gene therapy | pharmaphorum

Wednesday 22 July 2026

gene therapy

News

ICER backs sickle cell gene therapies at one-off cost of $2m

ICER backs sickle cell gene therapies at one-off cost of $2m

The influential Institute for Clinical and Economic Review (ICER) in the US has issued draft guidance suggesting that new gene therapies for sickle cell disease (SCD) from

News

eye health

Nanoscope gene therapy clears ph2 retinitis pigmentosa trial

Nanoscope Therapeutics has reported data from a mid-stage clinical trial that suggests its optogenetic therapy based on light-sensitive molecules is an effective treatment

News

PTC’s gene therapy for rare disease cleared for NHS use

PTC’s gene therapy for rare disease cleared for NHS use

Upstaza has shown impressive clinical results in children with fatal disorder AADC deficiency.

/themes/custom/pharmaphorum_theme/library/img/article-label-image/ondemand.png

R&D

Trials and tribulations of drug development webinar

Sponsored

Trials and tribulations of drug development

What does it take to establish new treatments for rare diseases?

News

CSL’s Hemgenix is first haemophilia B gene therapy in EU

CSL’s Hemgenix is first haemophilia B gene therapy in EU

Firm will work with payers to try to secure access for as many patients across EU as possible.

R&D

4th Gene Therapy Analytical Development Europe Summit 2023

Partner Content

4th Gene Therapy Analytical Development Europe Summit 2023

2-4 May, 2023 London, UK Actionable Insights to Optimise Analytical Development for Gene Therapy Products