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ReNAgade Therapeutics has burst onto the RNA technology scene by announcing a $300m financing round. With its delivery system, Cambridge, Massachusetts-based biotech says it aims to “address major limitations in RNA therapeutics by enabling the delivery of RNA medicines to previously inaccessible tissues and cells in the body.”
Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene therapy is a new therapeutic approach in which genes are used to treat or prevent diseases.
The arrival of COVID-19 brought precious few positives, but one that emerged for the pharmaceutical industry was the emergence and validation of mRNA vaccine technology to prevent disease. Pfizer already has a head start on competitors in the field, having achieved the first US FDA approval for an mRNA vaccine, alongside BioNTech.
The RNA Revolution: From mRNA Vaccines to RNAEditing. The age of RNA is officially here, and it’s here to stay as more than a passing life science trend. The power of its real-world application was demonstrated by the resounding success of the mRNA COVID-19 vaccines. RNA in the Making.
Dr Jennifer Harbottle, senior scientist in the R&D Base Editing team of PerkinElmer’s Horizon Discovery business, looks at progress made in the realms of biotechnology and next-generation diagnostics, vaccines and therapeutics, including the application of CRISPR-Cas9 geneediting in developing and refining cell therapies.
Acuitas’ LNP technology will support Bayer’s in vivo geneediting and protein replacement programs by specifically delivering RNA payloads to the desired target organ, the liver. ” “We are delighted to partner with Bayer in the area of gene therapy. Financial details were not disclosed.
Over 26 weeks of Sunlenca combined with other antiretroviral drugs, 81 percent of participants achieved HIV RNA suppression, reaching levels low enough to be considered undetectable. These findings signify a significant step forward in the development of AGT103-T as a potential gene therapy for HIV. percent in the placebo group.
An area of interest has been deciphering ALS’s genetic underpinnings and delivering functional copies of dysfunctional genes to the patient. The targeted ASOs aim to downregulate the expression of gene mutations that are associated with gain-of-function toxicity that leads to motor neuron loss in some ALS cases.
With each passing year, pharmaceutical companies around the globe strive to deliver cutting-edge medicines, therapies and vaccines that impact the lives of millions. Pfizer’s Top 5 Best-Selling Drugs of 2022: 1) Comirnaty Comirnaty is an mRNA-based vaccine indicated for the prevention of COVID-19. billion in 2022. for a pediatric dose.
From isolating SARS-CoV-2 in early January to sequencing its genome shortly thereafter and having a prototype vaccine against it within days, scientific process and progress have held steadfast throughout the pandemic. For example, investment in mRNA vaccines and viral vectors came well before January, many years before it.
The capabilities of Viroclinics and DDL allowed us to rapidly test repurposed drugs, new vaccines and antiviral solutions for the prevention and treatment of patients affected by COVID-19 in 2020. The opportunity here for our expanded CRO to advance personalised medical solutions is huge.
From leveraging artificial intelligence (AI) to streamline diagnostics and treatments to exploring the untapped potential of RNA-based therapeutics, biotechnology is shaping the future of healthcare and beyond. As of January 31, 2024, approximately 131 unique RNA-based therapies are in clinical development across various therapeutic areas.
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