Fulcrum Therapeutics reports promising late-stage data for sickle cell drug pociredir, with notable hemoglobin improvements.

Key Points GAAP revenue surpassed expectations, reaching $12.5 million in Q2 2025, Revenue reached $12.5 million, up approximately 45.3% from the prior year period. Net loss (GAAP) widened to $112.0 ...

After decades of excruciating pain, a breakthrough sickle cell disease treatment made all the difference for one patient.

Oregon will initially focus on providing lower-cost therapies for people living with sickle cell disease, a genetic blood ...

Born in Mumbai, Reshma moved to the United States at the age of 11. She earned her medical degree from Boston University and ...

The primary safety risks associated with gene therapy include immune reactions and off-target effects in unintended organs, such as the liver, lungs, and spleen. Nevertheless, Ali and Townsend note ...

Daniel Cressy will be the first patient in Louisiana to complete the gene therapy treatment for sickle cell disease.

We recently compiled a list of 10 Most Undervalued Healthcare Stocks to Buy According to Analysts. Vertex Pharmaceuticals ...

Some Boston public school students are spending their summer vacations learning and working at a global biotech company.

Agios Pharmaceuticals gears up for a pivotal 2025 with key PYRUKYND milestones, including FDA decisions for thalassemia.

A cure for genetic heart conditions could reach trials in only 5 years, and it “rewrites” DNA. Experts explain the potential ...

Gene Therapy Market Gene therapy market surges on breakthroughs in genome editing, rising chronic & genetic disease cases, regulatory appro ...