Pipeline

REALIZING THE POTENTIAL OF CELLULAR AND GENE THERAPY

We believe our CELL+GENE™ platform has the potential to reshape cellular medicine. While our authentic cells enable us to replace cells, restore function, and reverse disease, our engineered cells enable us to deliver payloads in ways never before thought possible, enabling therapies to treat previously intractable common and rare diseases.

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Current

Projected
12 Months
Area
Disease
Cell Type
Mechanism
Research
Preclinical
POC
IND-
Enabling
Early
Clinical
Late
Clinical
Neurology
Cardiology
Immunology
Parkinson’s Disease
Neurodegeneration
Demyelinating Disorders
Hirschsprung’s Disease
Heart Failure
Tolerance/Fibrosis
Graft vs Host Disease
Dopaminergic
Neuron
Microglia
Oligodendrocyte
Enteric Neuron
Cardiomyocyte
Macrophages
T-Reg Cells
Authentic Cell
Replacement
Engineered
Authentic Cell
Authentic Cell
Replacement
Authentic Cell
Replacement
Authentic Cell
Replacement
Engineered
Authentic Cell
Engineered
Authentic Cell

Lead Program in Parkinson’s Disease

BlueRock’s founders have pioneered the derivation of dopaminergic neurons that have demonstrated robust preclinical data to restore motor function and increased dopamine release as they mature. Our lead program is an authentic cellular therapy candidate to treat Parkinson’s disease, which we expect to advance into clinical development by the end of 2019, having already established preclinical proof of concept.

Why Parkinson’s Disease?

Parkinson’s Disease is the second most common neurodegenerative disorder, affecting an estimated 1.3 million people in the U.S. and greater than 7.5 million globally. It is a chronic and progressive disease that current treatments do not adequately address. The primary and most debilitating symptom is a progressive loss of motor control.

Using our authentic dopaminergic neurons as therapy, we aim to re-innervate the human brain and reverse degenerative disease, potentially restoring motor function to the millions of patients suffering from Parkinson’s.

CELL+GENE platform enables creation of cellular medicines with broad therapeutic potential