NOLA.com

First Louisiana patient to receive sickle cell gene therapy heads home after 6 weeks in hospital

Learn about the groundbreaking gene therapy for sickle cell disease at Manning Family Children's Hospital in Louisiana, ...
MPR

Gene Editing Candidate Lonvo-z Meets Endpoints in Phase 3 HAE Study

Lonvo-z is an in vivo CRISPR gene editing candidate that is designed to inactivate the kallikrein B1 gene, thereby permanently lowering kallikrein and bradykinin levels.
News-Medical.Net

Engineered cells boost production of gene editing delivery vehicles

Gene editing has emerged as a powerful approach for targeting the genetic causes of disease, but getting the editing ...
BioSpace

Intellia heads to FDA after notching first late-stage win for in vivo gene editor

With Phase 3 data in hand, Intellia Therapeutics is seeking approval for its in vivo CRISPR gene editing therapy for ...

New self-assembling polymers proven to be effective at gene delivery

A collaboration of scientists at the University of Manchester and the University of Birmingham has explored a more effective ...
Fierce Biotech

Astellas thins early pipeline, swaps out struggling rare disease gene therapy

Astellas Pharma has done some spring cleaning of its pipeline, dropping two phase 1 candidates and slamming the brakes on a ...

Apertura Gene Therapy and Scientists from the Broad Institute to Share Data on TfR1 CapX™, a Novel Capsid Targeting the Central Nervous System (CNS), at the American Society ...

Apertura Gene Therapy, a biotechnology company developing next-generation AAV capsids for delivering genetic medicines, along with scientists from the Broad Institute of MIT and Harvard, will present ...
The Manila Times

Taysha Gene Therapies to Present New Preclinical Data Supporting Construct Design of TSHA-102 for Rett Syndrome at the ASGCT 2026 Annual Meeting

In vitro data demonstratedself-complementary AAV9 (scAAV9) enabled ~30-fold higher MeCP2 protein expression compared to ...