From glaucoma to Stargardt disease, age-related macular degeneration (AMD) to retinitis pigmentosa, or a corneal transplant to Bietti’s crystalline dystrophy, the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) is working to bring some light to patients with age and congenital diseases that affect vision. From May 7-11, 2024, thousands of scientists are gathering in Baltimore to show their advances against the challenges of delivering genes and cells to the correct place, avoiding immunogenicity and improving diseases. Read More

Researchers from Xiangya Hospital of Central South University published data from a study that explored potential treatment targets for cutaneous squamous cell carcinoma (cSCC) by investigating the regulatory mechanisms of follistatin-like 1 (FSTL1), which has been shown to be implicated in a variety of cancers. Read More

Chronic granulomatous disease (CGD) is an immunodeficient disorder that is caused by mutations in genes that encode proteins of the nicotinamide adenine dinucleotide phosphate (NADPH) oxidase enzyme complex. Read More

Crispr Therapeutics AG has expanded its in vivo pipeline with two new programs, which utilize lipid nanoparticle (LNP)-based delivery of CRISPR/Cas9 gene-editing cargo to the liver. Read More

Université de Montréal has disclosed serine/threonine-protein kinase A-Raf, (ARAF) and/or B-Raf (BRAF) and/or RAF proto-oncogene serine/threonine-protein kinase (c-Raf) inhibitors reported to be useful for the treatment of cancer. Read More

The recent cyberattack on Change Healthcare, a part of the Unitedhealth Group, crippled reimbursement claims processing for thousands of providers for several weeks and potentially exposed troves of patient data. Congressional committees are investigating the attack, its scope, and Unitedhealth’s response. Please take a few minutes to fill out this short survey to help BioWorld understand how your company is responding to these threats. You can click through to the questions here. Read More

Vascular dysfunction that causes leakage of plasma proteins such as fibrinogen, fibrin deposits and innate immune cell activation is the cause of neurodegenerative ocular diseases, including diabetic macular edema, age-related macular degeneration or diabetic retinopathy. Read More

Norwegian University of Science and Technology (NTNU) has synthesized macrophage colony-stimulating factor 1 receptor (CSF-1R; CD115; c-Fms) inhibitors reported to be useful for the treatment of cancer, amyotrophic lateral sclerosis, Charcot-Marie-Tooth disease, macular degeneration, osteoarthritis, osteoporosis, rheumatoid arthritis and Alzheimer’s disease, among others. Read More

Allorion Therapeutics (Guangzhou) Co. Ltd. has identified phosphoramidate compounds targeting aldo-keto reductase family 1 member C3 (AKR1C3; 17β-HSD5) reported to be useful for the treatment of cancer. Read More

At the recently concluded ARVO meeting, Splicebio S.L. presented the first preclinical results on the company’s new candidate, SB-007, for the treatment of Stargardt disease, a rare genetic disease caused by mutations in the ABCA4 gene with no approved treatment on the market. Read More

Zhuhai Yufan Biotechnologies Co. Ltd. has divulged mitogen-activated protein kinase kinase kinase kinase 1 (MAP4K1; HPK1; MEKKK1) inhibitors reported to be useful for the treatment of cancer, chronic obstructive pulmonary disease, AIDS, hemolytic anemia, psoriasis, scleroderma, systemic lupus erythematosus and ulcerative colitis, among others. Read More

Raytone Biotechnology Co. Ltd. has described compounds based on oxidative stress and antiamyloid aggregations reported to be useful for the treatment of cataracts and presbyopia. Read More

Researchers from Indiana University presented data from a study that aimed to assess the therapeutic potential of interleukin-9 (IL-9) blockade in the context of lung cancer. Read More