UMass Chan Medical School and WhiteLab Genomics Partner to use AI to Optimize Genomic Medicines for Neurological Diseases
Worcester and Cambridge, Mass. – The Department of Genetic & Cellular Medicine at UMass Chan Medical School and WhiteLab Genomics, a techbio company specializing in AI for genomic medicine research and development, announced a strategic collaboration to develop in silico AAV payload design aimed at fine-tuning expression of therapeutic genes for neurological diseases exhibiting mitochondrial dysfunctions. This collaboration builds on research in developing gene therapies for these diseases led by Qinglan Ling 凌清岚, PhD, assistant professor of genetic & cellular medicine at UMass Chan.
Harnessing WhiteLab’s AI platform ALFRED, the partnership will utilize Synthetic Promoter Design to precisely modulate AAV payload expression in neurological diseases with mitochondrial dysfunctions.
Dr. Ling’s lab, in the Department of Genetic & Cellular Medicine, led by Guangping Gao, PhD, the Penelope Booth Rockwell Professor in Biomedical Research, leverages cutting-edge genetic approaches to understand neurological disorders with mitochondrial dysfunction, developing translational gene therapies to directly benefit patients. By combining cutting-edge gene delivery platforms with advanced computational approaches, the lab develops precise and effective genomic medicines for conditions such as Leigh syndrome, a severe primary mitochondrial disease, and neurofibromatosis type 2 (NF2-related) schwannomatosis. With expertise in nuclear and mitochondrial DNA–related disorders, Ling and colleagues integrate experimental and bioinformatics-driven strategies to overcome current barriers in genomic medicine, advancing the field toward safe, personalized, and translatable solutions for patients with devastating neurological conditions.
WhiteLab Genomics will use its proprietary platform ALFRED (AI-Led Framework for Rational Exploration in Drug Design) to analyze proprietary multi-omics data and accurately identify cell type and state-specific genes and regulatory sequences. The platform will subsequently use its proprietary AI modules to identify and rank conserved cis-regulatory sequences responsive to mitochondrial stress, permitting the generation of a Synthetic Promoter Library for driving transgene expression in disease cells.
About the Horae Gene Therapy Center
The interdisciplinary faculty at the Horae Gene Therapy Center is dedicated to developing therapeutic approaches for rare inherited disease for which there is no cure. The team utilizes state of the art technologies to either edit mutated genes that produce disease-causing proteins or introduce a healthy copy of a gene if the mutation results in a nonfunctional protein. MDs and PhDs work together to address the medical needs of rare diseases such as Canavan disease, Tay-Sachs and Sandhoff diseases, retinitis pigmentosa, cystic fibrosis, Huntington's disease and ALS. More common diseases such as cardiac arrhythmia and hypercholesterolemia are also investigated with the aim of treating a wide spectrum of diseases by various gene therapeutic approaches. As faculty of UMass Chan Medical School, center investigators participate in numerous ongoing clinical trials.
About UMass Chan Medical School
UMass Chan Medical School, one of five campuses of the University of Massachusetts system, comprises the T.H. Chan School of Medicine, the Morningside Graduate School of Biomedical Sciences, the Tan Chingfen Graduate School of Nursing, ForHealth Consulting at UMass Chan Medical School, MassBiologics, and a thriving Nobel-Prize-winning biomedical research enterprise. UMass Chan is advancing together to improve the health and wellness of our diverse communities throughout Massachusetts and across the world by leading and innovating in education, research, health care delivery and public service. It is ranked among the best medical schools in the nation for primary care education and biomedical research by U.S. News & World Report. Learn more at www.umassmed.edu.
About WhiteLab Genomics
WhiteLab Genomics, a pioneer in the accelerated development of genomic medicines, sits at the intersection of artificial intelligence and biology. Founded in 2019 and supported by Y-Combinator, the company is establishing itself as a key player in genomic medicine innovation. Its proprietary AI technology enables the analysis of complex biological data and the in silico design of optimized therapeutic candidates, thus reducing development time, costs, and associated risks. WhiteLab collaborates with major pharmaceutical companies (such as Sanofi), leading academic institutions, and innovative biotech companies. WhiteLab is a member of the prestigious French Tech 2030 program and recently joined Bayer Co.Lab in Cambridge (USA) as well as the Technology Network of Ginkgo Bioworks. Learn more at whitelabgx.com.
The Collaboration
“This collaboration exemplifies the power of AI and computational biology in designing precise genetic payloads tailored to disease-specific cellular contexts. We’re eager to demonstrate the precision of our Synthetic Promoter Design strategy to accelerate the development of genomic medicines.”
“The idea of this project started when we identified cytotoxicities with the first version of a gene therapy for SURF1 Leigh syndrome. It took us three more years to try different promoters and determine a gene therapy that is both safe and effective,we are excited to collaborate with WhiteLab to accelerate the development of safer and more effective gene therapies for more mitochondrial diseases as well as complex neurological and metabolic conditions.”
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