IN SHORT
We can restore overall RNA integrity and correct multiple proteins simultaneously.
Aptah's technology holds the potential to address a huge unmet medical need, ranging from Alzheimer’s to different kinds of cancer.
RNA WiCo™ - RNA Widespread Correction
At Aptah, we're pioneering a groundbreaking approach to combat age-related diseases:
Aptah Bio is working in the forefront of a novel and proprietary rejuvenation technology called RNA WiCo™ (RNA Widespread Correction) able to edit, modulate and control the expression of different RNAs in a unique way.
Its lead compound targets U1-snRNP and is the first drug designed to ensure the proper function of U1, leading to the expression of widespread full-length 3′UTRs. This directly contributes to the restoration of RNA integrity and the inclusion of non-coding RNA binding sites.
Results show the recovery of homeostatic protein expression, cell cycle, and a clear positive biological effect in different cell types and animal models. This therapy holds the potential to address a huge unmet medical need, ranging from Alzheimer’s to different kinds of cancer.
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APT20TTMG does not silence any specific target, but rather works as a template for accurate RNA processing.
It does not change physiological alternative splicing and polyadenylation.
Unveiling Our Solution:
A Three-Step Rationale
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Aging affects RNA integrity
RNA shortening leads to innumerous pathologies
Maintaining RNA integrity is crucial for healthy cells. Aging leads to an increasingly frequent and random loss of RNA integrity over time, resulting in overall RNA truncation, the production of harmful proteins, and the onset of age-related diseases.
Recently, researchers have identified the sporadic occurrence of premature cleavage and polyadenylation, which leads to shorter 3′UTRs and a reduction in important binding sites for microRNAs. Data strongly suggests that widespread reductions in the 3′UTR-based regulatory capacity of mRNAs are closely linked to the development of multiple sporadic diseases.
Ibañez-Solé; Barrio; Izeta, 2023. DOI: https://doi.org/10.1016/j.isci.2023.106368
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Aging affects RNA length
RNA shortening leads to innumerous pathologies
In the intricate world of genetic makeup, longer genes often encounter a greater risk of premature cleavage and splicing errors, paving the way for various diseases to manifest. Among these, neurodegenerative conditions, oncological challenges, metabolic imbalances, and ophthalmological ailments stand prominently. What's fascinating is the recurring pattern across diverse tissues and species, notably prevalent in individuals who have a history of smoking or sun exposure. This pattern also resonates deeply with those grappling with Alzheimer's disease and related afflictions. Such insights underscore the critical need for innovative solutions like ours, which aim to tackle these underlying genetic mechanisms, offering hope for improved health and longevity.
Ibañez-Solé; Barrio; Izeta, 2023. DOI: https://doi.org/10.1016/j.isci.2023.106368
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Our Target: U1 snRNP
U1 snRNPs (U1) regulates mRNA quality, variety and length.
Pre-mRNA splicing and polyadenylation are crucial steps in mRNA maturation. U1 snRNP, an essential component of the splicing machinery. Recent studies have shown that U1 snRNP also plays a global role in 3' end mRNA processing by preventing premature 3'-end cleavage and polyadenylation (PCPA), which is important for the full-length transcription of thousands of protein-coding and long noncoding genes.
Numerous studies have demonstrated abnormalities in U1 snRNP assembly and function in various sporadic diseases, including neurodegenerative diseases and various types of cancer.
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Our Target: U1 snRNP
U1 snRNPs (U1) regulates mRNA quality, variety and length.
At the heart of eukaryotic RNA length regulation lies the intricate orchestration known as 'U1 snRNP telescripting,' where the U1 snRNP plays a pivotal role. Among the vast array of eukaryotic protein-RNA complexes, few have garnered as much attention and scrutiny as the U1 snRNP. In the human context, the assembly of U1 snRNP is a complex affair, governed by a delicate interplay of protein-RNA and protein-protein interactions. This assembly is predominantly composed of three U1-specific proteins - U1A, U1-70 K, and U1C - along with seven Sm proteins and the U1 snRNA. Such meticulous assembly underscores the intricate molecular machinery that underpins essential cellular processes, highlighting the significance of U1 snRNP in the realm of genetic regulation.
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Our Technology:
RNA WiCo
RNA Widespread Correction
Our technology, represented by the lead compound APT20TTMG, restores mRNA quality and ensures the proper synthesis of multiple proteins by facilitating accurate U1 snRNP assembly.
This unique modulation-based compound is the result of advanced computer simulations and belongs to a new class of molecules:
Synthetic cDNA single-stranded
APT20TTMG holds promise for addressing a wide range of sporadic genetic disorders by serving as a template for precise RNA processing without affecting physiological alternative splicing and polyadenylation.
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Our Technologie:
RNA WiCo
RNA Widespread Correction
Our technology, represented by the lead compound APT20TTMG, restores mRNA quality and ensures the proper synthesis of multiple proteins by facilitating accurate U1 snRNP assembly. This unique modulation-based compound, approved by patent, is the result of advanced computer simulations and belongs to a new class of molecules: synthetic sncDNA single-stranded.
APT20TTMG holds promise for addressing a wide range of sporadic genetic disorders by serving as a template for precise RNA processing without affecting physiological alternative splicing and polyadenylation.
OUR PIPELINE
WHY APTAH
Current technologies are unable to address the great majority of diseases due to their multifactorial aspect and lack of etiology understanding.
OUR TEAM & LEADERSHIP
Marcelo do O
Chief Business Officer
Juliana M. Bottos, MD PhD
Scientific Advisory Board | Ophthalmology
Prof. George Church
Scientific Advisory Board
Dieter Weinand, MBA
Chairman of the Board
Rafael Bottos
Co-Founder & CEO
Caio Bruno Leal
Co-Founder & CSO
Camila Zimmer, PhD
Head of Preclinical Studies
Vanessa Sinatti, PhD
Head of Bioinformatics
Jonas Sister
Business Development
João Camargo
CFO
Ericks Sousa, PhD
Biochemistry
About 90% of diseases are sporadic, meaning they are not associated with inherited genetic mutations. However, their etiology remains a long-standing question for scientists.
While many diseases share fundamental biological processes necessary for their development and progression, they also differ in their reliance on specific pathways. In recent years, targeted therapies have been introduced with varying degrees of success. However, single-targeted therapies may only be effective for certain types of diseases and may not address malignancies that rely on multiple pathways, a common aspect in sporadic diseases.
