Resvita Bio Inc.’s RVB-003 has been awarded orphan drug designation by the FDA for the skin disorder Netherton syndrome. RVB-003 was previously granted rare pediatric disease designation.

Sun Pharma Advanced Research Co. Ltd. has filed an IND application with the FDA for SBO-154 for the treatment of solid tumors. A global phase I study is planned in advanced solid tumors.

Epicrispr Biotechnologies Inc. has secured $68 million in the first close of its series B financing to support the clinical development of EPI-321, a first-in-class epigenetic therapy for facioscapulohumeral muscular dystrophy (FSHD).

Pleco Therapeutics BV has held a successful pre-IND meeting with the FDA to discuss the continued development of PTX-252, an intravenous formulation designed to treat patients with acute myeloid leukemia (AML).

Palatin Technologies Inc. has obtained U.S. orphan drug designation for PL-7737 for leptin receptor (LEPR) deficiency, including obesity caused by this condition.

Sineugene Therapeutics Co. Ltd. has obtained IND clearance from the FDA for SNUG-01, a first-in-class tripartite motif protein 72 (TRIM72)-targeted gene therapy candidate for amyotrophic lateral sclerosis (ALS). A phase I/IIa trial will evaluate SNUG-01 in adults with ALS.

Everest Medicines Ltd. has obtained IND clearance from the FDA for EVM-14, an off-the-shelf mRNA cancer vaccine targeting multiple tumor-associated antigens and designed to treat various cancers, including non-small-cell lung cancer and head and neck cancer.

Entrada Therapeutics Inc. has received authorization from the U.K.’s Medicines and Healthcare Products Regulatory Agency (MHRA) and research ethics committee to initiate a phase I/II study of ENTR-601-45 for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation in the DMD gene amenable to exon 45 skipping.

CSPC Pharmaceutical Group Ltd. has obtained clearance from China’s National Medical Products Administration (NMPA) to conduct clinical trials with SYS-6040 for advanced solid tumors.