Leukogene Therapeutics Inc.’s lead product candidate LTI-214 (M2T-CD33) has been awarded orphan drug designation by the FDA for the treatment of acute myeloid leukemia (AML).

The U.S. FDA has granted orphan drug designation to Dewpoint Therapeutics Inc.’s DPTX-3186, its first-in-class condensate modulator for the treatment of gastric cancer. The designation follows the recent opening of Dewpoint’s IND application for DPTX-3186 earlier in October, and is the first orphan designation ever granted to a condensate-modulating therapeutic.

Exousia Pro Inc.’s subsidiary, Exousia AI, has received orphan drug designation from the FDA for its exosome-based treatment for malignant glioma.

Resolute Science Inc.’s RS-5 has been awarded orphan drug designation by the FDA for the treatment of soft tissue sarcomas.

Dewpoint Therapeutics Inc. has announced an IND in the U.S. for DPTX-3186, a first-in-class oral condensate modulator designed to selectively disrupt oncogenic Wnt/β-catenin signaling in tumors. Dosing is set to begin before year-end in a phase I/II trial conducted in partnership with cancer centers and opinion leaders in gastric and other Wnt-driven cancers.

Tr1x Inc. has obtained IND clearance from the FDA for TRX-319, paving the way for initiation of a phase I/IIa study in progressive multiple sclerosis (MS) early next year. TRX-319 is designed to combine targeted B-cell control with active anti-inflammatory signaling and T-cell regulation, with the goal of restoring immune balance.

Corventum Inc. has gained IND clearance from the FDA for CVT-130 for the prevention of anthracycline-related cardiotoxicity. In cancer patients, the use of anthracyclines as chemotherapy is effective but limited by cumulative heart damage. Protecting against this chemotherapy-induced cardiac injury could enable retreatment for patients with recurrent cancer.

Revir Therapeutics Inc.’s RTX-117 has been awarded orphan drug designation by the FDA for Charcot-Marie-Tooth disease. RTX-117 is a small-molecule therapy designed to activate eIF2B to restore translation of cap-dependent mRNAs to normalize protein expression.