Researchers from Fate Therapeutics Inc. presented preclinical data for the multiplexed-engineered, off-the-shelf chimeric antigen receptor (CAR) natural killer (NK) cell therapy, FT-522, as a potential therapeutic against autoimmune diseases.

Kate Therapeutics Inc. recently presented data on a novel muscle- and heart-targeted, liver de-targeted development candidate for the treatment of Duchenne muscular dystrophy (DMD) – KT-809.

Vrise Therapeutics Inc. has developed and presented a USP1 inhibitor, VRTX-531, for the potential treatment of BRCA1/2-mutated HDR+ breast cancer.

Researchers from Ikarovec Ltd. have presented preclinical data for the AAV8-IKC159V, the company’s lead bicistronic gene therapy being developed for the treatment of geographic atrophy, a late stage of geographic dry AMD.

Intravitreal VEGF inhibitors are the first-line treatment for vascular retinopathies, but they are invasive, painful and require frequent treatments.

Breast cancer is a heterogenous disease in terms of its prognosis and treatment response. Metabolic reprogramming is a potential therapeutic target because of its repercussion on oncogenesis.

The success of a vaccine, a gene editing design for an untreated disease, or achieving cell engraftment after several attempts, comes from years of accumulated basic science studies, thousands of experiments, and clinical trials. Innumerable steps precede hits in gene and cell therapies before a first-time revelation, and most of them are failures at the time. At the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) in Baltimore last week, several groups of scientists presented achievements that years ago looked impossible.