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BrainXell Therapeutics Unveils Promising Preclinical Data Demonstrating Efficacy of BXT-110, an Autologous iPSC-Derived Neuronal Therapy, in Parkinson's Disease

BrainXell is an industry leading producer of neurons and glia paving the way for advancements in neuroscience. (PRNewsfoto/BrainXell, Inc.)

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BrainXell, Inc.

Nov 03, 2025, 11:03 ET

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SAN DIEGO, Nov. 3, 2025 /PRNewswire/ -- BrainXell Therapeutics today announced the disclosure of its first scientific data, which will be presented at two conferences this month: the Cell Symposia | Gene and Cell-Based Therapies: Progress in Clinical Translation and the Society for Neuroscience (SfN) Annual Meeting. These presentations mark a significant milestone as the company advances its mission to develop regenerative cell therapies for neurodegenerative diseases.

Parkinson's disease (PD) is the second most common neurodegenerative disorder, characterized by the progressive loss of midbrain dopaminergic neurons in the substantia nigra. These neurons are essential for dopamine production and play a central role in regulating motor control, regulating mood, and other cognitive functions. Restoring dopaminergic neurons represents a therapeutic goal, and the use of induced pluripotent stem cells (iPSCs) provides a promising regenerative approach for PD.

"As a company, we are innovators in iPSC technology, and this represents an important advancement for BrainXell Therapeutics," said Katherine Vega Stultz, CEO. "Our data presentations reflect our commitment to rigorous science, innovation and deep expertise in iPSC biology and neuronal differentiation. Importantly, these results highlight our capability to advance development of iPSC- derived therapies that can be transformative for patients living with neurodegenerative diseases. We are excited to be advancing BXT-110 as a potential treatment of Parkinson's disease."

The data presented highlights BrainXell Therapeutics' progress in developing a robust, reproducible, and scalable approach for BXT-110, an autologous source of midbrain dopaminergic progenitor (mDAPs) for PD. Using both patient- and donor-derived iPSC lines, the team generated highly enriched mDAPs with strong consistency across on-target and off-target markers. In vivo studies demonstrated significant graft survival and substantial functional recovery in a 6-OHDA rodent model, with efficacy observed as early as 12 weeks and sustained for up to six months post-transplantation. Collectively, these results for BXT-110 establish an efficacious, highly reproducible personalized cell-therapy candidate, supporting its advancement as a potential disease-modifying treatment for PD.

About BrainXell Therapeutics

BrainXell Therapeutics, a division of BrainXell, Inc., is a San Diego–based biotechnology company pioneering regenerative cell therapies for neurodegenerative diseases. Founded on the research of Dr. Su-Chun Zhang, MD, PhD, Advising CSO, the company's lead program, BXT-110, is advancing toward IND-enabling studies for Parkinson's disease.

For more information, visit www.brainxell.com

This research was made possible by funding from the California Institute for Regenerative Medicine (CIRM), a state of California Agency that funds regenerative medicine, stem cell, and gene therapy research.

SOURCE BrainXell, Inc.

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