First Patient Dosed in the Phase 1/2 Study of BEAM-301 in Glycogen Storage Disease Type Ia, Beam’s Second Clinical Stage In ...

The "Sickle Cell Disease Drugs Market - A Global and Regional Analysis: Focus on Route of Administration, Distribution Channel, and Country-Level Analysis - Analysis and Forecast, 2025-2035" report ...

Precision gene-editing company Beam Therapeutics (NASDAQ: BEAM) had a Tuesday to forget on the stock market. Following the ...

Enrollment complete in the 12 mg dose cohort (n=16) of the Phase 1b PIONEER trial of pociredir in sickle cell disease (SCD); initiated the 20 mg dose cohort ― ― On track to provide clinical data from ...

Beam Therapeutics and Verve Therapeutics have each built their lead candidates on a technique billed as a safer alternative ...

You’ve likely heard people mention that they have inherited certain health conditions from their family. These are known as ...

For Vertex, the first quarter of 2025 brought good news and bad news. | Despite positive early signs for its newly launched ...

With AI, Google DeepMind’s AlphaFold has been able to unfold the difficult mystery around the protein-folding problem that ...

Apr. 11, 2025 — A multi-institutional team developed a chimeric antigen receptor (CAR) T cell-based strategy ... that are driven by mutations that involve gene fusions. The technology accurately ...

LCA10 is caused by mutations in the CEP290 gene and ... has been given the go-ahead by the agency. Vertex's gene-editing therapy for sickle cell disease, Casgevy, has been given the nod for ...

In conclusion, this new AI breakthrough offers great promise for the future of healthcare. It will make diagnosing genetic ...

Despite reports of a sickle cell patient being cured by gene therapy, columnist Oluwatosin Adesoye explains why she remains ...