This segment focuses on the development of enzyme-based therapeutics for rare metabolic diseases, addressing conditions with limited or no existing treatment options. Aeglea's lead product candidate, pegzilarginase, is the primary focus, targeting Arginase 1 Deficiency (ARG1-D). Research and development efforts are directed towards understanding the underlying mechanisms of these diseases and designing targeted enzyme therapies to restore metabolic balance. Clinical trials are conducted to evaluate the safety and efficacy of these therapies, with the goal of improving patient outcomes and quality of life. The company's approach involves engineering next-generation human enzymes to provide targeted treatment options for patients with significant unmet medical needs. Future opportunities include expanding the pipeline to address other rare metabolic disorders and exploring novel enzyme engineering techniques.

Aeglea's Homocystinuria program is centered around the development of AGLE-177, a polyethylene glycol (PEG) modified human enzyme designed to address the metabolic imbalances associated with homocystinuria. This program involves preclinical research to optimize the enzyme's activity and stability, followed by Phase 1/2 clinical trials to assess its safety and efficacy in patients with homocystinuria. The therapeutic approach aims to reduce homocysteine levels in the blood, mitigating the risk of cardiovascular complications and other health issues associated with the disease. The program leverages advanced protein engineering techniques to enhance the enzyme's pharmacokinetic properties and improve its therapeutic potential. Future development plans include advancing AGLE-177 through clinical trials and seeking regulatory approval to bring this novel therapy to patients in need.

The Cystinuria Program is focused on the development of AGLE-325, a preclinical candidate designed to address the underlying causes of cystinuria. This program involves extensive research and development efforts to understand the pathophysiology of cystinuria and identify novel therapeutic targets. The goal is to develop a targeted therapy that can reduce cystine levels in the urine, preventing the formation of kidney stones and reducing the risk of kidney damage. The program leverages advanced drug discovery techniques and preclinical studies to evaluate the safety and efficacy of AGLE-325. Future development plans include advancing AGLE-325 through preclinical studies and initiating clinical trials to assess its potential as a new treatment option for patients with cystinuria.