Teen Beats Deadly Leukemia With Life-Saving Gene-Edited Cell Therapy

A cutting-edge base-edited CAR T-cell therapy is opening a revolutionary new frontier for patients battling aggressive T-cell leukemia.

Vertex's gene therapy shows promise in younger children with blood disorders

Vertex Pharmaceuticals said on Saturday its gene therapy helped children aged between 5 and 11 years with sickle cell disease ...

California boy, 3, receives first-ever gene therapy for his rare disease: ‘It’s just so exciting’

A California toddler is the first person in the world to receive gene therapy to treat his devastating disease. Three-year-old Oliver Chu was born with a rare, genetic condition called Hunter syndrome ...

Abeona Therapeutics® Announces First Patient Treatment with ZEVASKYN® Gene Therapy

Treating our first patient is a proud moment for Abeona and a testament to the tireless resolve of our team,” said Vish ...
Technology Networks

From Clinical Implications to Modernizing Regulatory Frameworks: Noteworthy Advances To Watch in Cell and Gene Therapy R&D

Over the last decade, the number of cell and gene therapy clinical trials has more than tripled. In this article, several ...
ABC Columbia

Jack is Back! Chapin student returns to school following experimental gene therapy treatment

Two years ago, he was diagnosed with a terminal illness called Batten disease -- often leading to blindness, seizures, and ...
Pharmaceutical Technology on MSN

Potentially “groundbreaking” FDA gene therapy pathway leaves key issues unresolved

A new FDA pathway could speed bespoke gene therapies, but key questions over scope and commercial viability remain.
NJBIZ

FDA approves Novartis’ Itvisma gene therapy for SMA

Novartis’ Itvisma wins FDA approval as the first gene replacement therapy for spinal muscular atrophy (SMA) patients age 2 ...
BioPharma Dive

Novartis wins approval to use SMA gene therapy in older patients

Itvisma, an intrathecal version of Zolgensma, can be used in children, teens and adults to stabilize or improve motor ...

Genetix Participates in the American Society of Hematology's (ASH) Exclusive Press Program & Shares Recent Patient Experience Data from U.S. Commercial Gene Therapy Implementation

Please refer to the ASH 2025 online program for full session details and visit the Genetix booth (#1261) onsite.
pharmaphorum

Novartis' SMA gene therapy cleared for wider use in US

The new intrathecal formulation means the one-shot gene therapy is now available to all SMA patients, regardless of their age ...
The Times of Israel on MSN

In world first, Israeli scientists use RNA-based gene therapy to stop ALS deterioration

By adding a microRNA molecule to ALS models, Tel Aviv University, Ben-Gurion University and Weizmann Institute researchers say the damaged nerve cells 'even regenerated' ...