Imagine glass shards being smashed relentlessly into your bones over and over again. That is what the pain of sickle cell disease feels like, a young girl once told Velvet Brown-Watts, who three years ago started a local support group for sickle cell patients and their families. “I thought that...
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SEOUL, South Korea — SillaJen, Inc. (KOSDAQ: 215600) has submitted CSR to the US FDA on 06 Feb 2024 for REN026, a phase 1b/2a dose escalation and safety/efficacy evaluation study of Pexa-Vec in combination with cemiplimab in patients with metastatic or unresectable renal cell carcinoma (RCC). The study demonstrated an...
Bochum, Germany – Diabetes often remains undetected until it has already damaged organs or nerves. This is partly due to the fact that diagnosis at an early stage is time-consuming and difficult. An international team of researchers headed by Associate Professor Dr. Johannes Dietrich from the Department of Medicine I...
San Diego, Calif. – Scientists at UC San Diego have developed a machine learning algorithm to simulate the time-consuming chemistry involved in the earliest phases of drug discovery, which could significantly streamline the process and open doors for never-before-seen treatments. Identifying candidate drugs for further optimization typically involves thousands of...
Pittsburgh, Pennsylvania – In Lambert-Eaton myasthenic syndrome (LEMS), patients’ immune systems attack a critical nerve cell protein, causing muscle weakness and degeneration. But not always — some patients lack antibodies to this protein, the calcium channel. Using the MCell simulation software developed in collaboration with the Pittsburgh Supercomputing Center (PSC),...
Baltimore, Maryland – A single dose of the typhoid conjugate vaccine, Typbar TCV®, provides lasting efficacy in preventing typhoid fever in children ages 9 months to 12 years old, according to a new study conducted by researchers at University of Maryland School of Medicine’s (UMSOM) Center for Vaccine Development and...
In a new resource for the scientific community, published today in Nature Biotechnology, researchers in the lab of Neville Sanjana at the New York Genome Center (NYGC) and New York University (NYU) developed CRISPR-sciATAC, a novel integrative genetic screening platform that jointly captures CRISPR gene perturbations and single-cell chromatin accessibility genome-wide. With this technology, they...
A next-generation technology that allows the study of protein expression at the single-cell level and the location of the cells within the tumor microenvironment (TME) was feasible and provided information on the benefit of adding the immune checkpoint inhibitor atezolizumab (Tecentriq) to chemotherapy as neoadjuvant treatment for patients with early...
BOSTON, Mass. — Sionna Therapeutics, a life sciences company dedicated to developing highly effective and differentiated treatments for cystic fibrosis (CF), today announced that the first healthy subject has been dosed in a Phase 1 clinical trial of SION-109 following clearance of its Investigational New Drug application (IND) by the...
BOSTON — Sionna Therapeutics, a clinical-stage life sciences company dedicated to developing highly effective and differentiated treatments for cystic fibrosis (CF), today announced the presentation of preclinical data on highly potent Series 2 nucleotide binding domain-1 (NBD1) stabilizers, SION-719 and SION-451. These data show that SION-719 and SION-451 fully restore...