Algorithm predicts success of next-generation CRISPR edits, advancing push to treat genetic diseases
February 16, 2023 -- A machine learning algorithm has predicted the chances of successfully inserting a gene-edited sequence of DNA into a cell using a next-generation CRISPR-Cas9 approach. Read More
Adaptive Biotechnologies Q4 revenues rise 46% on NGS test volumes
February 15, 2023 -- Adaptive Biotechnologies on Tuesday reported fourth-quarter revenue of $55.2 million, up 46% compared with $37.9 million in Q4 2021. Read More
Challenges and remedies for manufacturing cell therapies at scale
February 3, 2023 -- Cell therapies have the potential to cure human diseases rather than just treat the symptoms, and they are potentially life-changing if they can be administered early in a patient’s lifetime. Read More
Aston University partners with longevity biotech to advance lung disease treatment
January 25, 2023 -- Aston University's work on small molecule inhibitors of transglutaminase 2 has spawned a biotech firm focused on treating the chronic lung disease idiopathic pulmonary fibrosis. Read More
Newborn screening for neuromuscular disorder leads to improved outcomes in Australian study
January 18, 2023 -- Newborn screening for spinal muscular atrophy (SMA) improves outcomes by enabling timely access to disease-modifying therapy, a prospective study published in the Lancet Child & Adolescent Health found. Read More
T-cell therapy fights viral infections following stem cell transplants
January 13, 2023 -- Baylor College of Medicine and Washington University School of Medicine researchers studied posoleucel, an investigational off-the-shelf T-cell therapy that simultaneously targets six different viruses. The results, published January 11 in the journal Clinical Cancer Research, showed promising antiviral efficacy and safety in a phase II clinical trial of patients who had undergone stem cell transplantation to treat blood diseases including cancer. Read More
Optimizing AAV vectors for gene therapy delivery
January 10, 2023 -- Adeno-associated viruses (AAV) can be engineered to deliver DNA to target cells. It is used extensively for the development of viral vector-based gene therapies in the pipeline. However, researchers encounter challenges with producing sufficient quantities of AAVs for gene therapy products, and they struggle to get delivery vehicles and their genetic payloads into the brain across the blood-brain barrier. Read More
Discovery of destructive CRISPR enzyme opens diagnostic, therapeutic applications for molecular scissors
January 4, 2023 -- The discovery of a CRISPR enzyme that destroys DNA and RNA in target cells has unlocked opportunities to use molecular scissors in diagnostics and to selectively destroy diseased cells. Read More
Cancer-killing vaccine may also prevent brain cancer
January 4, 2023 -- Harvard Stem Cell Institute scientists are harnessing a new method of turning cancer cells into potent, anti-cancer agents. Their new cell therapy approach to eliminating established tumors also trains the immune system to prevent cancer from recurring, providing long-term immunity. The NIH-funded study, published January 4 in Science Translational Medicine, showed promising results when the approach was tested on mice with glioblastoma, a deadly brain cancer. Read More
Common genetic cause of late-onset ataxia revealed
December 16, 2022 -- A Quebec-led international collaboration has discovered a previously unknown common genetic cause of late-onset cerebellar ataxia. The study, published December 14 in the New England Journal of Medicine, may potentially improve diagnosis and open new treatment avenues for thousands of people with this debilitating neurodegenerative condition worldwide. Read More
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