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. These diseases include myotonic dystrophy, spinocerebellar ataxias, spinal and bulbar muscular atrophy, and Friedreich ataxia.

Sen. Cassidy: Time for US FDA to reclaim its Orange Book duties

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. “Despite nearly two decades of requests from manufacturers for more clarity, FDA has still not told industry how it should list patents for drug-device combinations,” Cassidy said, adding that the agency’s continuing silence is “untenable.” Reminding Califf that Congress charged the FDA with administering the Orange Book, the senator said letting the FTC enforce Orange Book listings “is an extraordinary abdication of authority.”

Cartherics' AU$15M series B advances CTH-401 for ovarian cancer

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 (CAR) natural killer (NK) therapy CTH-401 for ovarian cancer, and to expand its pipeline to include other diseases. Cartherics CEO Alan Trounson told BioWorld that the funds raised will take Cartherics through to mid-2026, and the phase I Australian trial in ovarian cancer will begin in the fourth quarter of 2025. Existing investors and new unnamed private investors contributed to the round. The ovarian cancer product is an off-the shelf iPS cell product made from homozygous HLA cord blood cells that are made into iPS cells, and then gene-edited to introduce a CAR and knock out inhibitory genes that turn off immune cells from killing solid tumors, Trounson said.

Neurobo stock falls on phase I obesity data for DA-1726

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 to lose 11.7% of its value over two days. As previously reported by BioWorld, DA-1726 is a novel dual oxyntomodulin (OXM) analog agonist that functions as a glucagon-like peptide-1 receptor (GLP-1R) and glucagon receptor (GCGR). OXM is a naturally occurring gut hormone that activates GLP-1R and GCGR to help decrease food intake and increase energy expenditure. Designed as a once-weekly subcutaneously administered therapy, DA-1726 is in development for both obesity and metabolic dysfunction-associated steatohepatitis (MASH).

140,000 cells and 50M synapses make 1 adult fly brain

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. “I would not say that this is a final step. This is the first step,” Sebastian Seung, a professor at Princeton Neuroscience Institute and Department of Computer Science, told BioWorld.

BioWorld Insider podcast: Capricor’s CEO pursues a BLA and talks rare disease

Capricor Therapeutics Inc. just wrapped up a visit with the U.S. FDA and is prepping to file a BLA in October for its Duchenne muscular dystrophy treatment. Linda Marbán, Capricor’s CEO, is the guest on the newest BioWorld Insider podcast and she talks about deramiocel (CAP-1002), the company’s allogeneic cardiac-derived cell therapy, for treating the rare disease and how the FDA has made strong efforts in helping lay the groundwork for deramiocel. Marbán has been working on the Duchenne treatment for many years and she has strong insights into how to tackle a rare disease development program and how the FDA has changed its ways over time to help out. She has been in the biopharma space for more than 20 years and also is a co-founder of Capricor.

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