Durham-based Precision BioSciences, a gene-editing company, has received regulatory clearance to begin a clinical study of its potential treatment for Duchenne muscular dystrophy (DMD), a rare ...
The MarketWatch News Department was not involved in the creation of this content. AURORA, Colo., Oct. 1, 2025 /PRNewswire/ -- RheumaGen, Inc., a cell and gene therapy company engineering a new class ...
Beam Therapeutics (NASDAQ:BEAM) outlined a new liver-targeted development program for phenylketonuria (PKU) and announced a large, non-dilutive financing tied to its sickle cell disease launch plans ...
A new gene-editing technique enables the correction of multiple genetic mutations simultaneously, transforming the prospects for millions living with complex inherited diseases, such as cystic ...
ProQR Therapeutics advances RNA editing pipeline with upcoming CTA filing for AX-0810, while enhancing leadership and maintaining strong finances. ProQR Therapeutics N.V. announced significant ...
ProQR scientists report for the first time in the ADAR RNA editing field in vivo proof of target engagement (RNA editing) leading to meaningful changes in biomarkers in NHPs using Axiomer™ RNA Editing ...
A $14M grant will fund research on gene-editing therapies for rare metabolic diseases at the Perelman School of Medicine at the University of Pennsylvania (Penn) and Children's Hospital of ...
The NIH awarded a $26 million grant to the Perelman School of Medicine and the Children’s Hospital of Philadelphia to support gene-editing research. Credit: Liliann Zou The National Institutes of ...
As other gene editing programs fold or get sold, Arbor Biotechnologies has secured $73.9 million to advance its lead liver-targeting gene therapy into human trials. The series C was led by Arch ...
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