BPGbio and Icahn School of Medicine at Mount Sinai Publish Landmark Study on the Living Human Brain, Unlocking New Molecular Insights Into Neurodegenerative Disease

New findings challenge the longstanding assumption that postmortem tissue accurately represents the living brain — and can reshape the future of research and drug development for Alzheimer’s, Parkinson’s, and other neurodegenerative diseases

BOSTON and NEW YORK, Oct. 20, 2025 (GLOBE NEWSWIRE) -- BPGbio, Inc., a biology-first, AI-powered, clinical-stage biopharma focused on mitochondrial biology and protein homeostasis, together with the Icahn School of Medicine at Mount Sinai (MSSM), today announced the publication of groundbreaking findings from the Living Brain Project in PLOS ONE titled A study of RNA splicing and protein expression in the living human brain. The study, co-authored by researchers from both institutions, represents the most comprehensive comparison to date of living versus postmortem human brain tissue, analyzing RNA splicing, protein expression, and RNA-protein networks from the prefrontal cortex.

The results demonstrate that the vast majority of molecular features in the human brain differ between living and postmortem samples, with 95% of primary and mature RNA transcripts and over 60% of proteins showing significant differences in expression or processing.

These findings challenge the longstanding assumption that postmortem brain tissue is an accurate molecular representation of the living brain — an assumption behind decades of neuroscience research.

“Neurodegenerative diseases have not seen meaningful therapeutic progress in decades, and findings from this project suggest that we’ve been handicapped by not having access to tissue reflective of brain function,” said Alexander Charney, M.D., Ph.D., Director, The Charles Bronfman Institute for Personalized Medicine and Vice Chair, Windreich Department of Artificial Intelligence and Human Health at Icahn Mount Sinai and co-lead author. “The Living Brain Project is a game changer and represents a breakthrough path forward for the field. By working with BPGbio, whose capabilities in integrating multiomic data and AI are second to none, we are now able to generate actionable insights from living brain tissue that can reshape the future of drug development in Alzheimer’s, Parkinson’s, and other devastating diseases.”

“Partnering with Dr. Charney and team at Mount Sinai on this project is a testimony to the meaning of true collaboration and BPGbio’s research expertise and shared commitment to breaking new ground in medicine,” said Niven R. Narain, Ph.D., President and CEO of BPGbio. “The Living Brain Project paves the way for a new frontier in neuroscience research, and it has the potential to transform how future medicines will be developed. We look forward to collaborating with Mt. Sinai and commercial partners who share our vision to accelerate novel insights to create impact for patients and families.”

“This study underscores why the Living Brain Project is so critical,” said Michael A. Kiebish, Ph.D., VP of Platform and Translational Sciences, BPGbio and co-lead author. “By directly accessing and characterizing living brain tissue at multiple molecular levels, we are not just challenging old assumptions — we are opening entirely new avenues for target discovery and therapeutic innovation. These insights will fuel our biology-first AI platform and accelerate the development of the next generation of treatments for neurodegenerative disease.”

The Living Brain Project was made possible by the development of a safe and scalable method to collect prefrontal cortex biopsies during deep brain stimulation (DBS) procedures. To date, researchers have analyzed more than 500 living brain samples and matched postmortem controls, representing the largest such dataset ever assembled.

Key findings include:

• Widespread expression differences: 74% of primary RNA transcripts, 70% of mature RNA transcripts, and 61% of proteins differ between living and postmortem brain samples.
• Splicing and isoform variation: 73% of transcripts show significant differences in RNA splicing, altering how genes are processed into proteins.
• Distinct RNA-protein networks: Over 2 million RNA-protein correlations were analyzed, revealing markedly different co-expression patterns between living and postmortem tissue.
  
  

The study underscores the potential of the Living Brain Project to fuel new therapeutic discoveries in Alzheimer’s, Parkinson’s, and other neurodegenerative diseases, where actionable human data has been a limiting factor.

About the Living Brain Project

The Living Brain Project (LBP) is a collaborative research initiative led by the Icahn School of Medicine at Mount Sinai. Its mission is to generate, analyze, and openly share the world’s first large-scale molecular datasets from living human brain tissue, with the goal of transforming understanding and treatment of brain disorders.

Media contact: media@bpgbio.com

About BPGbio

BPGbio is a leading biology-first AI-powered clinical stage biopharma focused on mitochondrial biology and protein homeostasis. The company has a deep pipeline of AI-developed therapeutics spanning oncology, rare disease and neurology, including several in late-stage clinical trials. BPGbio’s novel approach is underpinned by NAi, its proprietary Interrogative Biology Platform, protected by over 500 US and international patents; one of the world’s largest clinically annotated non-governmental biobanks with longitudinal samples; and exclusive access to the most powerful supercomputer in the world. With these tools, BPGbio is redefining how patient biology can be modeled using bespoke Bayesian AI specifically designed for solving large-scale biology challenges. Headquartered in greater Boston, the company is at the forefront of a new era in medicine, combining biology, multi-modal data, and AI to transform the way we understand, diagnose, and treat disease. For more information, visit www.bpgbio.com.

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