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).

Ribox Therapeutics Ltd. has obtained IND clearance from the FDA for the company’s circular RNA therapy, RXRG-001.

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.