Kexing Biopharm Co. Ltd. has announced IND clearances this month by China’s National Medical Products Administration (NMPA) and the U.S. FDA for GB-18, a biologic product for the treatment of cancer cachexia.

Latus Bio Inc. is developing a new gene therapy, LTS-101, for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2), a form of Batten disease characterized by deficiency in the tripeptidyl peptidase 1 (TPP1) protein that leads to lysosomal dysfunction and neurodegeneration.

The lack of animal models that mimic human disease impedes the study of many pathologies that still lack treatment beyond symptom relief. This is what has happened so far with PURA syndrome, a rare disorder affecting brain development for which a mouse model has finally been developed. Other times, small and large models exist, but an effective treatment remains elusive, as is the case with Krabbe disease, a fatal disease in children that could be prevented with the advances in gene therapy.

Since the development of the base and prime editing technique by David Liu at the Broad Institute, their applications in biomedicine have continued to grow, reaching 17 clinical trials for base editing and one clinical assay for prime editing. The 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) marked a historic milestone this year by presenting the first case of treatment with base editors of a baby with a deadly metabolic disease.

Repair Biotechnologies Inc.’s REP-0003 has been awarded orphan drug designation by the FDA for the treatment of homozygous familial hypercholesterolemia (HoFH).

Cancer cachexia is a devastating condition that affects up to 80% of advanced cancer patients and causes approximately 2 million deaths worldwide annually. Cancer cachexia is characterized by uncontrolled weight loss and severe muscle wasting. Despite its significant impact, effective treatments remain elusive.