Gene therapy faces complexities in delivering treatments due to persistent safety concerns and daunting immune responses, but Next Generation Gene Therapeutics Inc. has found a way around this issue using dual-functional vectors to simultaneously remove harmful, mutated genes and replace them with normal, healthy genes to restore cellular function.
As an array of developers push their Friedreich’s ataxia (FA) drugs along, PTC Therapeutics Inc.’s recent update on its program piqued thirst for the 15-lipoxygenase inhibitor vatiquinone. Warren, N.J.-based PTC said the prespecified endpoint for two different FA long-term extension studies was met, with highly statistically significant evidence of durable treatment benefit on disease progression.
South Korean researchers developed a novel quantum dot and parallel-stacked organic light-emitting diode (QD-PSOLED)-based wearable patch capable of being “freely tunable” in real-time, and described as a flexible and commercial-level technology with wide applications for health care wearables.
Eli Lilly and Co.’s chief scientific officer, Daniel Skovronsky, called peresolimab, the PD-1 agonist previously in the works by the firm for rheumatoid arthritis (RA), a “really interesting mechanism” – but not interesting enough.
Positive findings from a phase III trial of semaglutide in metabolic dysfunction-associated steatohepatitis (MASH) is moving Novo Nordisk A/S to expand the glucagon-like peptide-1 (GLP-1) agonist’s indications in the U.S. and Europe, the Danish pharma said, as the MASH field sights more novel therapies.
Celltrion Inc. posted preclinical study results of two new antibody-drug conjugate (ADC) candidates – CT-P70 and CT-P71 – at the World ADC 2024 conference in San Diego Nov. 6, with plans to move the assets into clinical trials. Poster presentations of both ADC candidates “drew significant attention from the attendees” at the oncology meeting, Incheon, South Korea-based Celltrion said, while highlighting its efforts to transition from a biosimilar maker to a novel therapy developer.
Cell and gene therapy companies are the beneficiaries of positive changes along the regulatory path that the U.S. FDA is paving for them, according to a panel of executives who spoke at the BioFuture 2024 conference in New York.
In a pipeline shakeup, Tango Therapeutics Inc. has halted enrollment in a phase I/II study in order to push two other brain cancer drugs into development. TNG-908 had success in treating non-CNS solid tumors such as non-small-cell lung cancer and pancreatic cancer but missed the minimum pharmacokinetic exposure in clinical efficacy for treating glioblastoma.
Celltrion Inc. posted preclinical study results of two new antibody-drug conjugate (ADC) candidates – CT-P70 and CT-P71 – at the World ADC 2024 conference in San Diego Nov. 6, with plans to move the assets into clinical trials. Poster presentations of both ADC candidates “drew significant attention from the attendees” at the oncology meeting, Incheon, South Korea-based Celltrion said, while highlighting its efforts to transition from a biosimilar maker to a novel therapy developer.
Data from two Alzheimer’s tests being developed by Roche Holding AG showed high accuracy in detecting the disease in patients being tested for the condition. The tests measure biomarkers in the blood linked to Alzheimer’s and will help provide desperately needed information by letting patients know whether they have the disease or not, Margherita Carboni, Neurology Indication Lead at Roche Diagnostics told BioWorld.
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