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.
Japan’s PMDA has approved Aurion Biotech Inc.’s cell therapy, Vyznova, for the treatment of bullous keratopathy of the cornea, making it the first-ever approval of a cell therapy to treat corneal endothelial disease.
Estrella Biopharma Inc. has received FDA clearance of its IND application for lead product candidate EB-103, a T-cell therapy targeting CD19, a protein expressed on the surface of almost all B-cell leukemias and lymphomas.
Thymmune Therapeutics Inc. has secured US$7 million in seed financing to support its work in developing scalable thymic cell therapies to restore immune function in aging and disease.
A trio of proposed Medicare drug payment models that made a Feb. 14 debut in the U.S. is playing to mixed reviews. Two of the models to be tested by the U.S. Centers for Medicare & Medicaid Services (CMS) Innovation Center seem to “address the real problems underlying prescription drug pricing – patient out-of-pocket expenses and better payment systems that reward the value a medicine brings to the patient and the overall health care system,” said John Murphy, chief policy officer for the Biotechnology Innovation Organization. But he called the third model, which is expected to restrict Medicare payment for some Part B drugs that have indications with accelerated approval, “an attack on the accelerated approval pathway,” which Congress mandated to spur investment and innovation in areas of unmet medical need.
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.
Unexpected behavior of neutrophils unveiled by researchers at Stanford University could lead to a new type of immunotherapy to treat cancer. Although various studies have suggested that these cells are harmful due to their immunosuppressive characteristics, the scientists saw in them an opportunity to redirect them and eliminate tumors.
The efficacy of allogeneic cell therapeutics for regenerative or oncology indications can be compromised by the emergence of antibodies against those cells, as observed in clinical trials. To overcome this limitation, scientists from the University of California and collaborators have developed a gene engineering approach providing antibody protection for cell therapeutics. The new approach to protecting cells from antibody-mediated cellular cytotoxicity (ADCC) relies on the overexpression of the IgG receptor CD64.
Researchers at the Barcelona Institute of Science and Technology’s Center for Genomic Regulation (CRG) and Pulmobiotics Ltd. have used one bacterium to fight another. In mouse models, the team used engineered Mycoplasma pneumoniae to treat Pseudomonas aeruginosa, the chief culprit in ventilator-associated pneumonia (VAP).
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