One year after receiving CRISPR gene editing therapy at CHOP, a Delaware County baby is thriving, and doctors want to expand ...
After dumping its sole remaining gene therapy asset last year, Pfizer has decided to exercise its option for global rights to Beam Therapeutics’ liver-targeted gene editing candidate.
After teasing a new regulatory process for personalized genetic medicines at the end of last year, the FDA today unveiled draft guidance for an approval pathway that could see custom CRISPR therapies, ...
February 25, 2026 marks one year since KJ, an infant born with severe carbamoyl phosphate synthetase 1 (CPS1) deficiency, became the world's first person to receive a personalized CRISPR-based gene ...
Forget Vertex Pharmaceuticals -- CRISPR Therapeutics is a better buy right now.
The first gene therapies approved to treat sickle cell disease in December 2023 are struggling on the market. But there are ...
CRISPR gene therapy Casgevy can nearly eliminate sickle cell crises, but difficult stem‑cell collection is delaying access ...
UCLA researchers use lipid nanoparticles to insert full CFTR gene into airway cells, restoring function and opening new ...
Evox Therapeutics Ltd ("Evox" or the "Company"), a biotechnology company developing innovative therapies for genetically driven neurodegenerative diseases using a next-generation gene editing modality ...
The Food and Drug Administration aims to evaluate treatments for rare diseases based on plausible evidence that they would work — without requiring a clinical trial first.
CRISPR Therapeutics AG CRSP on Friday reported its fourth-quarter and full-year 2025 financial results, highlighting ...
Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...
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