A group of scientists from Basel University Hospital have designed an antibody-drug conjugate (ADC) that eliminated blood cancer cells without attacking healthy hematopoietic stem cells (HSCs), which they modified by base editing and transplanted to renew an altered blood system. They achieved this by focusing on the panhematopoietic marker CD45.
The U.S. FDA approved Orchard Therapeutics plc’s BLA for gene therapy atidarsagene autotemcel, making it the first treatment option for metachromatic leukodystrophy in the U.S. The one-time treatment, branded Lenmeldy, is indicated for children with presymptomatic late infantile, presymptomatic early juvenile or early symptomatic early juvenile disease.
A new method of CAR T-cell immunotherapy developed by researchers at the University of Pennsylvania Perelman School of Medicine could serve as a treatment for most blood cancers. Until now, CAR T-based immunotherapy for hematological malignancies has targeted the antigens CD19 for B cells, CD7 for T cells, BCMA for myeloma, and CD33 for AML.
Researchers have demonstrated that inhibiting mitophagy in ‘old’ hematopoietic stem cells (HSCs) completely restored their blood reconstitution capabilities, raising the prospect of addressing the age-related weakening of the immune system that stems from HSCs deteriorating over time.
Researchers have ameliorated both monogenic and complex inflammation-driven diseases through transplantation of hematopoietic stem cells with an inserted IL-1 receptor antagonist (IL-1RA) gene. The team showed that in animal models the transplanted cells worked better than monoclonal antibodies to reduce symptoms in systemic autoinflammatory diseases (SAIDs), a group of childhood-onset, lifelong diseases that vary in severity depending on the underlying mutation, but can be life-threatening.
A proof of concept of ex vivo genetic modification of cells from patients and their transplantation in mice has demonstrated, for the first time, the therapeutic possibilities of prime editing in sickle cell disease (SCD).
Base editing (BE), a technique that modifies a single nucleotide in living cells, has been successfully tested to resolve the CD3δ mutation in severe combined immunodeficiencies (SCIDs) and produce functional T cells. For now, scientists at the University of California, Los Angeles (UCLA), completed the study on patient stem cells and artificial thymic organoids, shortening the way for future clinical trials.
The science that led Garuda Therapeutics Inc. to a $62 million series B financing was a combination of hard work, luck and serendipity, according to co-founder and CEO Dhvanit Shah. At the Harvard Stem Cell Institute, Shah and his fellow researchers found that endothelial cells go through significant modifications before becoming hematopoietic stem cells. That simple discovery, as Shah told BioWorld, brought on research leading to the possibility that patients would not need a marrow donor before receiving a stem cell treatment.
Juvena Therapeutics Inc. co-founder and CEO Hanadie Yousef had the company’s name picked out several years before it was officially incorporated in 2017 to combine Yousef’s work in the mechanics of aging with an underutilized class of biologics and an advanced proteomics platform to tackle chronic and age-related diseases.
Two studies published back to back in Nature have looked at the accumulation of mutations in blood-forming stem cells with age, gaining new insights into how the overall landscape of such cells changes across the lifespan.