The field of gene and cell therapies continues to advance, offering potential treatments for a range of diseases. Several companies have recently achieved significant milestones in their clinical development programs.
Ultragenyx Pursues Accelerated Approval for MPSIII Gene Therapy
Ultragenyx is seeking accelerated approval from the FDA for UX111, a gene therapy designed to treat Mucopolysaccharidosis type IIIA (MPSIII). The Biologics License Application (BLA) is supported by data from the Phase 1/2/3 Transpher A clinical trial, which utilized CSF heparan sulfate data to demonstrate efficacy. MPSIII is a rare, inherited metabolic disorder that primarily affects the brain and spinal cord.
Arbor Biotechnologies' ABO-101 Cleared for US Trial in Primary Hyperoxaluria Type 1
Arbor Biotechnologies has received clearance to begin a US clinical trial for ABO-101, a CRISPR-based gene editing therapy targeting Primary Hyperoxaluria Type 1 (PH1). The Phase 1/2 redePHine trial will evaluate ABO-101 in both adult and pediatric patients with PH1. PH1 is a rare genetic disorder that leads to the overproduction of oxalate, which can cause kidney stones and kidney failure.
Allogene Therapeutics' ALLO-329 Advances to Phase 1 for Rheumatologic Diseases
Allogene Therapeutics has announced that the FDA has cleared ALLO-329 for a Phase 1 clinical trial in rheumatology indications. The planned Phase 1 RESOLUTION study will include patients with systemic lupus erythematosus, lupus nephritis, idiopathic inflammatory myopathies, and systemic sclerosis. ALLO-329 is an allogeneic CAR-T cell therapy, representing a novel approach to treating these autoimmune diseases. Cell therapy, particularly CAR-T, is expanding into autoimmune diseases like lupus and multiple sclerosis. While promising, safety, efficacy, and broad applicability remain uncertain.
uniQure Progresses with SOD1-ALS Gene Therapy Trial
uniQure is making progress in its clinical trial for AMT-162, a gene therapy targeting superoxide dismutase 1 (SOD1)-related Amyotrophic Lateral Sclerosis (ALS). The company received a recommendation from the study's Independent Data Monitoring Committee (IDMC) to proceed with enrollment for the trial's second cohort. AMT-162 aims to reduce the production of the mutant SOD1 protein, which is a key driver of this form of ALS.
