Beam Therapeutics Inc. has received clearance of its clinical trial authorization (CTA) application by the U.K.’s Medicines and Healthcare Products Regulatory Agency for BEAM-302, an in vivo base editor, as a potential treatment for patients with α1-antitrypsin deficiency (AATD).
Atamyo Therapeutics SAS has received clinical trial application (CTA) authorizations in Italy and France for ATA-200, its gene therapy for the treatment of γ-sarcoglycan related limb-girdle muscular dystrophy type 2C/R5 (LGMD2C/R5).
Scientists at the Karolinska Institutet in Sweden have found a new region in the c-Myc oncogene that would allow the development of a binding compound to target it. Their discovery is based on a structural switch that leads to open and closed conformations of the domain, allowing or not its interaction with a protein required for the oncogenic activity of c-Myc.
Researchers from Jinan University (Guangdong) and affiliated organizations reported new data detailing the discovery and preclinical characterization of novel fibroblast growth factor receptor 4 (FGFR4) inhibitors for the treatment of hepatocellular carcinoma (HCC).
Mucosa-associated lymphoid tissue lymphoma translocation protein 1 (MALT1) is a key regulator of physiological antigen receptor signaling in B cells and T cells, as it is the only component of the MALT1-BCL10-CARD11 (CBM) signalosome with proteolytic activity.
Cinfina Pharma Inc. has obtained FDA clearance of its IND application for CIN-110, a PYY3-36 analogue, allowing it to initiate a first in-human study in otherwise healthy subjects with obesity. CIN-110 is a stable and long-acting analogue of PYY3-36 being developed both as a monotherapy and for co-administration for obesity.
Ubiquigent Ltd. has signed an agreement with Debiopharm International SA to support the development of Debiopharm’s preclinical ubiquitin carboxyl-terminal hydrolase 1 (USP1) inhibitor program, Debio-0432.
Defence Therapeutics Inc. has announced the successful testing of a second-generation anticancer vaccine, ARM-002, using its lead anticancer molecule Accutox. When tested as a therapeutic vaccine in a melanoma cancer model, ARM-002 led to an 80% complete response when combined with an anti-PD-1 immune-checkpoint inhibitor.
Recent genome-wide association studies identified an association between low bone mineral density (BMD) and a single-nucleotide polymorphism (SNP) at the MALAT1 locus, but there is no functional evidence on the role of MALAT1 alterations in BMD or osteoporosis. Hence, scientists at MD Anderson Cancer Center aimed to assess the functional role of MALAT1 alterations in low BMD and osteoporosis.
Scientists from the Australian National University have discovered the gene mutation responsible for causing psoriasis, and the findings could lead to improved diagnosis and treatment for patients with psoriasis and psoriatic arthritis, a chronic inflammatory skin disease. “We were able to identify the gene that could be important in enabling this progression from a skin-only condition to a skin-and-joint condition,” lead study author Chelisa Cardinez told BioWorld.
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