What Celosia Therapeutics’ Phase 1b CTx1000 trial could reveal about TDP-43 targeting in ALS
Celosia Therapeutics has dosed the first ALS patient with CTx1000. Read why this TDP-43 gene therapy trial could reshape the ALS pipeline.
Pharma & Biotech
Pharma & Biotech
How Sensorion’s SENS-501 data could reshape gene therapy for congenital hearing loss treatment
Sensorion reports promising gene therapy data for congenital deafness. Discover what it means for hearing loss treatment and future clinical breakthroughs.
Pharma & Biotech
Gene therapy gains ground in bladder cancer as NCCN strengthens Ferring Pharmaceuticals’ ADSTILADRIN recommendation
ADSTILADRIN gains NCCN upgrade in bladder cancer. Discover what this means for gene therapy adoption, clinical use, and future NMIBC treatment trends.
Pharma & Biotech
Cathy Tie’s Origin Genomics targets germline gene correction under US IRB oversight
Origin Genomics launches in New York to advance germline gene correction and MRT under IRB oversight. Analyse what it means for US gene editing regulation.
Pharma & Biotech
GenSight Biologics begins LHON treatment under France compassionate access as REVISE Phase II moves forward
GenSight Biologics begins treating ND4-LHON patients under France's AAC compassionate access program. Analyse what REVISE and AAC mean for the regulatory pathway.
Pharma & Biotech
Affinia Therapeutics’ AFTX-201 Fast Track decision highlights rising interest in cardiac gene therapy
Affinia Therapeutics’ AFTX-201 wins FDA Fast Track for BAG3 cardiomyopathy. Explore what this means for cardiac gene therapy and future heart failure treatments.
Pharma & Biotech
Genethon advances Duchenne gene therapy trial while licensing Pompe tech to Bayer’s AskBio
Genethon advances gene therapies for Duchenne and Pompe disease. Find out what its nonprofit model and AskBio licensing deal mean for rare disease care.
Pharma & Biotech
Can ELEVIDYS meet its promise? Sarepta’s pivotal Duchenne gene therapy trial nears major milestone
Sarepta’s EMBARK 3-year readout could clarify ELEVIDYS’ long-term role in Duchenne care. Find out what’s at stake in this pivotal gene therapy trial.
Pharma & Biotech
Can AB-1009 reshape treatment for late-onset Pompe? AskBio prepares for first patient in U.S.
AskBio’s AB-1009 gene therapy for late-onset Pompe secures FDA IND clearance. Find out what this milestone means for AAV innovation and patient outlook.
Pharma & Biotech
Can SonoThera’s RIPPLE platform redefine nonviral delivery in oversized genetic payloads?
Can SonoThera’s ultrasound platform reshape gene therapy delivery? Find out how its $125M Series B targets DMD, ADPKD, and XLAS with nonviral precision.