Loqus23 Therapeutics Ltd. has closed a £35 million (US$43 million) series A financing, with the aim of supporting its work developing small-molecule somatic expansion inhibitors for the treatment of Huntington’s disease and other triplet repeat disorders.
Phosphodiesterase 4 (PDE4) is an enzyme known to play a role in inflammatory responses, and it has been pinpointed as an interesting therapeutic target for diseases like atopic dermatitis (AD), but limited efficacy and side effects have prevented the approval of oral formulations.
Darier disease (DD) is a rare cutaneous disease caused by heterozygous mutations in the ATP2A2 gene, which encodes sarco/endoplasmic reticulum calcium ATPase isoform 2 (SERCA2).
Shattuck Labs Inc. has announced a strategic shift to focus on SL-325, a death receptor 3 (DR3) antagonist antibody for the treatment of inflammatory bowel disease (IBD) and other inflammatory autoimmune diseases.
Epivario Inc. has been awarded a small business innovation research (SBIR) grant of $275,000 from the National Institute on Drug Abuse (NIDA) to advance research toward the development of a novel epigenetic regulator as a treatment for opioid use disorder.
Artelo Biosciences Inc. has presented data on its fatty acid-binding protein 5 (FABP5) inhibitor ART-26.12 from testing of its efficacy in preclinical models of psoriasis.
The FDA has awarded orphan drug designation to Papillon Therapeutics Inc.’s PPL-002, an experimental gene-modified CD34+ hematopoietic stem and progenitor cell (HSPC) therapy, for the treatment of Danon disease.
Scientists from 4D Molecular Therapeutics Inc. disclosed the preclinical evaluation of 4D-710, an aerosolized gene therapy that consists of a lung-specific evolved A101 capsid vector, the promoter CMV173 and the transgene codon-optimized human CFTRΔR.
Italian researchers have presented a case study of pediatric advanced sleep-wake phase syndrome in a 4-year-old girl. Autosomal dominant forms of this syndrome due to loss-of-function mutations in the gene coding casein kinase I isoform delta (CSNK1D) have previously been described.
In the year’s fourth-largest deal, Prime Medicine Inc. will collaborate with Bristol Myers Squibb Co. in a research collaboration and license agreement totaling $3.61 billion. The two companies plan to develop reagents for ex vivo T-cell therapies. While the programs and targets have yet to be disclosed, BMS is expanding its CAR T development, begun more than five years ago, with this deal.