First New York patient cured of sickle cell disease using Lyfgenia gene therapy

First In-Vivo Gene-Editing Therapy Successfully Saves Newborn's Life in 2025

In 2025, doctors and scientists in the U.S. successfully performed the world's first custom gene-editing therapy inside a living person—a ne

WHO publishes first global guidelines for managing sickle-cell disease in children and adolescents

This article presents the first WHO normative guideline on the diagnosis, prevention, and clinical management of sickle-cell disease (SCD) i

Columbia Researcher Discovers Rare Genetic Mutation That Provides Universal Antiviral Immunity

Columbia University immunologist Dusan Bogunovic discovered that a rare genetic mutation causing immune deficiency in some patients also gra

Seventh Person Cured of HIV Through Stem Cell Transplant, Second Without HIV-Resistant Cells

A man has become the seventh person to be cured of HIV after receiving a stem cell transplant for blood cancer treatment. Significantly, he

FDA Approves First Dual-AAV Gene Therapy for Genetic Hearing Loss Under Priority Voucher Program

The FDA has approved Otarmeni (lunsotogene parvec-cwha), the first-ever dual adeno-associated virus (AAV) vector-based gene therapy for trea

Teen Diagnosed with Rare Condition After Illness