BrainXell Therapeutics | iPSC Cell Therapy for Parkinson's Disease

OUR Mission

Driven by science.
Undaunted by challenges.

At BrainXell Therapeutics, we’re committed to making a meaningful difference in the lives of patients with neurogenerative diseases and neuro injury. Anchored in a deep heritage of world-class science, we are intentional about the choices we make to advance therapeutics that create new possibilities for patients.

Carving a new way forward for serious conditions like PD that have few options is inherently challenging. Our resolve to meet those challenges head-on through purposeful thinking and a focus on the long view have brought us this far – and will take us further.

About Our PD Therapy

About Us

BrainXell Therapeutics translates advanced cellular science into iPSC-derived therapies designed to regenerate the human central nervous system (CNS) after disease or injury. We’re putting the decade of iPSC experience gained at our legacy company, BrainXell, Inc., into our pipeline programs for devastating neurological diseases, including PD.

Our technology is rooted in the expertise and innovation of our Founder, Dr. Su-Chun Zhang, a global pioneer in stem cell neuroscience, who is also founder of BrainXell, Inc.

"When I founded BrainXell, Inc., I aspired to translate my work on iPSC-derived CNS cells into therapeutics for patients. The BrainXell Therapeutics team will move forward with the same intentional decision-making we employed to give it the best potential for long-term success in improving patients’ lives."

– Dr. Su-Chun Zhang

Disease focus

We’re moving on Parkinson’s disease

PD is the second most common neurodegenerative disorder, with a U.S. patient population expected to exceed 1.2 million by 2030. Current treatments only address PD symptoms; there are no FDA-approved disease-modifying therapies that halt or slow the progression of PD.

The motor and other symptoms of PD are caused by the progressive loss of midbrain dopaminergic (dopamine-producing) neurons. Cell therapies designed to replace these lost neurons with new ones could change the course of the disease.

Our Cell Therapy Method

science & technology

Translating a Decade of Innovation
Into Practical Therapies

Our unique and proprietary method for generating BXT-110 from iPSC-derived cells yields a cell therapy of the highest possible quality. Our science has shown us the right approach for producing an efficacious therapy that could offer PD patients the greatest benefit and unmatched disease-modifying potential.

Collecting the patient’s skin cells (fibroblasts)

A small sample of the patient's skin tissue, about the size of a pencil eraser, is collected during a minor biopsy, typically from the upper arm.

Fibroblasts are isolated from the sample and banked until ready for reprogramming.

Reprogramming with mRNA

We use mRNA – not viral vectors or plasmid DNA – to reprogram the patient’s fibroblasts into iPSCs.

mRNA is an advanced and safe way to reprogram cells, because it leaves no residual DNA behind and doesn’t integrate into the cell’s genome.

Unique spheroid formulation for delivery

We culture the progenitor cells in 3D suspension to produce spheroids (compact aggregates of cells).

Just as humans need good friends for good mental health, spheroids give progenitors the “friendly” cell-to-cell interactions they need to survive. Our formulation process consistently gives us high viability of cells for transplant.

Transplanting the spheroids to the patient’s brain

Our spheroids are never frozen; we deliver them fresh for transplant to the patient. We don’t lose cells to freeze/thaw.

With greater viability, more cells can become dopaminergic neurons, and with faster engraftment than single-cell formulations. Our progenitor cells could be the best for rebuilding the neuronal networks lost to PD – giving patients the disease-modifying therapy they need to reclaim their lives.

science & technology

Translating a Decade of Innovation
Into Practical Therapies

Collecting the patient’s skin cells (fibroblasts)

A small sample of the patient's skin tissue, about the size of a pencil eraser, is collected during a minor biopsy, typically from the upper arm.

Fibroblasts are isolated from the sample and banked until ready for reprogramming.

Reprogramming with mRNA

We use mRNA – not viral vectors or plasmid DNA – to reprogram the patient’s fibroblasts into iPSCs.

mRNA is an advanced and safe way to reprogram cells, because it leaves no residual DNA behind and doesn’t integrate into the cell’s genome.

Unique spheroid formulation for delivery

We culture the progenitor cells in 3D suspension to produce spheroids (compact aggregates of cells).

Transplanting the spheroids to the patient’s brain

Our spheroids are never frozen; we deliver them fresh for transplant to the patient. We don’t lose cells to freeze/thaw.

pipeline

BXT-110:
Disease-modifying potential

BXT-110 is an autologous, iPSC-derived midbrain dopaminergic progenitor (mDAP) cell therapy that BrainXell Therapeutics is developing for moderate-to-severe PD.

BXT-110 is produced by reprogramming a patient’s skin cells into mDAP cells. These cells are then transplanted into the patient’s brain, where we expect them to develop into new, functional dopaminergic neurons.

BrainXell Therapeutics is also developing iPSC-derived spinal interneuron progenitors to treat subacute and chronic spinal cord injury (SCI).

← Swipe to explore pipeline →

Program

Description

Potential
Indications

Discovery

Lead
Optimization

IND
enabling

Phase 1

Phase 2

Phase 3

Rights