Aptah is a biotech startup focused on novel RNA therapies initially targeting cancer and neurodegenerative diseases.Read more
Using our ASMO platform, we designed a molecule able to modulate pathogenic snRNPs, which arrest proteins that regulate autophagy, organelles transport, cell proliferation, growth and migration.Read more
Our first drug for neurodegenerative diseases is targeting snRNPs related to AD. Aggregation, over activation and mislocalization of snRNPs could contribute to neurodegeneration by altering patterns of protein synthesis, post translational modifications and arresting proteins that regulate autophagy, organelles transport (neurotransmitters) and synapses.Read more
Chairman of the Board
Scientific Advisory Board
Scientific Advisory Board
Co-founder and CEO
Co-Founder & CSO
Head of Preclinical Studies
Head of Bioinformatics
Head of Corporate Development
Co-Founder & Angel Investor
Aptah is a biotech startup focused on novel RNA therapies initially targeting cancer and neurodegenerative diseases.
By understanding the mechanisms underlying sporadic neurodegenerative and oncology diseases, we developed and patented a new technology platform called ASMO - Aptah Spliceosome Modulation Oligonucleotide - which is the backbone to produce innumerous synthetic molecules, rationally designed to modulate (not silence), in an unique way, specific proteins called snRNPs - small ribonucleoproteins (or RNA binding proteins), key elements responsible for protein aggregation, protein synthesis and many other physiological functions, thus, paving the way for the treatment of thousands of genetic disorders.
Our research focuses on the central concept of regulated protein-protein interaction and aggregation. Protein aggregation in neurodegenerative diseases and oncology is thought to occur as an unwanted byproduct of protein misfolding. Recent studies increasingly highlight the importance of RNA binding proteins and protein aggregation as part of a normal regulated, physiological mechanism controlling protein synthesis as well as many fundamental physiological functions, such as stress granules, transport granules and P bodies.
Our hypothesis is that dysfunction of snRNPs causes neurodegenerative diseases and cancer. Aggregation, over activation and mislocalization of snRNPs could contribute to neurodegeneration and cancer by altering patterns of protein synthesis, post translational modifications and arresting proteins that regulate autophagy, organelles transport, cell proliferation, growth and migration.
The ASMO platform comprises a new class of molecules capable of interacting in an innovative and versatile way with both multiple proteins and the transcript (pre-mRNA) at the same time, which allows the development of a completely new modality of therapies for the treatment of genetic disorders currently untreated, such as several oncological and neurodegenerative diseases.
ASMOs are not limited to the capabilities of the antisense oligos currently used to correct aberrations arising from specific mutations, but encompass a much broader capacity to address epigenetic dysfunctions, such as protein-protein interaction, unrelated to a specific base mutation, but to an epigenetic effect. Among its capabilities, the ASMO platform can meet customized goals such as preventing abnormal splicing linked to a specific mutation, preventing abnormal splicing linked to a dysfunction of splicing components common to all genes, or controlling expression levels of target transcript for different types of diseases, modulating this transcript(s) without silencing or inhibiting it, which makes the treatment much safer.
In competing technologies, such as those involving the use of antisense oligonucleotides (ASO) or RNA interference, the main focus is on inhibition, silencing or degradation methods of a specific site at the pre-mRNA or mRNA of a specific gene. Such approaches make sense for genetic diseases associated with mutations at specific sites, such as in intronic or exonic regions, allowing, for example, the silencing or degradation of isoforms of transcripts containing the mutation. However, such an approach is not suitable for diseases that do not involve single mutations, or for those which involve generalized dysfunctions (such as abnormal snRNPs aggregates), which can be strategically addressed by our technological platform.
Using our ASMO platform, we designed a molecule able to modulate pathogenic snRNPs, which arrest proteins that regulate autophagy, organelles transport, cell proliferation, growth and migration.
It`s mechanism of action is to modulate specific snRNPs which may aggregate in the cytosol and ignite abnormal cell cycle reentry, leading to over-proliferation of cells (cancer). Also, the same pathological mechanism is related to the autophagolysosomal dysfunction and other pathological mechanisms.
Besides the cell cycle reentry control mechanism, by modulating TAU protein, the molecule could be used as a microtubule stabilizer in order to address Taxanes-resistant cancers. Other indications include Breast Cancer, Prostate Cancer, Kidneys Cancer, Neuroblastoma and Glioblastoma.
Preliminary tests showed cytostatic behavior on cancer cells after 144 hours exposure to Aptah`s compound. Our findings indicate potential therapeutic approaches to avoid cell over proliferation specifically on cancer cells, with very low /no cytotoxicity.
Our first drug for neurodegenerative diseases is targeting snRNPs related to AD. Aggregation, over activation and mislocalization of snRNPs could contribute to neurodegeneration by altering patterns of protein synthesis, post translational modifications and arresting proteins that regulate autophagy, organelles transport (neurotransmitters) and synapses.
Preliminary results show significant increase of post-synaptic protein (PSD95), axonal transport and better morphology in both neurons derived from healthy and AD donors (iPSC - human neurons). RNA Seq confirms our mechanism of action and show that the molecule effectively changes the pattern of disease to a pattern similar to that of healthy neurons.
The compound is not neurotoxic, and also showed safe concentrations window for other main brain cell lines such as microglia and astrocytes. Our findings indicate that our molecule can be used for neuroprotection, a therapeutic approach to prevent the progression of the disease.
Senior positions at different pharmaceutical companies. During his 30-year career, he has held various responsibilities in sales, operations and strategy in various therapeutic areas.
These positions included heading business operations in markets in the United States, Asia-Pacific, Europe, the Middle East, Africa, and Latin America. He earned an M.S. in pharmacology and toxicology from Long Island University, New York, and a B.A. in biology from Concordia College, New York.
Luc Buée is a French scientist (Directeur de Recherche au CNRS - DRCE CNRS) Director of the Lille Neuroscience & Cognition Research Centre and Head of the Inserm laboratory "Alzheimer & Tauopathies" at the University of Lille, France. He is also the representative researcher elected by academia colleagues at the French Foundation Plan Alzheimer. He is the organizer of the Eurotau meetings.
Research focus: Alzheimer research, TAU and drug discovery.
He has received different awards:
Michael Raffi is Professor of Neurology at the Keck School of Medicine and Medical Director of the Alzheimer's Therapeutic Research Institute (ATRI). He received his MD and PhD degrees from Brown University and conducted neurogenetics research at Harvard Medical School. He completed his Neurology residency at the Johns Hopkins Hospital and fellowship in Neurodegenerative diseases at the University of California, San Diego. Dr. Raffi is a physician-scientist whose research focuses on developing new treatments for Alzheimer's disease (AD) including genetic form of AD that occurs in people with Down Syndrome (DS). He has led multi-center clinical trials for AD in DS as well as the first multimodal AD biomarker study in DS, the Down Syndrome Biomarker Initiative (DSBI). He is Principal Investigator of the NIH-funded Alzheimer's Clinical Trials Consortium - Down Syndrome (ACTC-DS), an international network of academic researchers with expertise in AD and DS. He serves as a scientific reviewer for the NH and Alzheimer's Association.
Co-founder of Aptah Bio, Mr. Bottos is graduated in Engineering at UFSC (BR) and is a Alumni student at Harvard Business School Endeavor Program. Former researcher at the Fraunhofer Institute in Germany. MIT Advisor in Brazil, Endeavor Global Member selected out of 3,258 candidates worldwide, Award Winning “Entrepreneur of the Year” - Brazil 2014 (Exame PME), founder of several startups, including Vesper Ventures in 2018, a company builder and the first venture capital fund 100% focused in Biotech in Brazil. Has exceptional skills and internationally recognized success in the field of business, specifically in the engineering technology industry, venture capital and biotech, having held executive and senior roles in highly successful startups. Mr. Bottos combines his advanced knowledge of entrepreneurial strategy with a fundamental understanding of engineering technology, allowing him to repeatedly succeed in applying cutting-edge technology to different industries, from manufacturing to biotech. Works as CEO of Aptah Bio Inc focused on strategically growing and expanding the company.
Founder of Aptah BioSciences, Caio Leal graduated in Biomedicine - Biological Sciences - and a postgraduate degree in Genetics PUC/GO - BR, with extensive experience in the area of biotechnology and emphasis on oligonucleotides, polynucleotides and Computed Aided Drug Design (CADD) associated with methods of computational biology and genetic engineering.
He worked for several years as director of the largest genetic support laboratory in the state of Goiás (BR), providing molecular and genetic diagnostic tests for large laboratories across the country. Also worked for many years as scientist adviser and consultant of pharmaceutical industries. Caio works as Chief Scientific Officer at Aptah Bio.
Pharmacist, M.Sc., and PhD in Pharmacology, postdoctoral in Technology Innovation. She has large experience as research scientist director conducting in vitro toxicology, ADME, proof of concept (efficacy), including in cosmetics and alternative methods to animal use. Broad-based background in pharmacological research, drug development and innovation. More than 10 years of experience with cell cultivation and 6 years in Contract Research Organization (CRO) with GLP environment. Experience with in vitro and in vivo physiology and pharmacology assays; animal models of diseases, molecular and cellular biology techniques and immunoassays, cell culture and drug safety evaluation. Experience in designing protocols and writing reports, and regulatory dossier. Supervision of graduate and undergraduate students and postdoctoral researchers. Speaker in many meetings and conferences. Emphasis on nonclinical development (safety and proof of concept) of small molecules and biological product, in several areas such as neurodegenerative diseases, cancer, inflammatory process, among others. Execution and/or coordination of more than 50 preclinical projects and development of new molecules. Camila works as Head of Preclinical Studies at Aptah Bio.
PhD in Computational and Systems Biology from Fundação Oswaldo Cruz (2017) and Master in Cellular and Molecular Biology also from Fundação Oswaldo Cruz (2013). She worked as a volunteer at the Functional Genomics and Bioinformatics Laboratory at Fundação Oswaldo Cruz. She was a research consultant in Bioinformatics in the Biotechnology coordination of the SENAI Institute for Innovation in Biosynthetics and Fibers - SENAI CETIQT, where she worked in the analysis of new generation sequencing (NGS) data generated on the sequencing platform of the SENAI Institute for Innovation in Biosynthetics and Fibers, focusing on genomics and metagenomics of microorganisms. She is currently the Head of Bioinformatics at Aptah Bio and has extensive experience in the field of structural bioinformatics, molecular protein modeling, computer-aided drug design, computational approaches to metabolic pathway analysis, and next-generation sequencing data analysis (NGS expertise) in the development of pipelines for automating bioinformatics analyzes in Python and Bash (shell script) languages.
Business manager with a legal background. Extensive international experience completing transactions and international business structures in Latin America, Asia and Europe. Developed corporate wide environmental compliance and business principles programs in conjunction with integrated management system committed to sustainability principles. Spanish and Portuguese languages. Specialties: International, mergers and acquisitions and business structures and transactions. Works as legal adviser at Aptah Bio.
Jonas Sister has a BS in Economics from the University of Sao Paulo, with specialization in International Management from Hogeschool Haarlem, in the Netherlands, and an MBA from Insper, in Brazil. He is a partner at Vesper Ventures, a biotech VC firm, early investor at Aptah Bio. Prior to joining Vesper, he was a private equity partner at BRZ Investimentos, where he served for ten years throughout the PE investment cycle, serving on the Board of Directors and other governance bodies of companies in the consumer goods, agribusiness and timberland sectors. His previous experience also includes seven years at Votorantim, a leading building materials group, in the areas of strategic planning and mergers and acquisitions, as well as direct investments in startups with proprietary capital in sectors such as biotech, SaaS, fintech and telecom. At Aptah, he participates in strategic planning, corporate structuring and business development initiatives.
Graduated in production engineering at the University of Santa Catarina and is a Alumni student at Harvard Business School Endeavor Program. Former researcher at the Fraunhofer Institute in Germany. Gabriel founded and worked for more than 12 years as CTO, CEO and Chairman of Welle Tecnologia Laser SA (the largest laser machine manufacturer in South America). In 2018, Gabriel co-founded Vesper Ventures SA and companies such as Exact Sales and GnTech.
Graduated in Medicine University in Rio de Janeiro (2004), has medical residency in radiology and diagnostic imaging, MBA in strategic management and of business by Fundação Getúlio Vargas (2018) and with extensive experience in molecular biology and genetics.
Alternative Investments Fund Manager with experience on venture capital operations and international funds structuring. Graduated in business management at the Federal University of the State of Santa Catarina (USFC) and MBA in Financial Markets at IPOG. Certified fund manager by ANBIMA (CGA) and licensed Fund Manager by CVM (Brazilian regulatory agency). He works as CFO at Vesper Ventures and as financial controller of investees, such as Aptah Bio.