Moderna advances experimental mRNA therapy for a rare genetic disease

Propionic acidemia is a rare hereditary disease that results from the mutation of two genes (frepik).

the boost whose platform messenger RNA Science surprises almost daily. this is a technology which revealed its importance during the pandemic – reflected in the efficacy and safety of vaccines against COVID-19– and is already showing encouraging results against a large number of diseases. from cancer to others distortions Without remedial one of two remedyThis platform has become a true revolution for human health.

Modern One of the biopharma that is in full swing towards a new frontier for healthcare. with a large number clinical trials Across the planet, and focusing on various diseases, mRNA-related news is on a path that sets this platform as one before and one after In humanity.

now in a Special Advance for InfobaeModerna reported that a Treatment experimental made with mRNA (called mRNA-3927) showed the first promising data for propionic acidemia ,the countrysidefor its abbreviation in English), a pathology that originates from “the” gene mutation PCCA and PCCB”.

This genetic disease it’s about a rare hereditary metabolic disorderserious, with a number of diseases And impermanence important, affecting one in 100,000 to 150,000 people Whole world.

In Phase I/II of development, Moderna specialists worked with 16 participants /Reuters/Brian Snyder/

“Inborn errors of metabolism are rare genetic disorders that change the way the metabolism Body metabolizes or breaks down Eat Often, they are caused by enzymes or proteins that are missing or not working fully,” he explained. Modern already infobae, stressing that “this disorders usually affect children at an early age,

According to U.S. National Institutes of Health (NIH), these genes, which are mutated in the pathology, “provide instructions for the production of two parts (subunits) of an enzyme called propionyl-CoA carboxylase, which plays a role in the normal breakdown of proteins.” Protein”. Specifically, it enzymes Process Various amino acids and helps break down some of the types fat and cholesterol in the body.

When these genetic mutations occur, the function of the enzymes is disrupted and therefore the normal breakdown of these molecules does not occur.

Result: Compounds such as a substance called propionyl-CoA, among others, manage to accumulate in the body brain and nervous system damageWhich generates serious health problems associated with the same pathology.

These disorders can affect children at an early age and affect the process of metabolizing food, reports Moderna.

the truth is that propionic acidemia It is characterized by recurrent life-threatening decompensated metabolic episodes and multisystem complications.

In other words, when these substances reach toxic levels, degeneration occurs that can produce neurological manifestations, cardiomyopathy, arrhythmias, growth retardation, recurrent pancreatitis, bone marrow suppression, and a predisposition to infection. Furthermore, in the long term, multi-organ complications occur, among other consequences.

one in exclusive preview For Infobae, Moderna experts detailed that the results of Phase I/II, totaled 16 participants, Over a year, showed encouraging results. “After initiation of mRNA-3927 treatment, most participants reported a metabolic decompensation episode (MDE) in the 12 months prior to dosing minor incident or did not present episodes of metabolic decompensation after treatment”.

temporary data Did rehearsal introduced in American Society for Gene and Cell Therapy (ASGCT) Annual Meeting, This Phase I/II study was established as a multicenter, open-label study designed to evaluate the safety, pharmacodynamics, and pharmacokinetics of mRNA-3927 in participants 1 year of age or older.

Some of the consequences of this pathology are lesions in the nervous system and brain (modern).

Similarly, from Biopharma he indicated that the trial used “a single approach”. dose escalation to evaluate intravenous administrationof drug, which was every 3 weeks in the initial dose and every 2 weeks thereafter. Overall, as the study progressed, volunteers He was to receive a total of 10 doses.

In the first phase, we sought to determine safety and tolerability, while in the second, pharmacology, evaluation of potential plasmatic biomarkers, and frequency and duration of episodes of metabolic decompensation (EDM).

In other words, a little simpler, how was it analyzed in this first phase (which was about 10 doses) Sure And satisfactory There was treatment, so side effects were analyzed, while in the second they began to analyze what was the “ideal” ratio of the drug for patients, what symptoms survived in the blood of volunteers and, moreover, how to deal with such disappointing results. How many times did this decomposition happen with

Moderna said, “There were no dose-limiting toxicities or study interruptions due to drug-related treatment-emergent adverse events (“TEAEs”). Side effects, While they highlighted the presence of “serious adverse events (“SAEs”) in eight participants, noting that “the majority were related to propionic acidemia and not related to mRNA-3927”. They also indicated that “the majority of events happened in the first dose.

For many experts, the messenger RNA platform has come to replace medicine (Freepik)

In that respect, Dr. Kyle Hollen, MD, Senior Vice President and Head of Development, Therapeutics and Oncology at Moderna said, “We continue to see encouraging results with mRNA-3927 as we enter this. dose expansion phase where we will further evaluate safety, efficacy And we will determine the recommended dose for future clinical studies.”

“This is the first clinical trial to report results of an mRNA therapy for intracellular protein replacement, and to date we have more than 13 years of experience in treating patients,” the expert emphasized and concluded. : “We look forward to continuing our efforts to explore the therapeutic potential of our mRNA platform for propionic acidemia and other rare diseases.”

Moderna is already positioned as one of the most innovative pharmaceutical companies, thanks to its mRNA platform. He Dr. Paul Burton, One of the world’s brightest scientific minds was behind this breakthrough infobae He They were already preparing to face and fight other diseases.

Safety, pharmacodynamics and pharmacokinetics were evaluated in Phase I/II. Referring to side effects, Moderna explained that there were “no dose-limiting toxicities or study interruptions” (Freepik)

,We all chased a dream and made it a reality with the Messenger RNA platform. Today we are prescribing a new medicine, Changing current treatments to provide answers to diseases that don’t have it. The future is ours”, he expressed with a mixture of force and pity, Medical Director of Moderna in a video call.

According to what he said, the company has “portfolio that aims to address infectious diseases that cause a considerable health burden, including respiratory viruses, latent viruses, global health threats, and now norovirus and Lyme disease.”

He even pointed out that “Moderna is advancing seven vaccine candidates against five viruses that cause cryptic infectionsFive of which are in clinical trials.

“Latent viruses can reactivate during times of stress or when immunity is compromised and cause clinical symptoms. The defining characteristic of latency is Human immunodeficiency virus (HIV) and members of the Herpesviridae family, including cytomegalovirus (CMV), Epstein-Barr virus (EBV), varicella-zoster virus (VZV), and herpes simplex virus (HSV)”Burton explained.

,There is no time limit. With the messenger RNA platform came the transformation of medicine. And we look forward to bringing concrete solutions to the health of millions of people in the years to come”, concluded Burton.

keep reading:

COVID, ALS, HIV and Cancer: What Vaccines Is Moderna Developing?
Moderna Director Paul Burton: “The messenger RNA vaccine will provide an effective response to many diseases that currently have no cure”
What is the power of messenger RNA vaccines to treat cancer?

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