Intellia Therapeutics Inc. followed up troubling news in May with a similar, and worse, update regarding the Magnitude and Magnitude-2 phase III trials with nexiguran ziclumeran, also known as nex-z, for patients with transthyretin amyloidosis with cardiomyopathy and polyneuropathy, respectively.
Durable reprogramming of human T cells may now be possible thanks to a new technique based on the CRISPRoff and CRISPRon methodology. Researchers from the Arc Institute, Gladstone Institutes, and the University of California San Francisco have stably silenced or activated genes in this type of immune cell without cutting or altering its DNA, making T cells more resistant, active, and effective against tumors.
Durable reprogramming of human T cells may now be possible thanks to a new technique based on the CRISPRoff and CRISPRon methodology. Researchers from the Arc Institute, Gladstone Institutes, and the University of California San Francisco (UCSF) have stably silenced or activated genes in this type of immune cell without cutting or altering its DNA, making T cells more resistant, active, and effective against tumors.
Two independent studies applied CRISPR-based genetic editing – one to treat leukemia and the other to target myeloma – to overcome the challenges faced by CAR T cells, such as exhaustion, impaired activation and fratricide, a phenomenon in which they attack each other.
There is still no effective vaccine or cure for HIV. Scientists are considering options ranging from longer-term antiretroviral therapy (ART) that space out injections by several years to long-lasting pre-exposure prophylaxis (PrEP) that acts as a vaccine while immunization is achieved. What else can be done?
There is still no effective vaccine or cure for HIV. Scientists are considering options ranging from longer-term antiretroviral therapy (ART) that space out injections by several years to long-lasting pre-exposure prophylaxis (PrEP) that acts as a vaccine while immunization is achieved. What else can be done? The “Innovations in HIV virology: Translating discoveries into novel therapies” symposium in basic science at the 13th IAS Conference on HIV Science (IAS 2025), which took place from July 13 to 17, 2025, in Kigali, Rwanda, showcased some of the new ideas that the scientific community are developing.
Aussie researchers have used CRISPR gene editing tools to “armor” chimeric antigen receptor (CAR) T cells to activate additional cancer-fighting proteins at the tumor site, enabling them to target cancer cells in solid tumors.
Aussie researchers have used CRISPR gene editing tools to “armor” chimeric antigen receptor (CAR) T cells to activate additional cancer-fighting proteins at the tumor site, enabling them to target cancer cells in solid tumors.
Investors found in an 8-K filing by Intellia Therapeutics Inc. the news of one case of liver-enzyme elevation in the ongoing phase III Magnitude study with nexiguran ziclumeran (nex-z, NTLA-2001), and in reaction pushed shares of the firm (NASDAQ:NTLA) down to close May 29 at $7.45, a loss of $2.21, or 23%, after the stock traded as low as $6.90 during the day.
Progressive multifocal leukoencephalopathy (PML) is a fatal demyelinating disease of the central nervous system caused by lytic infection of oligodendrocytes by pathogenic strains of the JC polyomavirus (JCV). This opportunistic virus has long persisted as a serious threat during periods of immunosuppression, and currently, there is no effective antiviral treatment available.