After 3 to 5 years of L-DOPA treatment, patients with Parkinson’s disease often experience significant symptom worsening, which continues to progress over time. There are currently no approved therapies that can slow the degeneration of dopaminergic neurons, nor are there any approved treatments capable of regenerating dopaminergic neurons.

Despite the availability of statins, PCSK9 monoclonal antibodies, and PCSK9 siRNA therapies, 20% to 60% of patients with hyperlipidemia still fail to achieve target LDL-C levels after treatment. Both PCSK9 antibodies and PCSK9 siRNA therapies exhibit fluctuations in efficacy within dosing intervals. Moreover, small molecules, antibodies, and siRNA therapies all face challenges with patient adherence. In real-world settings, approximately 75% of patients with atherosclerotic cardiovascular disease (ASCVD) and 97% of patients with heterozygous familial hypercholesterolemia (HeFH) do not achieve their LDL-C targets.

Current standard treatments for hepatitis B virus (HBV) infection have limited efficacy, requiring patients to remain on lifelong therapy. Achieving a functional cure for HBV can significantly reduce the risk of disease progression and hepatocellular carcinoma, and potentially free patients from the burden of lifelong treatment. However, the only available therapy today offers a functional cure rate of just 3–7%.

We are driven by our mission to develop one-time, curative genetic medicines for patients living with chronic diseases.