A Brazilian biotechnology startup has created a molecule capable of revitalizing and restoring human neurons through RNA therapies. The groundbreaking outcome, achieved by Aptah Bio, emerged from trials conducted in Canada, involving cells from the central nervous system of both healthy elderly individuals and those with Alzheimer’s.

“We developed a patent for a new RNA technology, which is a molecule that enters the nucleus of all cells and corrects the error that was occurring not in a specific protein or some specific RNA, but in the cell machinery. In other words, we can correct all toxic RNAs and Proteins with this single molecule.”

– Rafael Bottos

What are the causes?

As we age, breaks occur in the strands of our DNA, creating a kind of scar in the genetic code. This means that, as we advance in age, more scars form, compromising the process of translating the DNA code into the different proteins that make up our cells. This causes our body to start producing proteins improperly. It is precisely these toxic proteins that lead to sporadic diseases such as cancer and Alzheimer’s. “Research confirms that as we age, we lose the balance between long and short mRNAs. This happens throughout the body and in various species,” explains Rafael Bottos, CEO and co-founder of Aptah Bio, a biotechnology startup focused on new RNA therapies. “Science had already proven, and we also observed in our study, that older individuals have a proportionally higher number of short RNAs in the body, induced by the continuous and more frequent breakage of long RNAs, which is why we age and develop diseases.” He adds that, in addition to aging, habits such as smoking, sun exposure, and the use of illicit drugs also accelerate this process.

The study

Aptah has pioneered the development of the first compound capable of restoring the balance of mRNA sizes in cells during the production of all proteins. “This is unprecedented in science. Today, we have medications that reverse this shortening in specific proteins. Our drug regulates and controls the production of all proteins in our organism, ensuring the quality of gene production in all cells. We’re talking about over 100,000 different proteins,” says the startup’s chief scientist, Caio Leal. “We can prevent or reduce the risk of epigenetic modifications in DNA shortening mRNAs, which lead to diseases.”

In the biotech’s studies, over 3,000 neurons from both healthy and Alzheimer’s patients, each around 75 years old, were used. “We not only managed to rejuvenate a healthy cell from an elderly individual but also succeeded in restoring a diseased cell, returning its neuronal capacity and making it healthy again,” celebrates Rafael Bottos.

To validate the achievement, the team of scientists analyzed the synapses of cells subjected to Aptah Bio’s medication. Over 108 days, electrophysiological analyses were conducted, measuring the electrical properties of cells and tissues. “Researchers confirmed a 40% increase in synapses in diseased neurons,” adds Caio. “This means we can recover the activity of Alzheimer’s neurons and rejuvenate human neurons without any pathology.” According to CEO Rafael Bottos, the outlook is that with even more prolonged use of the medication, the results will be even more effective.

For a comparative study, Aptah selected mRNA strands producing four types of proteins directly linked to Alzheimer’s development within the neuron sample. In the healthy elderly patient, there was no significant alteration. In diseased cells, however, there was a recovery in the size of this mRNA, ceasing the production of the toxic forms of proteins and restoring their original function. “Furthermore, analyses on healthy neurons confirmed that the drug is beneficial for cells. Our drug only corrects where there is an error, a defect, without impacting healthy cells,” explains Rafael.

The process of cleansing these toxic cells does not happen overnight. This implies that, in the case of degenerative diseases, future treatment with this molecule will likely involve continuous use. “The expectation is that, beyond Alzheimer’s, the drug created by Aptah Bio will also be indicated for treating frontotemporal dementia, a disorder faced by actor Bruce Willis, and ALS, Amyotrophic Lateral Sclerosis, among many others,” concludes the CEO.

Aptah Bio

Established in 2020, Aptah Bio is a biotechnology startup specializing in innovative RNA therapies, initially targeting oncological and neurodegenerative diseases. The company has created a unique molecule capable of reshaping cells to regulate the production of various toxic proteins associated with sporadic genetic disorders, such as Alzheimer’s.

The biotech aims to address a range of age-related diseases with a single medication and has already achieved success in tests with cells (in vitro) and animals (in vivo). Headquartered in both California and Brazil, the company is gearing up to commence human trials early next year.

Aptah Bio was founded by Rafael Bottos, now serving as CEO, and Caio Bruno Leal, the company’s chief scientist. The Administrative Board includes renowned experts in the European and American markets, with Dr. Dieter Weinand, former global CEO of Bayer Pharma, serving as the Chairman of the Board.

As part of the Vesper Ventures portfolio, a venture builder fund focusing on advanced biotechnology, the startup is positioned for further growth.

Source: Olhar Digital

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