Verve Therapeutics Inc. has entered into an exclusive research collaboration with Eli Lilly and Co. focused on advancing Verve’s preclinical stage in vivo gene editing program targeting lipoprotein(a) (Lp[a]), a risk factor for atherosclerotic cardiovascular disease (ASCVD), ischemic stroke, thrombosis and aortic stenosis.

Researchers have ameliorated both monogenic and complex inflammation-driven diseases through transplantation of hematopoietic stem cells with an inserted IL-1 receptor antagonist (IL-1RA) gene. The team showed that in animal models the transplanted cells worked better than monoclonal antibodies to reduce symptoms in systemic autoinflammatory diseases (SAIDs), a group of childhood-onset, lifelong diseases that vary in severity depending on the underlying mutation, but can be life-threatening.

Researchers based at Uppsala University in Sweden have created an adeno-associated viral (AAV) vector treatment for glioma or glioblastoma that extended survival in a mouse model of the brain cancer. Writing in the May 11, 2023, issue of Cancer Cell, the researchers reported that by introducing an inflammatory protein known as LIGHT to the tumor microenvironment using the AAV treatment, the mice were better able to fight the cancer.

Myrtelle Inc. and Raaven Therapeutics AB have partnered on the development of novel recombinant adeno-associated virus (rAAV) vectors to advance gene therapy treatments for diseases of the central nervous system (CNS) in which myelin is affected.

Sparingvision SAS has obtained FDA clearance for its IND application for SPVN-06, its lead gene-independent therapy for the treatment of retinitis pigmentosa (RP). SparingVision has also submitted a clinical trial authorization (CTA) application to the French regulator (ANSM), which is currently under review.

Emulate Inc. has launched a new adeno-associated virus (AAV) transduction application for the Liver-Chip that enables gene therapy researchers to test the delivery efficiency and safety of AAV vectors in a validated, human-relevant model of the liver and get results in weeks.

The first gene therapy to treat severe hemophilia A was among the drugs recommended for European approval by regulators from the EMA’s CHMP at its monthly meeting. Manufactured by Biomarin Pharmaceutical Inc., Roctavian (valoctocogene roxaparvovec) was recommended for conditional marketing authorization in the EU for severe hemophilia A in adults who do not have factor VIII inhibitors and no antibodies to adeno-associated virus serotype 5.

Biotechs that tackle the effects of aging are beginning to make headlines: in January Altos Labs Inc. launched with a reported investment from Jeff Bezos. With Bezos getting involved with San Francisco-based Altos, the immediate reaction was that anti-aging biotechs would be there for the benefit of billionaires searching for eternal life. Not so, according to London U.K.-based Genflow Biosciences plc, which hopes to show that fighting aging is really about improving health as people age.