The FDA has awarded orphan drug designation to Papillon Therapeutics Inc.’s PPL-002, an experimental gene-modified CD34+ hematopoietic stem and progenitor cell (HSPC) therapy, for the treatment of Danon disease.

The FDA has awarded U.S. orphan drug designation to Eydisbio Inc.’s EYD-001 (formerly HS-276), a highly selective and potent, orally bioavailable TAK1 inhibitor for the treatment of systemic sclerosis. Eydisbio plans to initiate clinical trials in the near future.

The FDA has cleared an IND application for a phase I/II study of KSQ-004EX, KSQ Therapeutics Inc.’s second engineered tumor-infiltrating lymphocyte (eTIL) program. KSQ-004EX consists of TIL in which the genes encoding SOCS1 and Regnase-1 are inactivated using CRISPR/Cas9 gene editing.

Innate Pharma SA has obtained IND clearance from the FDA for IPH-4502, its novel and differentiated topoisomerase I inhibitor antibody-drug conjugate (ADC) conjugated to exatecan targeting Nectin-4 in solid tumors. In nonclinical models, IPH-45 was well tolerated and showed antitumor efficacy in vitro and in vivo.

Chengdian (Suzhou) Biopharmaceutical Co. Ltd. has received IND clearance from the FDA, allowing it to initiate a first-in-human phase I trial of CD-001.

Papillon Therapeutics Inc.’s PPL-001 has been awarded orphan drug designation by the FDA for Friedreich’s ataxia. PPL-001 is an experimental gene-corrected CD34+ hematopoietic stem and progenitor cell (HSPC) therapy.

Somite Therapeutics Inc.’s lead program, SMT-M01, has been awarded orphan drug and rare pediatric disease designations by the FDA for the treatment of Duchenne muscular dystrophy (DMD). The program leverages the company’s proprietary Alphastem artificial intelligence (AI) platform to develop a novel cell replacement therapy for DMD.

Cullinan Therapeutics Inc. has submitted an IND application to the FDA to evaluate its CD19 x CD3 bispecific T-cell engager, CLN-978, for the treatment of systemic lupus erythematosus (SLE).

Medigene AG has obtained FDA clearance of its IND application for lead program MDG-1015 for the treatment of advanced gastric cancer, ovarian cancer, myxoid/round cell liposarcoma and synovial sarcoma.

Abdera Therapeutics Inc.’s ABD-147 has been granted U.S. orphan drug designation for the treatment of neuroendocrine carcinoma. ABD-147 is a next-generation precision radiopharmaceutical biologic therapy designed to deliver actinium-225 (225Ac) to solid tumors expressing DLL3.