Science

Aptah`s molecule is the first and only compound that assures accurate RNA processing of multiple proteins. It works as a template for overall RNA quality control, altering the fate of diseased cells with no harmful effect on the healthy ones.

The great majority of diseases, including most age-related diseases, are NOT related to genetic mutations but rather to how the DNA is processed to produce all our proteins, the so-called RNA processing.

At Aptah Bio, we developed a novel molecule targeting a fundamental element in RNA processing that regulates the production of all our proteins. If this specific element fails to work properly, it leads to the production of multiple toxic proteins, a common event in age-related diseases, such as dementia and many kinds of cancer.

Aptah`s first molecule is the only compound that assures genome-wide RNA processing quality control, correcting multiple mRNAs and toxic proteins using one single compound. It works as a template, adjusting the transcription and splicing machinery and thus, altering the fate of diseased cells with no harmful effect on the healthy ones.

Oncology

Using our unique platform, we designed a molecule able to modulate transcription and cell metabolism. In sporadic cancers, truncated mRNAs and toxic proteins arrest autophagy, organelles transport, cell proliferation, growth, and migration.

Aptah`s compound is focused on controlling the transcription and splicing events that lead to cell abnormal metabolism and cell cycle. By modulating the nucleocytoplasmic transport, we are able to reduce the aggregation of nuclear content in the cytosol, which ignites abnormal cell cycle reentry, leading to over-proliferation of cells (cancer). Also, the same pathological mechanism is related to autophagolysosomal dysfunction and other pathological mechanisms.

In vitro and in vivo experiments indicate Aptah`s compound is able to significantly reduce the overproliferation of tumor cells, with a unique mechanism of action. Also, the compound was able to control the brain inflammation process, increase astrocyte plasticity, and significantly change important pathways such GFAP, AKT, and TAU (among others), exceeding the gold-standard treatment for reducing the tumor growth rate in different animals using IV route with no morbidity changes. 

Neurodegenerative Diseases

Our compound is targeting a key element in the transcription and splicing process directly linked to many sporadic neurodegenerative diseases. Our compound is able to correct multiple transcripts and avoid the synthesis and aggregation of multiple toxic proteins using the same compound.

RNA Seq (iPSC human neurons) confirms our mechanism of action by modifying multiple transcripts, and correcting premature polyadenylation and multiple abnormal proteins, not by silencing, but rather by correcting multiple pre-mRNA / mRNA. Physiology proteins are not changed, and toxic proteins are reduced significantly, including insoluble pTAU and AB fibrils. Results show a significant increase in electrophysiology signals in human neurons derived from Alzheimer`s disease patients (iPSC) after 65 days with no significant effect on healthy neurons.

The compound showed safe concentrations window for the major brain cell lines such as neurons, microglia, and astrocytes. In vivo experiments showed compound is able to increase astrocytes' plasticity and significantly reduce toxic proteins such as AB fibrils (Cortex) and insoluble pTAU (Hippocampus), among others. Interestingly, soluble TAU and physiological AB (healthy form) did not change showing strong evidence of the correct mechanism of action.

Our findings indicate that our molecule can be used for multiple neurodegenerative indications, such as Alzheimer`s, FTD, ALS, PSP, LATE, TBI, etc.

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.

• Former President & CEO Bayer Pharma AG
• Former President, Pfizer - Primary Care Canada/Asia
• Former President, Bristol Myers Squibb - Intercontinental Division

Professor at Harvard & MIT, Director of PersonalGenomes.org, which provides the world's only open-access information on human Genomic, Environmental & Trait data (GET).

His 1984 Harvard PhD included the first methods for direct genome sequencing, molecular multiplexing & barcoding. These led to the first genome sequence (pathogen, Helicobacter pylori) in 1994 . His innovations have contributed to nearly all "next generation" DNA sequencing methods and companies (CGI-BGI, Life, Illumina, Nanopore). 

This plus his lab's work on chip-DNA-synthesis, gene editing and stem cell engineering resulted in founding additional application-based companies spanning fields of medical diagnostics ( Knome/PierianDx, Alacris, Nebula Genomics, Veritas Genetics ) & synthetic biology / therapeutics (AbVitro/Juno, Gen9/Ginkgo, Editas, Egenesis, enEvolv, WarpDrive ). He has also pioneered new privacy, biosafety, ELSI, environmental & biosecurity policies. He is director of an IARPA BRAIN Project and NIH Center for Excellence in Genomic Science. His honors include election to NAS & NAE & Franklin Bower Laureate for Achievement in Science. 

He has coauthored 460 papers, 105 patent publications & one book (Regenesis).

Specialties: Biotechnology-development, sequencing, genomics, synthetic biology

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:

• Prix Claude Pompidou for Alzheimer's disease research (2013)
• Grand Prix Spécial des Sciences par la societé des Sciences de l'Agriculture et des Arts de Lille (2014)
• Prix Bernadette at Pierre DUBAN 2020 #FRM for Alzheimer's and related diseases.

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.

Titles:

• Medical Director - Alzheimer's Therapeutic Research Institute;
• Professor of Neurology at USC Keck School of Medicine
• Principal Investigator of the NIH-funded Alzheimer's Clinical Trials Consortium - Down Syndrome (ACTC-DS)

Ophthalmologist specializing in Retina and Vitreous.

She graduated as a doctor from the Federal University of Paraná, Brazil, where she obtained the NILO CAIRO EXTRAORDINARY PROFESSOR AWARD, as a Cum Laude outstanding student with the highest qualification.

She completed her training as an ophthalmologist at the Federal University of São Paulo (Unifesp), with subsequent subspecialization in Retina and Vitreous.

Master in Macular, Vitreous and Retina Surgery from the Autonomous University of Barcelona (UAB), Barraquer Ophthalmology Center (Barcelona), where she obtained an OVERALL RATING OF OUTSTANDING, in addition to the JOAQUÍN RUTLLÁN PRIZE for the best master's thesis.

Doctorate (PhD) in Ophthalmology and Visual Sciences from the Federal University of São Paulo (Unifesp).

Specialist in diagnosis and treatment of pathologies of the retina and macula, expert in age-related macular degeneration, diabetic retinopathy, as well as retinal vascular diseases.

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.

Titles:

• CEO, Vesper Ventures SA.
  
• Member of Endeavor Global;
  
• Board of Trustees member at the Fraunhofer Project Center for Advanced Manufacturing @ ITA.
• Former CEO Welle Laser SA.
  

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.