Pepgen Inc. has received a no objection letter from Health Canada for its clinical trial application (CTA) to initiate a phase I study of PGN-EDODM1 in patients with myotonic dystrophy type 1 (DM1). Initial data from the study are expected next year.

Uniqure NV has received FDA clearance of its IND application for AMT-260, the company’s gene therapy candidate that represents a potential one-time administered approach to treating refractory mesial temporal lobe epilepsy (MTLE).

Tscan Therapeutics Inc. has received FDA clearance of its IND application for TSC-203-A0201, a T-cell receptor (TCR)-engineered T-cell therapy (TCR-T) targeting preferentially expressed antigen in melanoma (PRAME). PRAME is expressed in melanoma and other solid tumors, including head and neck cancers and non-small-cell lung cancers. TSC-203-A0201 is specific for patients with HLA type A*02:01.

Harbour Biomed Ltd. has received FDA clearance of its IND application to commence a phase I trial of HBM-9033 in advanced solid tumors. Developed in collaboration with Medilink Therapeutics (Suzhou) Co. Ltd., HBM-9033 is an antibody-drug conjugate that specifically targets human mesothelin (MSLN), an upregulated tumor-associated antigen in various solid tumors, including mesothelioma, ovarian cancer, lung cancer, breast cancer and pancreatic cancers. The fully human monoclonal antibody in HBM-9033, generated from the Harbour Mice platform, binds preferably to membrane bond MSLN over soluble MSLN, which minimizes the interference of the shedding MSLN on the binding and internalization of the membrane bond MSLN.

Akava Therapeutics Inc. has announced FDA clearance of its IND application for AKV-9 (formerly NU-9) for the treatment of amyotrophic lateral sclerosis (ALS). The company is planning a first-in-human phase I study in healthy subjects.

Ideaya Biosciences Inc. has announced FDA clearance of an IND for a GSK-sponsored phase I/II trial of GSK-101 (IDE-705), a small-molecule inhibitor of Polϴ helicase, in combination with GSK's PARP inhibitor niraparib tosylate monohydrate for the treatment of tumors with BRCA or other homologous recombination mutations or homologous recombination deficiency.

Novarock Biotherapeutics Ltd., a subsidiary of CSPC Pharmaceutical Group Ltd., has received FDA approval of its IND application for NBL-028, a CLDN6-CD137 bispecific antibody.