Gene Therapy
29 articles RSS
NIH-Funded Team Engineers a Miniature CRISPR Nuclease That Fits Inside Viral Vectors, Clearing a Major Hurdle for In Vivo Gene Therapy
UT Austin and Metagenomi researchers engineered Al3Cas12f RKK, a compact CRISPR enzyme that achieves over 80% editing efficiency while fitting inside AAV delivery vehicles.
Prime Medicine Seeks FDA Accelerated Approval for First-Ever Prime Editing Therapy After Two Patients Achieve Durable Immune Restoration
Prime Medicine is pursuing accelerated approval for PM359, an autologous prime editing therapy that restored immune function in both patients treated for chronic granulomatous disease, marking the first clinical use of prime editing technology.
UCSF and Berkeley Scientists Engineer CAR-T Cells Directly Inside the Body, Clearing Tumors in Mice With a Single Injection
A two-particle gene-editing system published in Nature creates functional CAR-T cells in vivo, eliminating the costly weeks-long manufacturing process and clearing leukemia, myeloma, and solid tumors in preclinical models.
Life Biosciences Raises $80 Million to Fund First Human Trial of Epigenetic Cell Rejuvenation
The Boston biotech closes a Series D round to advance ER-100, the first FDA-cleared therapy that uses partial epigenetic reprogramming to reverse cellular aging in humans.
FDA Accepts Ultragenyx's Gene Therapy Application for Sanfilippo Syndrome, Setting September Decision Date for First-Ever Treatment
The FDA has accepted Ultragenyx's resubmitted BLA for UX111, a one-time gene therapy for the fatal childhood disease Sanfilippo syndrome Type A, with a decision expected by September 19, 2026.
Editas Medicine's CRISPR-Cas12a Therapy Achieves Functional Cure in 27 of 28 Sickle Cell Patients as RUBY Trial Results Land in NEJM
The RUBY trial of renizgamglogene autogedtemcel (reni-cel), a one-time CRISPR-Cas12a gene-editing therapy, showed that 27 of 28 patients with severe sickle cell disease had no painful crises after treatment, with hemoglobin levels normalizing to 13.8 g/dL by six months.
FDA Grants Accelerated Approval to First Gene Therapy for Severe Leukocyte Adhesion Deficiency, Capping a Two-Year Regulatory Journey
Rocket Pharmaceuticals' Kresladi becomes the first gene therapy approved for LAD-I, an ultra-rare immune disorder that leaves children vulnerable to fatal infections.
UC San Diego Engineers CRISPR Gene Drive That Spreads Through Bacteria to Strip Away Antibiotic Resistance
Researchers at the University of California San Diego have developed pPro-MobV, a second-generation CRISPR-based gene drive that spreads through bacterial populations via conjugal transfer to disable antibiotic resistance genes, demonstrating effectiveness even within biofilms.
Mass General Team's INSTALL Platform Solves Gene Therapy's Immune Toxicity Problem, Enabling Safe Non-Viral DNA Insertion in Living Mice
A Nature paper describes INSTALL, a gene editing platform using circular single-stranded DNA to insert therapeutic genes without triggering fatal immune responses, keeping mice healthy at doses where conventional DNA proved lethal.
FDA Lifts Clinical Hold on Intellia's CRISPR Gene-Editing Trial for Heart Disease After Patient Death and Safety Overhaul
The FDA has cleared Intellia Therapeutics to resume its MAGNITUDE Phase 3 trial of nexiguran ziclumeran for ATTR cardiomyopathy, five months after a patient death and subsequent clinical hold prompted a safety overhaul.
Sana Biotechnology Reports 14-Month Survival of Transplanted Islet Cells Without Immunosuppression in a Type 1 Diabetes Patient
Gene-edited pancreatic islet cells transplanted into a patient with type 1 diabetes continue producing insulin 14 months later with no immunosuppressive drugs, advancing the case for a functional cure.
In Vivo CAR-T Therapy Delivers First Human Proof-of-Concept for Autoimmune Reset, Bypassing Lab Manufacturing
MagicRNA's mRNA lipid nanoparticle therapy reprogrammed patients' own T cells inside the body, wiping out disease-driving B cells in refractory lupus patients without conventional cell manufacturing.