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.

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.

Actinium Pharmaceuticals Inc. has received FDA clearance of an IND application to study Iomab-ACT for targeted conditioning prior to a bone marrow transplant (BMT) in patients with sickle cell disease. 

Tiximed Inc. has received IND clearance for its inhibitor of thioredoxin-interacting protein (TXNIP), TIX-100, in individuals with type 1 diabetes. TIX-100 is a potent and specific inhibitor of TXNIP.

Zymeworks Inc. has received IND clearance from the FDA for ZW-191, the company’s novel folate receptor-α (FRα)-targeted topoisomerase I (TOPO1) inhibitor antibody-drug conjugate (ADC). Clinical development is set to begin this year, with filings to initiate studies outside the U.S. to be made in the second half of this year.

Myogenica Inc., a University of Minnesota startup company, has obtained IND approval from the FDA for Myopaxon, an induced pluripotent stem cell (iPSC)-derived muscle stem cell product to regenerate skeletal muscle. A planned study will evaluate intramuscular injections of Myopaxon in non-ambulatory adult patients with Duchenne muscular dystrophy.