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Personalized RNA drug shows early promise in rare ALS case

A man with a slow-progressing, genetically rare form of ALS became the first person to receive an antisense oligonucleotide therapy designed specifically to target his causal mutation. A year later, he reported improved symptoms and remains able to work as a physician, according to results published in Med.

Experimental CAR T Immunotherapy Clears Cancer in 3-Year-Old Boy

Researchers report that a young boy's cancer went into remission after receiving an experimental form of CAR T cell therapy. The treatment is part of ongoing efforts to adapt CAR T technology, previously effective mainly against blood cancers, to target solid tumors.

CAR-T Cell Therapy Clears Child's Metastatic Liver Cancer in Trial

A young boy with hepatoblastoma that had spread to his lungs and stopped responding to chemotherapy achieved complete remission after receiving an experimental CAR-T cell treatment. The therapy, part of the CARE study, engineered the patient's own immune cells to target glypican-3, a protein found on liver cancer cells, while also boosting the cells' potency with two added immune signaling proteins. Twelve months after treatment, doctors report no detectable trace of the cancer.

Adelaide researchers use tumor-targeted nanoparticles to reprogram cancer-fighting immune cells

University of Adelaide scientists led by Chunxia Zhao designed 'smart' nanoparticles that ferry mRNA specifically to tumor-associated macrophages, prompting them to produce CXCL9, a molecule that summons T cells to attack cancer. The approach, described this week in Science Advances, aims to reverse how tumors normally hijack macrophages to block immune attacks.

Algonquin trial tests BPd combination in relapsed multiple myeloma across nine Canadian sites

Researchers report interim results from the ongoing Algonquin study, a multicentre, open-label trial evaluating a BPd drug combination in patients with relapsed or refractory multiple myeloma. The analysis covers patients enrolled in the trial's second phase, with data collected through late October 2025 after enrollment closed in September 2022. Eligible patients had failed prior treatments including lenalidomide and proteasome inhibitors, but had not previously received pomalidomide or BCMA-targeted therapy.

Legend Biotech Names Novartis Executive Ingrid Zhang as New CEO

Legend Biotech has appointed Ingrid Zhang, an executive from Novartis, as its new chief executive officer. She fills a leadership vacancy that opened in July after the company's previous CEO resigned abruptly.

US Cancer Patients Turn to Shanghai for Cheaper CAR-T Immunotherapy

American cancer patients are increasingly traveling to China for CAR-T cell therapy, a personalized immunotherapy that reengineers a patient's white blood cells to fight cancer. The Wall Street Journal reports that Chinese providers, having scaled up production, now offer the treatment for $150,000 to $230,000 versus $550,000 to $850,000 in the US, with faster turnaround and included follow-up care.

Utah researchers study red-light therapy itPBM for brain injury recovery

Scientists at the University of Utah, including former BYU football player Larry Carr, are researching intranasal transcranial photobiomodulation (itPBM), a red-light therapy aimed at reducing brain inflammation and improving cognition, mood and motor function. A recent study of 26 BYU football players published in the Journal of Neurotrauma found the therapy may help protect neurological health without requiring reduced playing time or contact exposure.

Small trial: engineered bone-marrow cell infusion sharply cuts osteoporosis fractures

Researchers gave ten older women with severe osteoporosis a single infusion of their own bone-marrow cells, which had been lab-modified to better home in on bone tissue. Before the treatment the women fractured bones roughly every one to two years; afterward, low-impact fractures dropped to about once per decade, according to a study published in Cell.

Life Biosciences begins human trial of Yuancheng Lu's age-reversal gene therapy for glaucoma

Geneticist Yuancheng Lu, working in David Sinclair's Harvard lab, developed a reprogramming gene therapy in 2018 that regenerated crushed optic nerves in mice and restored their vision. That same therapy, now called ER-100, has been adapted for humans by the startup Life Biosciences, which in June injected it into the eye of a glaucoma patient as part of a clinical trial.

Deaths of two children in Chinese gene-therapy trials raise oversight concerns

Two children died in separate gene-editing clinical trials in China, prompting scrutiny from researchers, ethicists and legal scholars over how such studies are approved and monitored. Investigators say the trials involved ethical breaches that could carry lasting consequences for China's biomedical research sector.

Deaths of two children in Chinese gene-therapy trials spark scrutiny of oversight

Two children died in separate Chinese gene-editing trials — a boy in a 2025 trial by HuidaGene Therapeutics and a six-year-old girl in March 2024 after treatment developed by researchers at Shanghai Jiao Tong University and Xinhua Hospital. Shanghai Jiao Tong University has launched an investigation into the girl's case and a related study published in Nature, while HuidaGene disclosed the boy's death publicly in August.