New gene therapy improves the life of 18-year-old with Sickle Cell Disease which affects 5,000 New Jersey residents living ...

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

An annual review of clinical trials for Alzheimer's disease highlights a growing number of active trials—and drugs—in the ...

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 ...

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 ...

Q1 2025 Earnings Call Transcript May 27, 2025 BioLineRx Ltd. beats earnings expectations. Reported EPS is $1.39, expectations were $-0.84. Operator: Ladies and gentlemen, thank you for standing by.

Pfizer (NYSE: PFE) is a great example, with its shares down around 57% below the previous high. When a stock plunges as much ...

These states represent about 84% of Medicaid beneficiaries with sickle cell disease. Insurers ... may need additional education about the CGT pipeline to see value and have interest in CGT ...

followed by advancements in sickle cell disease and broader pipeline opportunities. The company’s strong financial position and strategic focus on rare diseases underscore its growth potential ...

Dublin, April 29, 2025 (GLOBE NEWSWIRE) -- The "Sickle Cell Disease- Pipeline Insight, 2025" has been added to ResearchAndMarkets.com's offering. The report offers an in-depth look into the sickle ...