Broad Institute

JULY 24, 2025

Murphy New treatments are sorely needed for Ebola virus infections, which cause rare, yet severe, outbreaks. Related news Imaging combined with genetic screening of cells enhances genomic discoveries Although outbreaks of Ebola virus are rare, the disease is severe and often fatal, with few treatment options.

Virus

BioPharma Drive: Drug Pricing

JUNE 11, 2025

The startup, which is backed by the venture arms of Sanofi, Roche and Novartis, is using dual adeno-associated viruses to help overcome the packaging constraints of current genetic medicines.

Packaging

Science Daily: Pharmacology News

JUNE 24, 2025

Scientists have uncovered a stealthy tactic used by the SARS-CoV-2 virus: one of its proteins can leap from infected cells to healthy ones, effectively tricking the immune system into attacking the body’s own tissues.

Virus

Chemical Biology and Drug Design

JUNE 19, 2025

Thienopyrimidines exhibit multifarious features, mainly, this is due to their structural resemblance with purine bases, such as guanine and adenine. Their intricate molecular structures enable them to interact with specific molecular targets, such as enzymes, receptors, and cellular signaling pathways, leading to their therapeutic efficacy.

FDA Approval

Covalent Modifiers

FEBRUARY 25, 2025

The resulting compounds displayed antiviral activity against live virus that was irreversible after washout due to the covalent binding mechanism. We demonstrate the utility of this approach using the SARS-CoV-2 spike-ACE2 proteinprotein interaction and identify multiple covalent macrocyclic inhibitors that disrupt this interaction.

Metabolic Stability

Chemical Biology and Drug Design

NOVEMBER 10, 2024

Combined with network pharmacology and experimental verification, this study found that platycodin D and luteolin in Jiegeng ( Platycodonis Radix ) may regulate inflammation through the IL-17 signaling pathway to treat influenza virus pneumonia.

Virus

Science Daily: Pharmacology News

JUNE 20, 2025

Stunningly, blocking this single enzyme shuts the virus down completely. Researchers discovered that it actively reshapes the 3D structure of the human genome within hours of infection, using host enzymes like topoisomerase I to gain access to crucial genetic machinery.

Virus

Broad Institute

MAY 21, 2025

The team has successfully created over 1000 of them, and each consist of: a harmless adeno-associated virus (or AAV) that acts like a shuttle capable of transporting specially designed DNA into the cell; a segment of DNA (an enhancer) that acts like an activation switch to mark or trigger a change in how the cell functions.

Disease

Broad Institute

JULY 21, 2025

Moreover, the function of their Atp1a3 protein was restored in the brain, and their motor and cognitive deficits were ameliorated. The scientists delivered the prime editors to cells in mice using clinically validated viruses called AAVs, which are already used in FDA-approved gene therapies targeting brain cells.

Disease

Drug Target Review

JULY 21, 2025

Viral vectors use a virus’s blueprint to deliver genetic material into cells. Delivery methods are also key, with viral vector-based systems being the most common. The two most used vectors are Adeno-associated viruses (AAVs), which boast low immunogenicity and long-term gene expression, and lentiviruses, which offer stable gene transfer.

Drug Development

The Pharma Data

JUNE 24, 2025

A significant proportion of gene therapies currently in development utilize recombinant adeno-associated virus (rAAV) vectors due to their favorable safety profile and ability to effectively deliver genetic material to target cells. However, the gene therapy sector faces its own unique set of obstacles.

Therapies

Broad Institute

JANUARY 14, 2025

But the researchers needed to deliver the base editors to the brain. Building on previous work by the vector-engineering lab of Ben Deverman at the Broad, the team developed a pair of adeno-associated viruses (AAVs) to package and deliver the base-editing machinery to brain cells.

Disease

Broad Institute

FEBRUARY 27, 2025

One group set apart from the rest, and its members were particularly intriguing because they were encoded by genes with regularly spaced repetitive sequences reminiscent of an essential component of CRISPR systems. These were the TIGR-Tas systems. In some, the RNA-binding region is adjacent to a DNA-cutting part of the protein.

RNA

The Pharma Data

JUNE 15, 2025

While elevations in liver enzyme values are recognized as a class-related side effect with adeno-associated virus (AAV)-based gene therapy, the exact mechanisms that contribute to these abnormalities remain unclear.

Immune Response

DrugBank

JUNE 18, 2025

Initially approved for dengue fever, post-market data revealed that the vaccine increased the risk of severe disease in patients who had never been exposed to the virus before vaccination. Conversely, Sanofi’s Dengvaxia vaccine case highlights the consequences of inadequate post-market surveillance.

Drug Development

Broad Institute

MAY 27, 2025

To shuttle the base editors to specific cells in mice, the researchers packaged them into dual AAV9 vectors, an adeno-associated virus designed to deliver cargo to neurons. The base editors stabilized the repeat tracts in mouse models of Friedreichs ataxia and Huntingtons disease.

DNA

The Pharma Data

JUNE 9, 2025

This new monoclonal antibody therapy is designed to prevent lower respiratory tract disease caused by respiratory syncytial virus (RSV) in neonates and infants born during or entering their first RSV season. Food and Drug Administration (FDA) granting approval for ENFLONSIA™ (clesrovimab-cfor).

FDA Approval

Science Daily: Pharmacology News

JULY 23, 2025

HIV antivirals may be the key to stopping HTLV-1, a deadly virus with no cure. In a decade-long study, researchers successfully suppressed the virus in mice and discovered a way to kill infected cells, offering hope for the first preventative and curative treatments.

Virus

The Pharma Data

JUNE 13, 2025

Importantly, this will enable them to produce mRNA vaccines during periods between health crises, retaining trained personnel, technical expertise, and production capacity while retaining the ability to respond quickly in the event of future disease outbreaks.

Vaccine

The Pharma Data

JUNE 26, 2025

CDC panel backs long-acting monoclonal antibody to protect infants during their first RSV season; drug also included in federal Vaccines for Children program Merck , operating as MSD outside the United States and Canada, has announced a key regulatory milestone for its respiratory syncytial virus (RSV) prevention program. As the U.S.

Vaccine

The Pharma Data

JUNE 15, 2025

This update comes after two cases of fatal acute liver failure (ALF) were reported in non-ambulatory DMD patients who received Elevidys, a phenomenon that underscores the ongoing complexities and safety signals related to adeno-associated virus (AAV)-mediated gene therapy.

Regulations

The Pharma Data

JUNE 9, 2025

Sanofi Ramps Up Early Global Distribution of Beyfortus to Meet Growing RSV Season Demand, Enhancing Protection for Infants Worldwide In a proactive move to ensure readiness for the 2025-2026 respiratory syncytial virus (RSV) season, Sanofi has announced that shipments of Beyfortus® (nirsevimab) will begin in early Q3 2025.

Virus

Codon

JUNE 5, 2025

If the same virus invades the cell a second time, the gRNA’s spacer sequence will bind to the matching viral DNA sequence, then be cut by the Cas protein. .” Despite its association with genome editing, 1 CRISPR didn’t start out as a tool for fighting genetic disease.

DNA

New Drug Approvals

JUNE 28, 2025

By binding to the active site of the protease, the drug prevents this cleavage process, effectively halting viral assembly and impeding the virus’s ability to produce future virions. [1] This protein is a crucial enzyme responsible for cleaving viral polyproteins into functional subunits essential for viral replication.

Clinical Trials

New Drug Approvals

JULY 8, 2025

1428321-10-1 Pritelivir mesylate is an antiviral drug currently under development, specifically targeting herpes simplex virus types 1 and 2 (HSV-1 and HSV-2). Pritelivir (development codes AIC316 or BAY 57-1293 ) is a direct-acting antiviral drug in development for the treatment of herpes simplex virus infections (HSV).

Virus

FDA Law Blog: Biosimilars

APRIL 7, 2025

However, several of Zenrelias promotional materialsspecifically, FDA looked at a consumer-directed website, a product brochure and risk tracker, and a slide deckomitted the specific phrase from modified live virus vaccines from the boxed warning found in the Important Safety Information.

Vaccine

Drug Target Review

MAY 26, 2025

. “Because analytical questions from both manufacturers and regulators are increasing, we have to think smarter with novel technologies,” he says. “Refeyn, for example, has a technology which can investigate and provide data on the aggregation of viruses, which is a key attribute for QC release of some gene therapy drugs.”

Drug Development

National Institute on Drug Abuse: Nora's Blog

NOVEMBER 25, 2024

3 Evidence on transmission of hepatitis C virus (HCV) is less clear, but in Europe, where SSPs are more widely used, high SSP use is associated with a 76% reduction in HCV transmission. Use of SSPs in places where they have been allowed to operate in the United States is associated with a 40-60% reduction in HIV transmission.

Drugs

Codon

NOVEMBER 1, 2024

More recently, Michael Elowitz's group — also at Caltech — published a method to study changes in RNA molecules within living cells.

DNA

BioPharma Drive: Drug Pricing

JULY 10, 2025

Most Popular Merck to buy Verona and its lung drug in $10B deal Trump says pharmaceutical, copper tariffs coming ‘very soon’ Deep Dive Protein degraders: chasing undruggable targets Lilly gets FDA OK of modified dosing for Alzheimer’s drug Library resources Podcast Digital Behavioral Therapy for Fibromyalgia Custom content for Medrio Webinar - on demand (..)

Vaccine

BioPharma Drive: Drug Pricing

JULY 10, 2025

Most Popular Merck to buy Verona and its lung drug in $10B deal Trump says pharmaceutical, copper tariffs coming ‘very soon’ Deep Dive Protein degraders: chasing undruggable targets Lilly gets FDA OK of modified dosing for Alzheimer’s drug Library resources Podcast Digital Behavioral Therapy for Fibromyalgia Custom content for Medrio Webinar - on demand (..)

Drugs

BioPharma Drive: Drug Pricing

JULY 18, 2025

While the two treatments are constructed differently, they both use the same kind of engineered virus to deliver a replacement gene to the body’s muscles. The man died of complications from acute liver failure in June.

Therapies

Science Daily: Pharmacology News

JULY 12, 2025

A cat named Pepper has once again helped scientists discover a new virus—this time a mysterious orthoreovirus found in a shrew. Researchers from the University of Florida, including virologist John Lednicky, identified this strain during unrelated testing and published its genome.

Virus

The Pharma Data

JUNE 11, 2025

With this approval, MAVYRET becomes the first and only DAA treatment approved to treat both acute and chronic hepatitis C virus (HCV) infections in patients as young as three years old, with or without compensated cirrhosis, using an eight-week regimen. No one should die of hepatitis C.”

FDA Approval

The Pharma Data

JUNE 19, 2025

BT524 is a lyophilized, virus-inactivated fibrinogen concentrate that can be reconstituted and administered rapidly, ensuring a more controlled delivery of the needed clotting protein. Grifols’ BT524, developed by its subsidiary Biotest, offers a more standardized and potentially safer alternative.

Therapies

The Pharma Data

JULY 3, 2025

One such initiative includes a collaboration with the Bill & Melinda Gates Foundation aimed at developing low-cost monoclonal antibodies (mAbs) for infectious diseases such as malaria and respiratory syncytial virus (RSV). This partnership has recently reached a key milestone, resulting in a second disbursement of $1.5 million award.

International

Science Daily: Pharmacology News

JUNE 6, 2025

Scientists have uncovered over 200 new giant viruses lurking in ocean waters that not only help shape marine ecosystems but also manipulate photosynthesis in algae. These massive viruses once nearly invisible to science are now being exposed using powerful supercomputing and a new tool called BEREN.

Virus