Gene Therapy - Search News

uniQure advances gene therapy trial for Fabry disease

LEXINGTON, Mass. - uniQure N.V. (NASDAQ: NASDAQ:QURE), a gene therapy company with a market capitalization of $767 million, announced today the completion of enrollment for the first cohort in a Phase I/IIa trial of AMT-191,

Hosted on MSN · 6d

Gene Therapy Stabilizes Rare Chronic HPV Disease

For recurrent respiratory papillomatosis (RRP), novel gene therapy to jump-start immunity to the human papillomavirus (HPV) that causes it was safe and yielded complete response in many cases, a small pivotal trial showed.

National Institute for Health and … · 3d

Groundbreaking one-off gene therapy approved for severe sickle cell disease

People in England to benefit from groundbreaking one-off gene therapy for severe sickle cell disease approved by NICE

Reuters · 3d

UK health system to offer cutting-edge gene therapy for sickle cell disease

Britain's National Health Service (NHS) will provide a cutting-edge gene therapy that aims to cure sickle cell disease, the National Institute for Health and Care Excellence (NICE) said on Friday.

labiotech · 7d

Why are there only 20 cell and gene therapies in Europe?

There are 20 cell and gene therapies on Europe's market versus 43 in the U.S. Find out more about this ecosystem.

BBC · 3d

NHS to offer 'groundbreaking' sickle cell gene therapy

The gene therapy Casgevy, which will now be offered to some patients in England, could help people live disease-free, experts say.

Health and Me on MSN · 1d

Gene-editing Therapy Could Be The Cure To Sickle Cell Disease

In 2019, the first CRISPR clinical trials were conducted to treat sickle cell diseases and in 2020, this treatment was injected directed into a human patient to treat hereditary blindness. The National Institute for Health and Care Excellence said it could suit around 50 patients per year.

GB News on MSN · 2d

NHS to offer 'groundbreaking' sickle cell therapy as patients given 'very real prospect of cure'

A groundbreaking £1.65million gene therapy treatment offering hope for sickle cell disease patients has been approved for use on the NHS in England. The one-time treatment, called Casgevy (exa-cel), has shown remarkable success in clinical trials,

ITN on MSN · 3d

Revolutionary gene-editing therapy for sickle cell approved

NHS Karla Sofia Gascón apologizes for old tweets, says she is 'deeply sorry to those I have caused pain' Everything we know about China's new 6th gen fighter jet... AstraZeneca cancels £450m investmen

Hosted on MSN · 3d

‘Much-needed hope’ as CRISPR gene editing therapy recommended for sickle cell disease

Experts hailed exciting news for some people with sickle cell disease in England as the health watchdog decided to grant access to gene-editing therapy on the NHS. View on euronews

The Scientist5h

A Nanoparticle Delivery System for Gene Therapy

Genetic medicines could treat certain diseases, however, the current delivery systems available—viral vectors and lipid nanoparticles (LNPs)—pose unique challenges for gene therapy applications. For ...

Managed Healthcare Executive2d

Gene Therapy’s Pipeline Has a Steady Flow of More Than 2,000 Candidates

The majority are in the very early preclinical stage of development, but applications for regulatory approval have been ...

Spur Therapeutics Announces Successful End-of-Phase 2 Meeting with FDA for FLT201, Its Gene Therapy Candidate for Gaucher Disease

FDA aligned on single-arm Phase 3 study to support potential accelerated and full approval On track to dose first patient in Phase 3 trial in second half of 2025 LONDON, Feb. 03, 2025 (GLOBE NEWSWIRE) ...