WhiteLab Genomics Presents In Vivo Data for AI-Designed Genomic Medicine Platform, Demonstrating Targeted Brain Delivery After IV Injection
New results suggest a potential path toward non-surgical genomic medicines for neurological diseases.
Boston, MA & Paris - WhiteLab Genomics presented new in vivo data at the ASGCT Annual Meeting, showing that viral vectors designed entirely using its AI platform, ALFRED, achieved approximately 50-fold higher DNA enrichment in the brain compared to AAV9, the current industry-standard gene delivery vector for neurological diseases, following a single intravenous injection in mice, while remaining undetectable in the liver.
The findings were generated in collaboration with Dr. Françoise Piguet’s GENOV lab at the Paris Brain Institute
The results address one of the biggest challenges in genomic medicine - delivering therapies to the brain without invasive surgery. Today, many gene therapies for neurological diseases require direct injection into the brain or spinal fluid because the blood-brain barrier prevents most medicines from reaching the central nervous system (CNS) through the bloodstream. Even AAV9, one of the most widely used delivery vehicles in gene therapy, can lead to significant exposure in organs like the liver, creating potential safety and dosing limitations.
WhiteLab’s data suggest a potential alternative: AI-designed vectors capable of reaching the brain through a standard IV injection while minimizing off-target liver exposure.
Moving Beyond Trial-and-Error Gene Therapy Design
Most gene therapies today are developed through large-scale experimental screening approaches that test hundreds of millions of viral variants with limited understanding of why certain vectors work. WhiteLab Genomics is taking a different approach.
Its proprietary ALFRED platform uses AI to identify the specific cell-surface receptors that control tissue access and then designs vectors around those biological mechanisms. Instead of relying on random screening, the platform rationally engineers vectors based on predicted biological interactions.
This enables:
- Rational design of gene delivery vectors from the start
- Smaller, more efficient experimental libraries
- Faster development with clearer translational rationale
In Vivo Proof-of-Concept Results
In wild-type mice, ALFRED-designed vectors demonstrated:
- ~50x higher DNA enrichment in the brain compared to AAV9
- No detectable liver signal
- Functional targeting following standard intravenous administration
“Crossing the blood-brain barrier without surgery has been one of the biggest unsolved problems in genomic medicine. These results validate both our platform and our belief that AI can fundamentally change how gene therapies are designed, moving the field from brute-force screening toward rational engineering.”
WhiteLab Genomics is currently applying its AI-driven platform across multiple genomic medicine programs with ongoing studies in larger animal models
Gene therapy developers interested in R&D collaboration, licensing, or access to WhiteLab's AI-designed capsids for CNS or other tissue-targeting applications are welcome to contact bd@whitelabgx.com.
About WhiteLab Genomics
WhiteLab Genomics is a TechBio company dedicated to unleashing the full power of genomic medicine with Artificial Intelligence. Founded in 2019 and backed by Y Combinator and the French Tech 2030 program, WhiteLab operates at the intersection of AI, computational biology, and structural biology, with offices in Paris, Boston, and Montréal.
WhiteLab’s proprietary AI platform enables the rational design and optimization of delivery systems including viral and non-viral vectors, as well as RNA and DNA payloads. The company supports partners across a wide range of therapeutic areas, including but not limited to oncology, neurology, ophthalmology, auditory disorders, and rare diseases.
WhiteLab’s mission is to unleash the potential of genomic medicine through advanced artificial intelligence, empowering scientists to design safer, more effective therapies from discovery through preclinical stages.
WhiteLab collaborates with leading companies, including Sanofi, Cytiva and Debiopharm, and benefits from a strong network of top-tier academic partners such as the Brain Institute (ICM), the Vision Institute, the University of Massachusetts, and Généthon.