XTalks

DECEMBER 22, 2021

The RNA Revolution: From mRNA Vaccines to RNA Editing. The age of RNA is officially here, and it’s here to stay as more than a passing life science trend. RNA technology is not new nor has its potential been surprising. RNA in the Making. So why did this perceived RNA ‘revolution’ take so long?

Life Science 98

Life Science RNA Vaccination Vaccine 98

pharmaphorum

NOVEMBER 24, 2020

The FDA has approved Alnylam’s gene silencing drug Oxlumo, the first treatment for primary hyperoxaluria type 1 (PH1), an ultra-rare and life-threatening genetic disorder. The post FDA approves Alnylam’s ultra-rare disease drug Oxlumo appeared first on.

FDA Approval 52

FDA Approval Gene Silencing Drugs RNA 52

pharmaphorum

JANUARY 10, 2022

Previously, messenger RNA (mRNA) therapies were a niche part of the global R&D pipeline, now a wide section of the public is at least familiar with the name of this type of therapy. Pfizer already has a head start on competitors in the field, having achieved the first US FDA approval for an mRNA vaccine, alongside BioNTech.

RNA 111

RNA Vaccination Vaccine Pharma Companies 111

XTalks

NOVEMBER 30, 2023

New FDA-Approved Treatments For HIV HIV treatment involves the administration of combined antiretroviral therapy (ART) to effectively suppress the viral load, maintain or enhance immune function and reduce the risk of opportunistic infections and cancers commonly associated with HIV. aiming to end the HIV epidemic by 2030.

FDA Approval 111

FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

XTalks

JUNE 5, 2023

RSV belongs to the family of negative-strand RNA viruses and was first identified in 1955. The genetic material of RSV (respiratory syncytial virus) encodes 11 proteins. The Global RSV Vaccine Market Multiple RSV vaccines have received FDA approval or are in advanced stages of clinical trials. How Does Abrysvo Work?

Vaccination 98

Vaccination Vaccine FDA Approval Protein 98

The Pharma Data

NOVEMBER 24, 2020

FDA Approves Oxlumo (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1. Food and Drug Administration (FDA) approved Oxlumo (lumasiran) injection for subcutaneous use, the first-ever therapy available for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.

FDA Approval 52

FDA Approval Production RNA Medicine 52

Worldwide Clinical Trials

MAY 30, 2024

From drugs previously FDA-approved for gallstone diseases, Parkinson’s disease, and even cancer, researchers are working tirelessly to test and establish whether these pharmacotherapies with well-defined safety profiles may have any potential for efficacy in treating ALS.

Gene 204

Gene Clinical Trials Clinical Trials Gene Therapy 204

The Pharma Data

NOVEMBER 18, 2020

.” A handful of other tests have been cleared by the FDA for at-home collection of samples, but all must then be shipped to a lab for processing, The New York Times reported. Like PCR, the new test repeatedly copies genetic material until it reaches detectable levels. But the new at-home test relies on similar principles.

FDA Approval 52

FDA Approval Vaccination Vaccine Genetics 52

XTalks

SEPTEMBER 4, 2024

Alnylam Pharmaceuticals announced promising results from its HELIOS-B Phase III clinical trial evaluating vutrisiran, an investigational RNA interference (RNAi) therapeutic for treating transthyretin amyloidosis with cardiomyopathy (ATTR-CM). As of March 2024, the FDA has approved six small interfering RNA (siRNA) therapies.

Trials 52

Trials RNA Protein Gene Editing 52

The Pharma Data

JANUARY 21, 2021

In the ATLAS study, CABENUVA met the primary endpoint for noninferiority (the proportion of participants with plasma HIV-1 RNA ?50 50 copies per milliliter [c/mL] at Week 48), with a comparable number of patients receiving either CABENUVA or their daily current antiretroviral regimen (CAR) having an HIV-1 RNA level ?50

FDA Approval 52

FDA Approval RNA Drugs Containment 52

XTalks

OCTOBER 26, 2023

The US Food and Drug Administration (FDA) approved new updated COVID-19 vaccines from both Pfizer-BioNTech and Moderna that target the Omicron XBB.1.5 In May, the FDA approved GSK’s Arexvy as the first RSV vaccine, which was shortly followed by the approval of Pfizer’s RSV vaccine Abrysvo.

Vaccination 105

Vaccination Vaccine RNA Manufacturing 105

XTalks

NOVEMBER 7, 2022

Immunofluorescent staining of CTCs is a CTC isolation method that has been used since the first US Food and Drug Administration (FDA) approval in 2013 for the CellSearch ® CTC enumeration platform. Regulatory Approvals for CTC-Based Assays in Oncology. Table 1: Some examples of cfDNA-based assays that are approved by the US FDA.

Clinical Trials 98

Clinical Trials Clinical Trials Trials FDA Approval 98

pharmaphorum

DECEMBER 23, 2020

There was some steady progress in neurology – in February FDA approved Lundbeck’s eptinezumab prophylactic treatment for migraine, the last from a gang of four drugs from a new class. It was the third approval from Alnylam’s pipeline of RNA interference therapeutics to make it to market. months, compared with 6.7

Marketing 96

Marketing Gene Gene Silencing Drugs 96

The Pharma Data

DECEMBER 16, 2020

To this point, Moderna has only submitted two months of follow-up safety data, and the FDA typically requires six months for a full approval. intends to ship just shy of six million doses of Moderna’s vaccine once the FDA approves EUA.

Vaccination 52

Vaccination Vaccine Immune Response FDA Approval 52

The Pharma Data

DECEMBER 8, 2020

The assumption is SciNeuro will start with Parkinson’s disease and pain in addition to rare genetic diseases, after scaling up staffing. . Laying down a new track for RNA processing, Remix launched with $81 million in financing. Pear’s reSET, reSET-O and Somryst are the first PDTs to receive FDA approval for treating disease.

RNA 52

RNA Antibody Life Science Protein 52

STAT News

MARCH 6, 2023

Cambridge-based Moderna recently reported encouraging results from a study of another type of personalized cancer vaccine it developed with the pharmaceutical giant Merck; it uses messenger RNA technology to target advanced melanoma. But more work remains before personalized vaccines  can be greenlighted.

Vaccination 98

Vaccination Vaccine Drugs Clinical Trials 98

The Pharma Data

DECEMBER 31, 2020

Food and Drug Administration (FDA) issued Emergency Use Authorization for two COVID-19 vaccines: Pfizer-BioNTech’s BNT162b2 and Moderna’s mRNA-1273. The messenger RNA (mRNA) vaccines encode a form of the spike (S) protein of SARS-CoV-2 virus. FDA Approves First-In-Class Topical Klisyri to Treat Actinic Keratosis .

Vaccination 52

Vaccination Vaccine FDA Approval Hormones 52

The Pharma Data

NOVEMBER 29, 2020

The FDA approved the drug on November 24. PH1 is an ultra-rare genetic disease marked by overproduction of oxalate, which causes deposits of calcium oxalate crystals in the kidneys and urinary tract and can lead to painful and recurrent kidney stones, nephrocalcinosis, progression to kidney failure, and system organ dysfunction.

Genetic Disease 52

Genetic Disease Antibody Licensing Licensing 52

WCG Clinical

MAY 28, 2024

CAR-T Cells Target Harmful B Cells in Lupus CAR-T cell technology, which uses genetic engineering to direct white blood cells to attack specific molecular targets, was originally proposed for treatment of HIV infection and hematological malignancies. The currently approved CD-19 CAR products are autologous, ex vivo cellular therapies.

In-Vivo 40

In-Vivo Clinical Trials Clinical Trials FDA Approval 40

The Pharma Data

OCTOBER 21, 2020

The long-acting regimen of cabotegravir and rilpivirine is an investigational regimen for the treatment of HIV-1 infection in adults to replace the current antiretroviral regimen in patients who are virologically stable and suppressed (HIV-1 RNA less than 50 copies/mL). Source: GSK . Posted: October 2020. Source link.

Clinical Development 52

Clinical Development Development Medicine Clinical Trials 52

The Pharma Data

DECEMBER 18, 2020

In making this determination, the FDA can assure the public and medical community that it has conducted a thorough evaluation of the available safety, effectiveness, and manufacturing quality information. The Moderna COVID-19 Vaccine contains messenger RNA (mRNA), which is genetic material.

Vaccination 52

Vaccination Vaccine Production Manufacturing 52

XTalks

AUGUST 30, 2022

Such therapies may involve small interfering RNA (siRNA) or oligonucleotides in viral transfection platforms, large molecules and antibodies. Direct CNS administration is also FDA-approved for multiple small molecules and ASOs. Targeting the CNS directly has been successful in modern medicine. The patients are waiting!

Trials 83

Trials Drug Delivery Drugs Gene 83

XTalks

DECEMBER 24, 2020

However, for virologists like Lori Frappier, PhD, professor in the department of molecular genetics at the University of Toronto, the outbreak of SARS-CoV-2 was less of a surprise and more of an inevitability. She told Xtalks that, “Experts have always known that this kind of pandemic was a possibility, especially with coronaviruses.

Life Science 111

Life Science Vaccination Vaccine Gene 111

Pharmaceutical Technology

APRIL 24, 2023

Since then, the field of nanomedicine has steadily progressed to reach high points such as the successful use of nanotechnology to deliver messenger RNA (mRNA)-based Covid-19 vaccines. Covid-19 vaccines from Moderna and Pfizer /BioNTech, which use lipid nanoparticles, became the only two FDA-approved vaccines for almost all ages.

Drug Delivery 147

Drug Delivery Vaccination Vaccine Research 147

XTalks

JANUARY 6, 2025

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.

Gene Therapy 111

Gene Therapy RNA Gene Editing Gene 111

XTalks

FEBRUARY 20, 2025

In hereditary angioedema (HAE), for example, donidalorsen an investigational RNA-targeted prophylactic treatment has demonstrated impressive efficacy. With an FDA decision expected by August 21, 2025, donidalorsen could offer a reliable, patient-friendly option for those affected by unpredictable, severe HAE attacks.

Gene Therapy 59

Gene Therapy Gene Research Genetics 59