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Results For "gene-therapy"

115 News Found

Boehringer and AnGes sign manufacturing agreement for HGF gene therapy product
Biotech | August 25, 2025

Boehringer and AnGes sign manufacturing agreement for HGF gene therapy product

Collaboration reinforces Boehringer Ingelheim’s expertise in microbial contract development and manufacturing


FDA lifts hold on Rocket Pharma's gene therapy trial
News | August 22, 2025

FDA lifts hold on Rocket Pharma's gene therapy trial

Rocket will also collaborate with investigators to implement an immunomodulatory regimen more closely reflecting that administered in the Phase 1 pediatric cohort


Roche updates on Elevidys gene therapy for Duchenne muscular dystrophy in EU
News | July 31, 2025

Roche updates on Elevidys gene therapy for Duchenne muscular dystrophy in EU

Elevidys is currently the only approved gene therapy that targets the root cause of Duchenne


Sarepta refuses to comply with FDA request to stop shipping gene therapy Elevidys
Drug Approval | July 21, 2025

Sarepta refuses to comply with FDA request to stop shipping gene therapy Elevidys

FDA requests Sarepta Therapeutics to suspend distribution of Elevidys and places clinical trials on hold for multiple gene therapy products following 3 deaths


Miltenyi Biotec and THSTI partner to launch CAR-T Cell and gene therapy training curriculum
News | July 21, 2025

Miltenyi Biotec and THSTI partner to launch CAR-T Cell and gene therapy training curriculum

This collaboration aims to bridge India’s talent and translational gap in this rapidly growing field by equipping professionals with practical expertise in the manufacturing of advanced therapies.


USFDA approves first gene therapy for treatment of aromatic L-amino acid decarboxylase deficiency
Drug Approval | November 16, 2024

USFDA approves first gene therapy for treatment of aromatic L-amino acid decarboxylase deficiency

Aromatic L-amino acid decarboxylase deficiency is a rare genetic disorder that affects the production of some neurotransmitters


Pfizer announces positive topline results from Phase 3 study of hemophilia A gene therapy candidate
Diagnostic Center | July 26, 2024

Pfizer announces positive topline results from Phase 3 study of hemophilia A gene therapy candidate

Giroctocogene fitelparvovec study meets primary and key secondary objectives of superiority compared to prophylaxis


European Commission approves Pfizer’s Durveqtix gene therapy for adults with hemophilia B
Drug Approval | July 26, 2024

European Commission approves Pfizer’s Durveqtix gene therapy for adults with hemophilia B

A one-time dose of DURVEQTIX has reduced bleeds post-treatment compared to standard of care with a median annualized bleed rate (ABR) of zero bleeds (range 0 to 9.9)


Pfizer updates on Phase 3 study of investigational gene therapy for ambulatory boys with Duchenne Muscular Dystrophy
Diagnostic Center | June 13, 2024

Pfizer updates on Phase 3 study of investigational gene therapy for ambulatory boys with Duchenne Muscular Dystrophy

The primary endpoint in the final analysis was assessed by change in the North Star Ambulatory Assessment at one year after treatment


Lily announces positive gene therapy result for genetic hearing loss
Diagnostic Center | January 25, 2024

Lily announces positive gene therapy result for genetic hearing loss

Hearing restoration was observed within 30 days of a single administration of AK-OTOF in the initial AK-OTOF-101 study participant