Arbor Biotechnologies Inc.’s ABO-101 has been awarded orphan drug and rare pediatric disease designations by the FDA for the treatment of primary hyperoxaluria type 1 (PH1).

Ariceum Therapeutics GmbH’s radiopharmaceutical 225Ac-SSO110 ([225Ac]satoreotide tetraxetran) has been awarded orphan drug designation by the FDA for the treatment of small-cell lung cancer (SCLC). [225Ac]Satoreotide is a first-in-class Actinium-labeled somatostatin SST2 receptor antagonist.

Cour Pharmaceuticals Development Co. Inc. has obtained IND clearance from the FDA for CNP-103, a nanoparticle in development to address the underlying autoimmunity of type 1 diabetes. A first-in-human phase Ib/IIa trial will begin later this year and will enroll adults (aged 18-35 years) and pediatric patients (aged 12-17) who have stage III or newly diagnosed (within the last 6 months) type 1 diabetes as well as C-peptide >0.2 ng/mL.

Auron Therapeutics Inc. has received FDA clearance of its IND application for its oral KAT2A/B degrader AUTX-703 in hematological malignancies. A phase I proof-of-concept trial in acute myeloid leukemia (AML) will open enrollment this quarter, supported by a recently completed $27 million series B financing.

HCW Biologics Inc. has received clearance of its IND application from the FDA to initiate a first-in-human phase I trial to evaluate HCW-9302 in patients with moderate to severe alopecia areata.

Seed Therapeutics Inc.’s ST-01156 has been awarded orphan drug designation for the treatment of Ewing sarcoma as well as rare pediatric disease designation by the FDA.

Allogene Therapeutics Inc. has obtained IND clearance from the FDA for a rheumatology basket study of ALLO-329.

Ocugen Inc. has received IND clearance from the FDA for OCU-500, an inhaled mucosal vaccine for COVID-19. The National Institute of Allergy and Infectious Diseases (NIAID) will sponsor and conduct a phase I trial of OCU-500 administered via inhalation into the lungs and intranasally as a spray.

Tikun Therapeutics Inc. has obtained U.S. orphan drug and rare pediatric disease designations for its programs in familial dysautonomia, namely its rAAV2-U1a-hELP1 gene replacement therapy for the treatment of optic neuropathy in familial dysautonomia and BPN-36964 for systemic treatment of familial dysautonomia.

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).