Tr1x Inc. has announced that the FDA has cleared its IND application for its engineered Tr1 Treg cell therapy TRX-103 for treatment-refractory Crohn’s disease.
After receiving Orphan Drug Designation earlier this year, Satellos Bioscience Inc. announced that the FDA has granted Rare Pediatric Disease Designation to SAT-3247 for the potential treatment of Duchenne muscular dystrophy (DMD).
Chengdu Westgene Biopharma Co. Ltd. (Westgene) has received approval from both China's National Medical Products Administration (NMPA) and the FDA for its therapeutic cancer vaccine WGc-043 to enter first-in-human trials.
Meta Pharmaceuticals Inc. announced that the FDA has granted rare pediatric disease designation to its investigational new drug META-001-PH for the treatment of primary hyperoxaluria (PH), an autosomal recessive metabolic disorder in which oxalate is overproduced and deposited in the body.
Innorna Co. Ltd.’s IN-016 has received orphan drug status from the U.S. FDA for the treatment of progressive familial intrahepatic cholestasis (PFIC). Earlier in July, IN-016 had been granted Rare Pediatric Disease Designation. PFIC is a group of rare genetic disorders associated with defects in bile acid secretion or transport, resulting in unwanted bile accumulation within the liver.
Umoja Biopharma Inc. has received clearance from the FDA to begin a first-in-human trial of its CAR T product UB-VV111. The company expects to dose the first patient by the end of 2024.
Gigagen Inc., a subsidiary of Grifols SA, has received clearance from the FDA of its IND application to conduct a phase I trial of GIGA-2339 for the treatment of hepatitis B virus (HBV) infections.
Intellia Therapeutics Inc. has received clearance from the U.K. Medicine and Healthcare products Regulatory Agency (MHRA) to initiate a phase I/II study of NTLA-3001 for the treatment of α1-antitrypsin deficiency (AATD)-associated lung disease.
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