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).
Pulmobiotics Ltd., which was founded in 2019, is developing cell therapy for lung diseases, including lung cancer. But unlike other cell therapies for cancer, this one is based not on harnessing T cells but on engineering bacteria. The team has engineered Mycoplasma pneumoniae to deliver various therapeutic proteins to the lung, depending on the therapeutic indication.
After long years of painstaking work, the commercialization of cell and gene therapies picked up pace in 2022, with multiple approvals. More progress is expected in 2023, with several firsts in the offing and products for larger patient populations reaching the market.
A combination of bioengineering techniques on normal cell binding proteins could be the method of the future for selective cell binding. Scientists at the University of California, San Francisco (UCSF) have created a synthetic glue based on the expression of membrane receptors to establish the desired connection between cells. The results may be applied in different fields of cell biology or biomedicine, such as regeneration and wound repair, including the nervous system, or cancer.
Arcellx Inc. signed a deal that could be worth almost $4 billion with Gilead Sciences Inc.’s unit Kite Pharma Inc. to push forward Arcellx's lead late-stage candidate CART-ddBCMA for relapsed or refractory multiple myeloma. The arrangement brings $225 million up front plus an equity investment of $100 million, along with as much as $3.9 billion in milestone payments. Arcellx CEO Rami Elghandour said the firm sorted through a number of suitors interested in the program. Data at the American Society of Clinical Oncology meeting “catalyzed a number of discussions and a broad set of interests. We felt of the possibilities out there, [Kite/Gilead is] the partner of choice in this space.”