ProQR Therapeutics Unveils New RNA Editing Programs; AX-0810 Phase 1 Target Data Due This Quarter

ProQR Therapeutics (NASDAQ:PRQR) outlined multiple pipeline updates and development priorities during its virtual investor ...

AIRNA Announces First Patient Dosed in Phase 1 Trial of AIR-001, Potential Best-in-Class RNA-Editing Therapy for Alpha-1 Antitrypsin Deficiency (AATD)

AIRNA, a biotech company pioneering RNA-editing therapeutics to transform the lives of patients with rare and common ...

ProQR Highlights Pipeline Expansion and Multiple Upcoming Clinical Catalysts at Investor and Analyst Event

AX-0810 clinical target engagement data in healthy volunteers on track for this quarter; biliary atresia selected as initial Phase 2 ...

ProQR Announces Partnership with Ginkgo Bioworks and Formation of AI Advisory Board

ProQR and Ginkgo Bioworks to collaborate on autonomous lab high throughput data generation to support ProQR’s AI-enabled drug discovery for ...

Growing optimism about ProQR Therapeutics (PRQR)’s RNA editing platform and future clinical catalysts

ProQR Therapeutics N.V. (NASDAQ:PRQR) earns a spot in our list of the top 10 stocks under $5 that could triple. As of March 19, 2026, analysts remain confident in ProQR Therapeutics N.V. (NASDAQ:PRQR) ...

Clinical trial shows gene editing works for β-Thalassaemia, too

Now, following up on that success, a large Chinese collaboration has followed up with a description of an improved gene ...
News Medical

RNA editing enzyme found to trigger neuroinflammation in Parkinson's

Parkinson's disease (PD) is a neurodegenerative disorder in which the α-synuclein protein abnormally aggregates within brain cells, causing neuronal damage. Through international collaboration, ...
Phys.org

RNA editing tool can take some of the risk out of gene therapy

The ability to correct disease-causing genetic mistakes using genome editors holds great promise in medicine, but it is not without risk. When this type of "genetic surgery" is performed on DNA, for ...

Down syndrome study suggests early RNA editing shifts may reshape fetal brain circuits

A collaborative research study co-led by scientists at the Icahn School of Medicine at Mount Sinai and the Liber Institute ...