Business, energy, technology, markets and global industry news from Business News Today

gene therapy

PharmaDeviceNews gene therapy
Representative image: Vertex Pharmaceuticals’ expanded FDA approval for CASGEVY opens CRISPR gene therapy to children aged 2 and older with sickle cell disease and transfusion-dependent beta thalassemia.
Pharma & Biotech

Vertex expands CASGEVY to young children with sickle cell disease and beta thalassemia

Explore how the FDA’s CASGEVY age expansion could reshape early gene therapy for sickle cell disease and beta thalassemia. Read more.

Read More
Representative image: A clinician reviews muscle MRI scans as Epicrispr Biotechnologies’ EPI-321 delivers early evidence of increased lean muscle volume in patients with facioscapulohumeral muscular dystrophy.
Pharma & Biotech

Epicrispr’s one-time EPI-321 therapy shows early muscle growth after DUX4 silencing

Read how Epicrispr’s early EPI-321 muscle gains could reshape FSHD drug development, and what the small trial still cannot prove.

Read More
Representative image: A neurological researcher reviews advanced brain scans as uniQure prepares a potential FDA filing for AMT-130, its experimental gene therapy designed to slow Huntington’s disease progression.
Pharma & Biotech

FDA accepts uniQure’s AMT-130 filing strategy after months of regulatory uncertainty

Explore why the FDA reopened uniQure’s AMT-130 approval path and what evidence, surgical and confirmatory trial risks remain before review.

Read More
Representative image: A paediatric audiology assessment highlights the clinical focus behind Sensorion’s SENS-601 gene therapy program for GJB2-related hearing loss.
Pharma & Biotech

Sensorion’s SENS-601 strategy signals a sharper bet on larger genetic hearing loss markets

Find out how Sensorion’s SENS-601 regulatory push could reshape GJB2 hearing loss gene therapy and clinical strategy.

Read More
Representative image of a pediatric therapy consultation, illustrating how Biogen’s salanersen breakthrough therapy designation could shape the next phase of spinal muscular atrophy treatment and rare neurology care.
Pharma & Biotech

Can salanersen help Biogen defend its rare neurology franchise beyond Spinraza?

Find out how Biogen’s salanersen could reshape spinal muscular atrophy treatment after FDA breakthrough therapy status.

Read More
Representative image of a global healthcare policy discussion on hemophilia and inherited bleeding disorders, highlighting how the World Health Assembly resolution could influence diagnosis, treatment access and rare disease care equity.
Features & Analysis

A rare disease policy shift just landed at the WHO. Could it change hemophilia care worldwide?

Find out how the WHO hemophilia resolution could reshape diagnosis, treatment access, prophylaxis and bleeding disorder care worldwide.

Read More
Representative image: Scientists review DNA delivery and lipid nanoparticle research in a genetic medicines lab as Eli Lilly’s Engage Bio acquisition highlights the growing race to solve non-viral drug delivery challenges.
Pharma & Biotech

Eli Lilly expands genetic medicines toolkit with Engage Bio’s non-viral DNA delivery system

Eli Lilly’s Engage Bio deal is small by its standards, but DNA delivery may become a big battleground in genetic medicines.

Read More
Representative image: A clinician reviews bladder imaging during a urology consultation, reflecting Ferring Pharmaceuticals’ ADSTILADRIN re-induction data and the growing focus on bladder-sparing treatment strategies for BCG-unresponsive non-muscle invasive bladder cancer.
Pharma & Biotech

Can Ferring turn real-world ADSTILADRIN use into a broader NMIBC treatment strategy?

Bladder-sparing therapy needs better sequencing. Ferring’s ADSTILADRIN re-induction signal raises hope, but durability is the real test.

Read More
Representative image of non-viral gene therapy research, showing how lipid nanoparticles, CRISPR-based editing and advanced delivery platforms are gaining attention as viral vector safety concerns reshape genetic medicine.
Features & Analysis

Can non-viral gene therapies solve the safety problem holding back genetic medicine?

Viral vectors built gene therapy’s first era. Safety, redosing and manufacturing pressure are pushing non-viral platforms into the spotlight.

Read More
Representative image: Advanced AAV gene therapy manufacturing platforms such as AAVnerGene’s AAVone 2.1 are drawing attention as biotech developers look to improve vector productivity, full-capsid yield, and scalable production economics.
Pharma & Biotech

Why AAVnerGene’s AAVone 2.1 could matter for next-generation gene therapy manufacturing

AAV gene therapy needs better economics. AAVnerGene’s AAVone 2.1 tests whether single-plasmid production can ease the scale-up squeeze.

Read More