A preclinical study presented at the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, showed a new epigenetic editing technology that enables durable gene silencing using ELXRs, short for Epigenetic Long-Term X-Repressors. With this approach, scientists at Scribe Therapeutics Inc. successfully inhibited the expression of the PCSK9 gene, a key regulator of cholesterol metabolism, in human cells, mice and nonhuman primates.

As the many challenges facing cell therapies are being addressed, the CAR T field continues to evolve beyond its original design of T cells engineered to target hematological malignancies. During the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, several studies showed how this technology is being redefined as programmable and adaptable immune cells with expanded functional versatility.

Experimental drugs that directly inhibit the NSD2 enzyme have shown potential as an effective strategy against hard-to-treat cancers, such as lung and pancreatic tumors driven by KRAS mutations. The therapeutic mechanism involves reversing a histone H3 methylation that promotes open chromatin and the expression of oncogenes.

Experimental drugs that directly inhibit the NSD2 enzyme have shown potential as an effective strategy against hard-to-treat cancers, such as lung and pancreatic tumors driven by KRAS mutations. The therapeutic mechanism involves reversing a histone H3 methylation that promotes open chromatin and the expression of oncogenes.

While people living with HIV can lead virtually normal lives thanks to antiretroviral therapy (ART), HIV persists in a latent state within cellular reservoirs that scientists do not know how to eliminate. “Transcription is a critical step in the viral life cycle. … But there are currently no drugs suppressing HIV transcription, and that may be one of the reasons why current antiretroviral therapy is not curative,” Melanie Ott told the audience at the 13th IAS Conference on HIV Science this week in Kigali, Rwanda.

The variety of blood cells decreases with age. Some are lost, while others become dominant, leading to a loss of functional diversity. This, in turn, weakens the immune system in older individuals and increases the risk of developing hematological diseases. Scientists in Barcelona have developed a method based on DNA methylation that works like a barcode. EPI-Clone identifies and traces the origin of blood cells to measure the complexity of these clones in aging humans and mice.

The variety of blood cells decreases with age. Some are lost, while others become dominant, leading to a loss of functional diversity. This, in turn, weakens the immune system in older individuals and increases the risk of developing hematological diseases. Scientists in Barcelona have developed a method based on DNA methylation that works like a barcode. EPI-Clone identifies and traces the origin of blood cells to measure the complexity of these clones in aging humans and mice.

Epicrispr Biotechnologies Inc. has secured $68 million in the first close of its series B financing to support the clinical development of EPI-321, a first-in-class epigenetic therapy for facioscapulohumeral muscular dystrophy (FSHD).

The first complete DNA methylation atlas of 39 human cell types reveals which genes can talk or are silenced, depending on whether their sequence is linked to methyl group epigenetic modification that regulates their expression. This map of on-and-off switches shows differences between the alleles inherited from the father and those from the mother, providing a view of gene expression that can be explored in health and disease independently of the information contained in the DNA sequence.

While first-generation glucagon-like peptide-1 receptor agonists have clearly taken the obesity market by storm, generating billions of dollars for Novo Nordisk A/S and Eli Lilly and Co., several other companies are developing follow-on products that could clean up the tolerability and adherence issues of Wegovy (semaglutide) and Zepbound (tirzepatide).