Loqus23 Therapeutics Ltd. has raised £35 million (US$46.6 million) in a series A to take forward small molecules it has discovered for the treatment of Huntington’s disease and other conditions that are driven by DNA mismatch repair (MMR). MMR fixes DNA insertions, deletions and misincorporation errors that occur during transcription and/or cellular replication. Smaller repairs are directed by MutSalpha, a protein that binds single base mismatches, while MutSbeta handles larger insertion/deletion loops. Huntington’s and other triplet repeat diseases are caused when trinucleotide repeats accumulate in somatic DNA to the extent that they interfere with protein expression. Read More
It’s well past time for the U.S. FDA to end its silence on what device patents can be listed in the Orange Book as part of a drug-device combination product, Sen. Bill Cassidy (R-La.) said in an Oct. 1 letter that took FDA Commissioner Robert Califf to task for letting the FTC do the FDA’s job. Read More
Immunotherapy company Cartherics Pty Ltd. raised AU$15 million (US$10.3 million) in an oversubscribed series B round that will support the first clinical trial for lead chimeric antigen receptor natural killer therapy CTH-401 for ovarian cancer, and to expand its pipeline to include other diseases. Read More
Neurobo Pharmaceuticals Inc., of Cambridge, Mass., reported top-line phase Ia study results of its obesity drug candidate, DA-1726, Sept. 30, causing the company’s shares to lose 11.7% of their value over two days. Read More
A collaboration led by the Flywire Consortium and comprising hundreds of scientists has completed a whole map of the adult fruit fly brain after several decades of collaborative work. By using electron microscopy and three-dimensional reconstruction supported by AI tools, the researchers have revealed the neural wiring of the Drosophila melanogaster brain, a connectome of 140,000 neurons with 50 million synaptic connections. In the future, researchers could possibly use this map as an artificial in silico model to study the brain as a simulator through its connections, though a lot of work remains to be done for this. Read More
Linda Marbán, CEO of Capricor Therapeutics Inc., discusses deramiocel (CAP-1002), the company’s allogeneic cardiac-derived cell therapy, for treating Duchenne muscular dystrophy. The company just wrapped up a visit with the U.S. FDA and is prepping to file a BLA for its treatment of the rare disease. Read More
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Eirion, Gritstone, Hamlet, Incarda, Jaguar, Kiromic, Kura, Myrtelle, Plus, Remedy, Repare. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Astrazeneca, Bpgbio, Clinuvel, Daiichi, Formycon, Fresenius, Invivyd, Ionis, J&J, Oryzon, Papillon, PTC, Sagimet, Thryv. Read More
