Gene therapy can partly restore sight in blind people, researchers reveal

Optogenetic therapy, based on Nobel prize-winning science, involves turning nerve cells on and off using light ...

Is Ultragenyx Pharmaceutical (RARE) Undervalued Following EMA Validation For Its Sanfilippo Gene Therapy?

Ultragenyx Pharmaceutical (RARE) is back in focus after the European Medicines Agency validated its Marketing Authorisation ...
Ophthalmology Times

EURETINA 2026: 3 gene therapy programmes for nAMD approaching phase 3 readouts

At EURETINA 2026, Arshad Khanani, MD, FASRS, reviews sura-vec, 4D-150 and ixo-vec, 3 nAMD gene therapies in phase 3 with ...
Newswise

Andelyn Biosciences and PQP Group Partner to Streamline EU and UK Supply Pathways for Cell and Gene Therapy Sponsors

Andelyn Biosciences ("Andelyn"), a leading US-based patient-focused cell and gene therapy Contract Development and ...
Pharmaceutical Technology on MSN

Scientists claim Nobel Prize for approach behind light-activated gene therapy

The Nobel Prize committee says that optogenetics will lay the foundations for a new era in neuroscience, while pharma ...
Stocktwits on MSN

Stocktwits Pharma Pulse: Roche faces FDA decision, gene-therapy stocks step into focus — the week’s biotech watchlist

Lilly won FDA approval last week to expand Jaypirca’s use to patients starting treatment for certain blood cancers. ・Ocular ...

What Krystal Biotech (KRYS)'s VYJUVEK Gene Therapy Momentum Means For Shareholders

Krystal Biotech’s VYJUVEK gene therapy for dystrophic epidermolysis bullosa has already delivered strong commercial traction, ...

FDA Approves First Gene Therapy for Pediatric Patients with Sanfilippo Syndrome Type A

The U.S. Food and Drug Administration today approved Fayuvi (rebisufligene etisparvovec-hopf), the first treatment for pediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA), also known as ...

What’s The Latest On CRISPR Gene Editing Technology?

CRISPR is one of this generation’s most profound innovations, with the potential to fundamentally alter the realm of modern ...
Nature

Gene therapy for rare lysosomal storage disorder nabs FDA approval

The US FDA has approved Ultragenyx Pharmaceutical’s rebisufligene etisparvovec (Fayuvi) for paediatric patients with ...
STAT

Drug meant to make gene therapy safer may also make it less effective

Jason is a general assignment reporter, with particular focus on genetic medicine and rare disease. Confidential tips can be sent on Signal at JasonMast.77. BOSTON — A medicine increasingly used to ...
Forbes

A Turning Point For Gene Therapy: What One Tragic Event Reveals About The Promise And Peril Of Genetic Medicine

Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...