease ... to Sanofi’s pipeline with the acquisition ...

Sanofi SNY announced that the FDA has granted orphan drug designation to its investigational BTK inhibitor, rilzabrutinib, ...

Discover BioLineRx's Q1 2025 highlights, including APHEXDA growth, cost reductions, pipeline expansion, and updates on oncology trials.

Beam Therapeutics faces slow pipeline progress and high R&D costs despite a $500M cash boost. Learn why patience is key for ...

For investors still seeking companiies with attractive long-term prospects, let's consider two in the biotech industry: ...

Leaders from the world of cell and gene therapy shared their personal stories and policy recommendations in an extraordinary roundtable conducted by the FDA in front of its recently-appointed top ...

Intellia has leveraged its expertise in Crispr/Cas9 gene editing to advance a pipeline of in vivo and ex vivo therapies for diseases with high unmet medical needs. We believe Intellia's proprietary ...

Two great examples are Berkshire Hathaway (NYSE: BRK.A) (NYSE: BRK.B) and Vertex Pharmaceuticals (NASDAQ: VRTX). Warren ...