Herophilus, Inc.

SAN FRANCISCO--(BUSINESS WIRE)--Herophilus, a leading biotechnology company with the mission to discover neurotherapeutics to cure complex brain diseases, today announced that Genentech, a member of the Roche Group, has acquired its proprietary high-throughput human organoid-based drug discovery platform comprising the Orchard™, Orchestra™, and OrCA™ technologies. Herophilus will continue to advance its own ongoing neurotherapeutic research programs. This transaction provides the Genentech and Roche R&D units with these three components of Herophilus’ discovery platform: Orchestra: Comprehensive hybrid-cloud enterprise-scale software system for instrument and automation control, experiment tracking, provenant data and metadata management, high-performance microscopy imaging and video data visualization and exploration, data annotation and rapid machine learning pipeline development OrCA: Modular Organoid Culture and Assay scaled experimental system, comprising five function-specific workcells and a library of optimized biology protocols for low-variability, tuned organoid culture and high-content, high-throughput phenotypic assays Orchard: Protocol library for high-quality reprogramming and genetic engineering of organoid-optimized stem cell lines and access to a diversified neurological disease patient stem cell biobank About Herophilus Herophilus is a San Francisco-based neurotherapeutics company focused on curing complex brain diseases. The company combines brain organoid science, systems neuroscience, robotic automation, and advanced machine learning techniques to scale the ability to discover novel drugs for complex neurological and psychiatric diseases. The company has pioneered the use of multi-modal phenotypic screening in complex in vitro models to discover characterizations of diseases never before achievable. These deep phenotypes are exploited to identify novel therapeutic targets and drug treatments for disorders for which traditional discovery techniques have proved least successful, including neurodevelopmental, psychiatric and neurodegenerative diseases. To learn more, visit www.herophilus.com.

SAN FRANCISCO--( BUSINESS WIRE )--Herophilus announced today that in vivo studies underway of its lead candidate HRP-12975 are funded in part by the Rett Syndrome Research Trust (RSRT). HRP-12975 is the first small molecule therapy for Rett Syndrome with the potential to reverse the root cause of the disease, MECP2 deficiency. HRP-12975 is the first novel disease-modifying chemical entity discovered with any brain organoid discovery platform and thus represents an industry first and validation for the approach of human-led in vitro experimental drug discovery. HRP-12975 causes reactivation of the MECP2 gene on the silenced X chromosome in brain cells of Rett patients. This strategy avoids the risk of MECP2 overexpression inherent with many proposed gene therapies. MECP2 overexpression is known to cause MECP2 duplication syndrome, an extremely severe and often fatal neurodevelopmental disease. HRP-12975 was discovered by screening a highly curated chemical library of 12,000 target-annotated compounds for MeCP2 reactivation in neurons and astrocytes using 3D Rett patient organoid models on Herophilus’ discovery platform. With the support of RSRT, Herophilus is now generating efficacy and safety data of HRP-12975 using genetic mouse models of Rett Syndrome. “ We are thrilled to support the next phase of therapeutic development for molecules identified from Herophilus’ novel approach to discovering x-reactivators using patient-derived brain organoids. Reactivation of the silent MECP2 gene is an important therapeutic strategy for us and we are encouraged by Herophilus’ progress ,” said Monica Coenraads, CEO of the Rett Syndrome Research Trust. “ The Rett Syndrome Research Trust has been instrumental in catalyzing therapeutics development for a devastating, currently incurable, neurodevelopmental disease, and we are grateful for the support and recognition of the potential of our program ,” said Saul Kato, PhD, CEO and co-founder of Herophilus. Herophilus has worked with the RSRT since 2018. As a foundation for Herophilus’ Rett Syndrome discovery program, they collaborated on the most comprehensive biobank of inducible pluripotent stem cell lines and organoids derived from Rett patients. About the Rett Syndrome Research Trust RSRT is a nonprofit organization singularly focused on achieving a cure for Rett syndrome and related disorders caused by defects in the MECP2 gene. Since its founding in 2008, RSRT has awarded $64 million, more than any other Rett organization in the world, to leading scientists pursuing targeted research on Rett. RSRT funds and spearheads global scientific and clinical activities advancing the most promising curative approaches. To date, every biopharmaceutical company pursuing a cure for Rett syndrome is doing so because they leveraged discoveries and resources incubated with RSRT funding. About Herophilus Herophilus is a San Francisco-based neurotherapeutics company focused on curing complex brain diseases. The company’s platform combines brain organoid science, systems neuroscience approaches, robotic automation, and advanced machine learning techniques to scale the ability to discover novel drugs for complex neurological and psychiatric diseases. In an industry-first approach, the company employs multi-modal phenotypic screening to discover characterizations of diseases never before achievable. These “deep phenotypes” are exploited to identify novel therapeutic targets and drug treatments for disorders for which current discovery techniques have proved least successful, including neurodevelopmental, psychiatric and neurodegenerative diseases. To learn more, visit www.herophilus.com .

SAN FRANCISCO--(BUSINESS WIRE)-- Herophilus announced today the publication of “Neuroimmune Cortical Organoids Overexpressing C4A Exhibit Multiple Schizophrenia Endophenotypes” in bioRxiv, the preprint server for biology. The findings further demonstrate the emerging role the neuroimmune axis may play in the development of schizophrenia and in providing a platform for schizophrenia drug discovery. The paper reports results from an ongoing research collaboration between Herophilus and Cerevel Therapeutics launched in December of 2021. The starting point for the research was Herophilus’ proprietary NICO™ (neuro-immune cerebral organoid) model which allows the study of neuroimmune interactions between microglia, neurons, and astrocytes in a realistic human tissue-like context. The NICO™ model is a component of the Herophilus Discovery Engine, which powers the use of human-derived, complex in vitro experiments for scaled, AI-driven drug discovery. “Herophilus’ human neuroimmune organoid model helps to illuminate important insights into the complex biology of the human brain and the role microglia play in disease pathology. Our scientific collaboration with Herophilus has been a rewarding journey,” said Philip Iredale, Vice President, Biology, Cerevel Therapeutics. “This collaboration is a great example of the commitment by a leading neuroscience company such as Cerevel to invest in cutting-edge approaches to develop potentially disease-modifying therapies for one of the most difficult and common brain diseases,” said Saul Kato, PhD, CEO of Herophilus. “The C4A NICO™ model built as part of this research collaboration can now potentially be used for the discovery of novel compounds that may prevent or reverse schizophrenia in patients, or for the discovery of biomarkers that may help detect the disease and track its progression before psychiatric symptoms manifest in a patient.” There is a growing body of evidence implicating the neuroimmune system in the origin of schizophrenia and other complex brain diseases. Recent population-based genetics studies showed that higher levels of C4A correlate with increased schizophrenia risk. C4A is a key molecule of the complement pathway that is implicated in microglia-driven synaptic pruning. Thus, overexpression of C4A in the Herophilus NICO™ system was used to study the impact that a schizophrenia risk gene may have on the neuroimmune response. The C4A NICOs were extensively profiled across several readouts of inflammation and neuroimmunological dysfunction. Several endophenotypes, biological markers of a disorder suspected to be part of the causal chain between genetic factors and ultimate patient symptoms, were observed in the C4A neuroimmune organoids. These included: modulation of inflammatory genes, release of secreted cytokines, and increased uptake of synapses by microglia. About Herophilus Herophilus is a San Francisco-based neurotherapeutics company focused on curing complex brain diseases. The company’s platform combines brain organoid science, systems neuroscience approaches, robotic automation, and advanced machine learning techniques to scale the ability to discover novel drugs for complex neurological and psychiatric diseases. In an industry-first approach, the company employs multi-modal phenotypic screening to discover characterizations of diseases never before achievable. These “deep phenotypes” are exploited to identify novel therapeutic targets and drug treatments for disorders for which current discovery techniques have proved least successful, including neurodevelopmental, psychiatric and neurodegenerative diseases. To learn more, visit .