The Muscular Dystrophy Association (MDA)’s Kickstart program has announced receipt of both U.S. orphan drug and rare pediatric disease designations in support of work for congenital myasthenic syndrome caused by choline acetyltransferase (CHAT) gene deficiency. The FDA awarded the orphan drug designation to AVCHAT-01X (AAV serotype 9 human choline acetyltransferase).
Researchers from Universidad Carlos III de Madrid presented the development and preclinical characterization of a novel specific radiotracer for the diagnosis of Clostridioides difficile infection (CDI).
Ideaya Biosciences Inc. has announced FDA clearance of an IND application for IDE-275 (GSK-959), a small-molecule inhibitor of Werner helicase (WRN) discovered by Ideaya in collaboration with GSK plc.
AKT1 E17K is the most frequent gain of function mutation of the AKT1 gene. This mutation promotes pathologic localization of AKT1 to the plasma membrane and has been shown to induce leukemia in mice. Current options for AKT1 E17K-driven tumors are limited.
Ottimo Pharma Ltd. has emerged from stealth with a focus on developing innovative cancer therapies for solid tumors. The company’s lead program is jankistomig, a PD-1/VEGFR-2 bifunctional antibody.
The advantages of affibodies vs. antibodies are that they have a smaller size, better penetration and faster extravasation, can be produced both recombinantly and synthetically, and show robustness regarding protein scaffold.
Some rare skin diseases not only reduce the quality of life of patients, but also can be devastating conditions, leading to amputations or death. At the 31st annual congress of the European Society of Gene and Cell Therapy (ESGCT), held last week in Rome, different laboratories showcased their approaches to editing mutations related to this group of diseases.
Privately held Dyno Therapeutics Inc. has added another notch to its adeno-associated virus (AAV) vectors development portfolio in a deal with the Roche Group that includes $50 million up front and ultimately could top $1 billion. Dyno will help in developing next-generation AAV vectors, optimized by artificial intelligence, to target neurological diseases.
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