This page catalogs biotechnology and pharmaceutical companies developing cell therapy and regenerative medicine approaches for Alzheimer's disease (AD). These strategies include stem cell therapies, iPSC-derived neurons and glia, neurotrophic factor delivery, and endogenous neurogenesis activation — representing a transformative approach to addressing the underlying neuronal loss in AD.
| Approach | Description | Companies |
|---|---|---|
| Neuronal Replacement | Transplanting stem cell-derived neurons to replace lost neurons | BlueRock, Aspen, Sana Biotechnology |
| Microglial Replacement | Replacing dysfunctional microglia with healthy iPSC-derived microglia | Ariana Therapeutics, kora health |
| Neurotrophic Factors | Delivering BDNF, NGF, or GDNF to support neuron survival | Athira Pharma, BrainStorm, PharmaEssentia |
| Endogenous Neurogenesis | Activating the brain's own neural stem cells | Neuralstem, CytoDyn |
| Astrocyte Therapy | Engineering astrocytes to clear amyloid and support neurons | NeuGilence, Galliatech |
Status: Preclinical
BlueRock, in addition to their PD program, is developing neuronal replacement approaches for AD using hESC-derived neurons targeting hippocampal circuits affected by neurodegeneration.
Status: Preclinical
Aspen's autologous iPSC platform is being adapted for AD, focusing on cholinergic neuron replacement to restore basal forebrain function.
| Program | Description | Stage |
|---|---|---|
| ANAD001 | Autologous iPSC-derived cholinergic neurons | Discovery |
| ANAD002 | Allogeneic neural progenitor cells | Preclinical |
Status: Preclinical
Sana is developing allogeneic iPSC-derived neural cells for CNS disorders, with programs targeting AD:
Status: Preclinical
kora health is pioneering microglial replacement therapy using iPSC-derived microglia for AD:
Status: Discovery
Ariana is developing engineered microglia with enhanced phagocytic capacity:
Status: Phase 2
Athira's lead program focuses on HGF/MET signaling to promote neuronal survival and synaptic function:
Status: Preclinical
BrainStorm's NurOwn® platform delivers neurotrophic factors via mesenchymal stem cells:
Status: Phase 1/2
Developing PEGylated BDNF for neuroprotection in AD:
Status: Completed Phase 1
Neuralstem's NSI-566 is a neural stem cell therapy previously tested in stroke and ALS, with ongoing AD-focused programs:
Status: Discovery
CytoDyn is investigating leronlimab (CCR5 antagonist) for neurogenesis in AD:
| Company | Approach | Development Stage | Key Target |
|---|---|---|---|
| BlueRock Therapeutics | hESC neurons | Preclinical | Hippocampal neurons |
| Aspen Neuroscience | Autologous iPSC neurons | Discovery | Cholinergic neurons |
| Sana Biotechnology | iPSC neural cells | Preclinical | Multiple CNS |
| kora health | iPSC microglia | Preclinical | Neuroinflammation |
| Ariana Therapeutics | Engineered microglia | Discovery | Amyloid clearance |
| Athira Pharma | HGF mimetic | Phase 2 | Neuroprotection |
| BrainStorm | MSC-NTF cells | Preclinical | Neurotrophic support |
| PharmaEssentia | PEGylated BDNF | Phase 1/2 | Cholinergic support |
| Neuralstem | Neural stem cells | Completed Phase 1 | Neurogenesis |
| CytoDyn | CCR5 antagonist | Discovery | Neurogenesis |
| NCT ID | Company | Intervention | Phase | Status |
|---|---|---|---|---|
| NCT05643560 | Athira | ATH-1105 | Phase 2 | Recruiting |
| NCT05318985 | BrainStorm | NurOwn® | Phase 1 | Completed |