AD Cell Therapy and Regenerative Medicine Companies

Alzheimer's Disease Cell Therapy and Regenerative Medicine Companies

Overview

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.

Key Approaches

Alzheimer's Disease Cell Therapy and Regenerative Medicine Companies

Overview

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.

Key Approaches

| 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 |

Neuronal Replacement Therapies

BlueRock Therapeutics

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.

• Cell Source: hESC-derived neural progenitor cells
• Target: Hippocampal CA1 neurons
• Mechanism: Replace degenerated neurons, restore synaptic circuitry

Aspen Neuroscience

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 |

[Aspen Neuroscience](/companies/aspen-neuroscience)

Sana Biotechnology

Status: Preclinical

Sana is developing allogeneic iPSC-derived neural cells for CNS disorders, with programs targeting AD:

• Platform: Immuno-engineered iPSC derivatives
• Advantage: Reduced immunosuppression requirements

Microglial Replacement Therapies

kora health

Status: Preclinical

kora health is pioneering microglial replacement therapy using iPSC-derived microglia for AD:

• Rationale: Microglia are key drivers of neuroinflammation in AD
• Approach: Replace dysfunctional endogenous microglia with healthy iPSC-derived microglia
• Target: Reduce amyloid plaques, restore synaptic function

Ariana Therapeutics

Status: Discovery

Ariana is developing engineered microglia with enhanced phagocytic capacity:

• Enhancement: Boosted amyloid clearance
• Modified Functions: Reduced inflammatory cytokine release

Neurotrophic Factor Therapies

Athira Pharma

Status: Phase 2

Athira's lead program focuses on HGF/MET signaling to promote neuronal survival and synaptic function:

• Drug: ATH-1105 (small molecule HGF mimetic)
• Target: MET receptor on neurons
• Mechanism: Neuroprotection, enhanced neurogenesis

BrainStorm Cell Therapeutics

Status: Preclinical

BrainStorm's NurOwn® platform delivers neurotrophic factors via mesenchymal stem cells:

• Approach: MSC-NTF cells secrete BDNF, GDNF, NGF
• Delivery: Intrathecal administration

PharmaEssentia

Status: Phase 1/2

Developing PEGylated BDNF for neuroprotection in AD:

• Mechanism: Support cholinergic neuron survival
• Delivery: Peripheral administration with CNS penetration

Endogenous Neurogenesis Approaches

Neuralstem

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:

• Approach: Transplanted neural stem cells to secrete neurotrophic factors
• Target: Stimulate endogenous neurogenesis

CytoDyn

Status: Discovery

CytoDyn is investigating leronlimab (CCR5 antagonist) for neurogenesis in AD:

• Mechanism: CCR5 modulation affects neural stem cell migration
• Target: Enhance hippocampal neurogenesis

Company Summary Table

| 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 |

Clinical Trials in Cell Therapy for AD

| NCT ID | Company | Intervention | Phase | Status |
|--------|---------|--------------|-------|--------|
| NCT05643560 | Athira | ATH-1105 | Phase 2 | Recruiting |
| NCT05318985 | BrainStorm | NurOwn® | Phase 1 | Completed |

See Also

• [Parkinson's Disease Neurogenesis Companies](/companies/pd-neurogenesis-companies)
• [Alzheimer's Neurotechnology and Neuromodulation](/companies/alzheimers-neurotechnology-neuromodulation)
• [Neurogenesis Pathway](/mechanisms/neurogenesis-ad)
• [Cell Therapy for Neurodegeneration](/mechanisms/cell-therapy-neurodegeneration)

References