Repair Biotechnologies Inc. has received encouraging feedback from a pre-IND meeting with the FDA as it works toward an IND filing to conduct a phase Ib study of its REP-0003 mRNA therapy in patients with homozygous familial hypercholesterolemia (HoFH).

Axcynsis Therapeutics Pte Ltd. has gained IND clearance from the FDA for AT03-65 for the treatment of patients with CLDN6-positive solid tumors. Axcynsis is planning to initiate a phase I trial in the U.S. during the current quarter in patients with advanced CLDN6-positive solid tumors.

Pheno Therapeutics Ltd. has received clinical trial authorization (CTA) from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) for its lead candidate, PTD-802. The program will progress to a first-in-human phase I trial.

Rise Therapeutics LLC has gained IND clearance from the FDA to initiate a phase I trial of R-5780 in patients with cancer.

Ips Heart Inc. has been awarded orphan drug designation by the FDA for its GIVI-MPC stem cell therapy for Becker muscular dystrophy. GIVI-MPC has the unique ability to create new muscle with full length dystrophin in Becker muscular dystrophy.

Pulsesight Therapeutics SAS has submitted a clinical trial authorization (CTA) to the French authority ANSM seeking to conduct a first-in-human phase I trial of PST-611 for dry age-related macular degeneration (AMD)/geographic atrophy (GA).

CSPC Pharmaceutical Group Ltd. has obtained clinical trial approval from China’s National Medical Products Administration (NMPA) for SYS-6041, an antibody-drug conjugate, for advanced solid tumors.