iPSC-derived neural precursor cell (NPC) therapy for Huntington’s disease (HD)—a genetic neurodegenerative disorder caused by a CAG repeat expansion in the HTT gene—has shown strong preclinical evidence of restoring lost neurons and improving behavioral and pathological outcomes, supporting its potential to alleviate disease symptoms.

An iPSC-derived NPC therapy for ischemic stroke—a condition in which cerebral blood flow is blocked, leading to neuronal death and resulting in motor, sensory, and cognitive impairments—demonstrates preclinical promise by promoting tissue regeneration and functional recovery through both cell replacement and supportive mechanisms.

An iPSC-derived NPC therapy for Alzheimer’s disease (AD)—a progressive neurodegenerative disorder marked by memory loss, cognitive decline, and behavioral changes due to amyloid-beta plaques, tau tangles, and widespread neuronal loss—has improved memory and pathology in animal models through both replacement of lost neurons and neuroprotective effects. However, the diffuse and complex pathology of AD poses practical challenges to clinical translation.

Small molecule drug development for Huntington’s disease (HD) targets the underlying cause—a CAG repeat expansion in the HTT gene that produces the toxic mutant huntingtin protein (mHTT)—as well as downstream disease pathways. Compared to larger biologics such as antisense oligonucleotides (ASOs) or gene therapies, small molecules offer the advantage of oral bioavailability and improved blood-brain barrier (BBB) penetration, enabling less invasive delivery strategies.

Small molecule drug development for Alzheimer’s disease (AD) aims to create orally bioavailable compounds that effectively cross the BBB and target the disease’s core features—amyloid-beta (Aβ) plaques, tau tangles, neuroinflammation, and neuronal loss—as well as its symptomatic manifestations such as cognitive impairment. This approach offers a practical alternative to biologics like monoclonal antibodies, which often require intravenous administration.