CRISPR/Cas9 improves ASOs efficacy
A study by the University of Basel and Roche has unveiled a method to enhance the efficacy of RNA-based therapies, such as antisense oligonucleotides (ASOs), which are used to treat genetic disorders like ALS and Duchenne muscular dystrophy. The research identified that the intracellular transport speed of ASOs significantly affects their therapeutic impact. By employing CRISPR/Cas9 screening, the team pinpointed genes – notably AP1M1 – that influence ASO activity. Inhibiting AP1M1 slowed endosomal transport, allowing ASOs more time to escape into the cytoplasm, thereby increasing their effectiveness without additional dosing. These findings suggest that modulating intracellular trafficking can improve RNA therapy outcomes and may offer new strategies against infections by affecting pathogen processing within cells.
AbbVie’s CAR-T acquisition
AbbVie has announced its acquisition of Capstan Therapeutics in a deal valued at up to $2.1 billion. Capstan specializes in developing in vivo CAR-T cell therapies, particularly for autoimmune diseases. Their lead candidate, CPTX2309, is currently in preclinical development. The acquisition forms part of AbbVie's strategy to diversify its pipeline, especially following the loss of patent protection for its arthritis drug, Humira. The deal includes an upfront payment of $250 million, with additional milestone-based payments. AbbVie aims to leverage Capstan's technology to enhance its capabilities in immunology and cell therapy. The transaction is expected to close in the second half of 2025, subject to regulatory approvals.
CRISPR Therapeutics in TIME magazine’s 100 Most Influential Companies
CRISPR Therapeutics has been named to TIME’s 2025 list of the 100 Most Influential Companies, recognizing its pioneering work in gene editing. The company gained prominence with the FDA approval of Casgevy, a CRISPR-based therapy for sickle cell disease and beta thalassemia. Under CEO Samarth Kulkarni, CRISPR Therapeutics is expanding its focus to more prevalent conditions like high cholesterol and autoimmune diseases, aiming to make gene editing treatments more accessible and affordable. TIME highlighted this strategic shift as a significant step toward broader impact in healthcare.
Gear-shift for aging research
Shift Bioscience has introduced a refined ranking system for virtual cell models to enhance gene target discovery in aging research. The study identifies limitations in traditional benchmarking methods, which often favor average predictions over biologically meaningful outcomes due to control biases and weak perturbations. To address this, the team developed differentially expressed gene (DEG)-weighted metrics, including weighted mean squared error (WMSE) and weighted delta R², along with calibrated baselines and DEG-aware optimization objectives. These improvements aim to better assess model performance, highlighting models that effectively predict gene-specific perturbations. By implementing these metrics, Shift Bioscience seeks to accelerate its therapeutic pipeline, focusing on uncovering new targets for rejuvenation treatments.
Research
CGT normalizes SCD hemodynamics
A team of researchers from Novartis Institutes for Biomedical Research, St Judes Children’s Research Hospital, and the University of Chicago, have co-authored a study titled "Normalization of Cerebral Hemodynamics After Gene Therapy in Adults With Sickle Cell Disease", assessing how CRISPR-Cas9-based gene therapy impacts cerebral blood flow (CBF) in adults with sickle cell disease (SCD). The researchers evaluated three adults who received autologous hematopoietic stem cells edited at the HBG1/HBG2 promoters to induce fetal hemoglobin (HbF) production. MRI-based CBF measurements were taken at baseline and at 12 and 24 months post-infusion. All patients showed significant reductions in whole-brain CBF after gene therapy, with no signs of new neurological complications or infarcts. These changes suggest normalization of cerebral hemodynamics, likely driven by sustained increases in hemoglobin and HbF. Unlike hydroxyurea or transfusion therapy, the improvements observed after gene therapy were more durable. While limited by a small cohort, the findings support gene therapy’s potential to reduce stroke risk and preserve neurocognitive function in SCD.
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