This article is part of a Rare Disease series sponsored by DeepIntent. With Rare Disease Month wrapping up and Rare Disease Day taking place on Saturday, MM+M is celebrating a handful of campaigns ...

Beam Therapeutics Inc. buy thesis: base-editing gene therapy leaders, risto-cel & BEAM-302 catalysts, strong cash runway.

With 300 million people living with rare diseases, experts say new gene and cell therapies are giving families in Wisconsin hope for lasting treatment options.

A high dose of sickle cell drug pociredir boosted fetal hemoglobin and reduced red blood cell destruction in adults with ...

CRSP shares jump on earnings report, strong pipeline for gene therapy. Revenue grows with increased patient treatment and analyst confidence.

Nemours Children’s Hospital is now a qualified treatment center to offer a transformative gene therapy to treat sickle cell ...

You Look Fine” is a groundbreaking new documentary, offering an intimate, first-person account of living with sickle cell disease.

“It can hit you out of nowhere,” she tells TODAY.com.

As Uganda rolls out mandatory nationwide screening of newborns for sickle cell disease this month, a gene therapy celebrated in the United States and Europe remains financially out of reach for most ...

Fulcrum Therapeutics, Inc. ( FULC) Q4 2025 Earnings Call February 24, 2026 8:00 AM EST Good morning, and welcome to the Fulcrum Therapeutics conference call to discuss 12-week data from the ...

Fulcrum Therapeutics reports promising Phase 1b data for pociredir in sickle cell disease and a strong cash position to fund development into 2029, boosting investor confidence.

CRISPR gene therapy Casgevy can nearly eliminate sickle cell crises, but difficult stem‑cell collection is delaying access ...