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.
The Global Health Innovative Technology (GHIT) Fund has announced a total investment of approximately ¥1.64 billion (US$10.8 million) in four projects for the development of new drugs for malaria and neglected tropical diseases.
“Prenatal therapies are the next disruptive technologies in health care, which will advance and shape the future of patient care in the 21st century,” said Graça Almeida-Porada, a professor at the Fetal Research and Therapy Center of the Wake Forest Institute for Regenerative Medicine in Winston-Salem, North Carolina. At the American Society of Gene & Cell Therapy (ASGCT) annual meeting in Baltimore on May 5, 2024, Almeida-Porada introduced the first presentation of the scientific symposium “Prospects for Prenatal Gene and Cell Therapy.”
MicroRNAs (miRNAs) are small noncoding RNAs that play important roles in immune response regulation in autoimmune disease such as autoimmune hepatitis. Researchers investigated whether Δ8-tetrahydrocannabinol (THC), which is present in marijuana, could prevent the development of autoimmune hepatitis in treated mice by altering miRNA expression.
Vyne Therapeutics Inc. has received IND clearance from the FDA allowing it to initiate a first-in-human phase Ia study of VYN-202, an oral small-molecule BD2-selective BET inhibitor for autoimmune diseases.
Autoantibodies can promote inflammation and activate complement pathways and immune cell responses causing tissue damage in autoantibody-driven diseases. Researchers from Seismic Therapeutic Inc. have presented data on S-1117.
Repertoire Immune Medicines Inc. has entered a multiyear strategic collaboration with Bristol Myers Squibb Co. (BMS) to develop tolerizing vaccines for up to three autoimmune diseases.
Females have a much greater risk of developing an autoimmune disease than males do. Eighty percent of autoimmune disease patients are female, and specific disorders can have an even more lopsided ratio – 90% of systemic lupus erythematosus (lupus) and almost 95% of Sjögren’s disease patients are female.
Biolojic Design Ltd. announced that Nektar Therapeutics Inc. has exercised its license option to develop a program resulting from the companies’ 2021 research collaboration, established to design and test agonistic antibodies targeting tumor necrosis factor receptor type II (TNFR2).
Researchers from Biohaven Pharmaceuticals Inc. presented preclinical data for the first-in-class extracellular bispecific IgG degrader, BHV-1300, being developed for the treatment of neuroinflammatory and autoimmune disease.
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