In a proof-of-concept study, researchers demonstrated the effectiveness of a potential new therapy for Timothy syndrome, an often life-threatening and rare genetic disorder that affects a wide range of bodily systems, leading to severe cardiac, neurological, and psychiatric symptoms as well as physical differences such as webbed fingers and toes. The treatment restored typical cellular function in 3D structures created from cells of people with Timothy syndrome, known as organoids, which can mimic the function of cells in the body. These results could serve as the foundation for new treatment approaches for the disorder. The study, supported by the National Institutes of Health (NIH), appears in the journal Nature.
RevOpsis Therapeutics secures $16.5M seed round: The San Carlos, CA-based biotech will use the funds to complete IND-enabling studies for its lead candidate, RO-104, for neovascular age-related macular degeneration and hire key management staff. First-in-human studies of RO-104 are expected to start in 2025, the company said.
“We’re in a frothy time,” acknowledges Alexandra Snyder, the head of R&D at Generate:Biomedicines.
The Colombian government on Wednesday issued a compulsory license for a commonly used drug to treat HIV, the first time the government there has stepped in to manufacture a drug without the drugmaker’s permission.
For the first time, researchers have used neural stem cells from rats from one species to restore the sense of smell in mice without olfactory cells.
CHICAGO, April 25 (Reuters) - U.S. officials have strengthened measures to contain the further spread of the first known outbreak of H5N1 or bird flu in dairy cows, which has now spread to 33 herds in eight states and bled over into the country's milk supply.
Freenome, a maker of blood-based cancer tests, will lay off more than 100 employees, or 20% of its workforce, a spokesperson for the company told Endpoints News.
Scientists have developed a sustainable new way of making complex molecules, which could greatly reduce waste produced during drug manufacturing, a study suggests.
DETROIT, April 24, 2024 /PRNewswire/ -- In observance of Parkinson's Disease Awareness Month, Quest Research Institute (Quest), a site within Alcanza Clinical Research's network located in Detroit, emphasizes the importance of raising awareness about Parkinson's disease (PD), recognizing its symptoms, and understanding the available clinical research avenues for patients.
Nature Publication Highlights the Benefits of Tropis® Intradermal When Used to Administer a First-in-Class Self-Amplifying mRNA Vaccine