Drug Target Review

APRIL 9, 2024

In a previous study, we showed that these peptides decorate all cellular RNAs and thus impair the binding of RNA-binding proteins to RNA. Among other discoveries, the group developed inhibitors of the ATR kinase and showed their potential for cancer therapy.

RNA 64

RNA Cell Biology Clinical Development Disease 64

The Pharma Data

MARCH 8, 2021

In both studies, >98% of participants who initiated treatment with Biktarvy and remained in the study achieved and maintained an undetectable viral load (HIV-1 RNA <50 copies/mL) through four years of follow-up (n=235/237 for study 1489, n=241/243 for study 1490). If appropriate, anti-hepatitis B therapy may be warranted.

Treatment 52

Treatment Therapies RNA Laboratories 52

Drug Target Review

SEPTEMBER 18, 2024

What specific cost-cutting measures can AI provide in the manufacturing process of cell and gene therapy, and how do these compare to traditional methods? The cell and gene therapy (CGT) space provides a great example of the potential of AI. AI can vastly improve cell culture, a critical step in cell therapy production.

Therapies 52

Therapies Doctors RNA Production 52

Drug Target Review

FEBRUARY 5, 2024

Adult laboratory mice heal injuries with scar tissue, but spiny mice have the unique ability to regrow lost skin and regenerate musculoskeletal tissues in their body. It also showed that increasing and maintaining this type of signalling in laboratory mice could initiate a regenerative response instead of scarring.

Laboratories 59

Laboratories Immune Response RNA Regulations 59

Drug Target Review

SEPTEMBER 28, 2023

Researchers were able to use our synthetic RNA controls as a reference to verify and validate assays. Once a specific mutation is detected, clinicians can look through available precision medicine therapies to find one to treat the disease or develop a new personalised therapy to treat a patient’s specific form of disease, such as cancer.

DNA 116

DNA Organic Chemistry Science Virus 116

The Pharma Data

JANUARY 17, 2021

That’s not the case with RNA-targeted drugs. Traditional companies have neither the technologies nor the financial incentives needed to develop ultra-rare therapies. “It’s the efficiency of antisense technology, coupled with its other characteristics, that makes n-Lorem’s approach feasible.”. We’re the middleman,” he said.

Disease 52

Disease Clinical Trials Small Molecule Treatment 52

The Pharma Data

JULY 12, 2021

“Our decades-long commitment to fighting the epidemic is stronger than ever as we continue to build on our legacy of research and innovation in HIV,” said Dr. Joan Butterton, vice president, Global Clinical Development, Infectious Diseases, Merck Research Laboratories. “We If appropriate, initiation of anti-HBV therapy may be warranted.

Pharmacokinetics 52

Pharmacokinetics Clinical Trials Trials Treatment 52

Broad Institute

JUNE 27, 2024

Science (2024) Related content New gene delivery vehicle shows promise for human brain gene therapy My Quest to Cure Prion Disease — Before It’s Too Late | Sonia Vallabh | TED Prion diseases lead to rapid neurodegeneration and death and are caused by misshapen versions of the prion protein in the brain. “As

Disease 142

Disease Therapies Clinical Trials Engineering 142

Drug Target Review

AUGUST 28, 2024

Once a patient develops advanced cirrhosis/end-stage liver disease there are no specific therapies to significantly avoid major decompensations and death in the next few years. Could you describe the platform of macrophage biology and cell engineering used by Resolution Therapeutics in developing their cell therapies?

Therapies 116

Therapies Disease Engineering Medical Schools 116

Broad Institute

JUNE 28, 2023

Today, scientists use these reactions to produce customizable DNA and RNA molecules that enable genetic sequencing, drug and vaccine development, pathogen tests, cancer diagnostics, and many aspects of basic biomedical research. “I Longer synthetic DNA and RNA molecules are also critical for modern biologic drugs.

DNA 98

DNA RNA Science Therapies 98

The Pharma Data

JANUARY 21, 2021

Prior to initiating treatment with CABENUVA, oral cabotegravir (VOCABRIA) and oral rilpivirine (EDURANT ® ) should be administered for approximately one month to assess the tolerability of each therapy. In the ATLAS study, CABENUVA met the primary endpoint for noninferiority (the proportion of participants with plasma HIV-1 RNA ?50

FDA Approval 52

FDA Approval Treatment RNA FDA 52

Codon

APRIL 14, 2025

Some, like predicting microbial growth rate from genetics , predicting CRISPR editing efficiency at a locus given a guide RNA , and protein stability prediction are in progress but would benefit from more support, especially to make solutions widely applicable and available to potential users. Inside the FutureHouse laboratory.

Protein Expression 88

Protein Expression Engineering Research RNA 88

Drug Target Review

SEPTEMBER 4, 2023

I had earlier proposed the use of Palmatine and Silver Nitrate in the efficacy against the RNA structure of the COVID-19 virus. 7,9 Dihydrofolate reductase-thymidylate synthase (DHFR) have been successfully exploited for the therapy of bacterial infections and other parasitic diseases such as malaria. References: Antunes Luisa (2023).

Drugs 111

Drugs RNA DNA Disease 111

Codon

JULY 27, 2023

But what happens if you restore these cone cells, using gene therapy? A LEGO robot , made by undergraduate students at Arizona State University, pours sucrose gradients (a tube with dense liquid at the bottom, and less dense liquid at the top), which are used to separate, say, proteins from RNA by spinning them really fast in centrifuges.

Therapies 52

Therapies RNA DNA Engineering 52

Codon

JULY 27, 2023

But what happens if you restore these cone cells, using gene therapy? A LEGO robot , made by undergraduate students at Arizona State University, pours sucrose gradients (a tube with dense liquid at the bottom, and less dense liquid at the top), which are used to separate, say, proteins from RNA by spinning them really fast in centrifuges.

Therapies 52

Therapies RNA DNA Engineering 52

Codon

APRIL 2, 2023

A giant dataset about the rat brain, and how neurons connect to each other, was collected and pooled together from 20 different research laboratories. Read A new DNA polymerase variant, called RT-KTq I614Y, can directly detect RNA modifications, including pseudouridine (Ψ) and queuosine (Q). Gene Therapy.

DNA 52

DNA RNA Engineering Licensing 52

The Pharma Data

OCTOBER 25, 2021

Week 48 Data Met Safety and Efficacy Endpoints in Adults with HIV-1 Infection Currently on Antiretroviral Therapy. At 48 weeks, both trials met their primary efficacity endpoint of chance of actors with HIV-1 RNA situations ? Merck Research Laboratories. “

Treatment 52

Treatment Trials RNA Clinical Trials 52

Codon

APRIL 9, 2023

” — Harold Morowitz 🔥 Ten Amazing Things (that happened this week…) A CAR-T therapy was tested in 27 children with neuroblastomas. Small snippets of double-stranded RNA were sprayed onto hot pepper plants to control a pest, called Frankliniella occidentalis. Molecular Therapy. Gene Therapy.

Therapies 52

Therapies RNA Engineering Vaccine 52

The Pharma Data

DECEMBER 8, 2020

Curexsys is founded by Herbert Stadler, a serial biotech entrepreneur, and Jens Gruber, a former group leader of Medical RNA Biology who is going to lead Curexsys as Chief Scientific Officer. They contain proteins, nucleic acids and metabolites, which carry information from secreting to receiving cells.

Laboratories 52

Laboratories Disease Production Therapies 52

The Pharma Data

NOVEMBER 18, 2021

As previously reported, at 48 weeks, both trials met their primary efficacy endpoint of percentage of participants with HIV-1 RNA levels ?50 All clinical studies provide important learnings to help us in the fight against HIV, and we are grateful to the patients and investigators for their contributions.”.

Clinical Trials 52

Clinical Trials Treatment Trials RNA 52

The Pharma Data

FEBRUARY 25, 2022

Pfizer’s COVID-19 therapy Paxlovid, for example, targets Mpro. Pell ABSL-3 Laboratory for Advanced Biological Research at Penn State, and discovered that eight of them had dose-dependent antiviral activities against SARS-CoV-2. ” The findings published today (Feb. 25) in the journal Communications Biology.

FDA Approval 52

FDA Approval FDA Molecular Biology Virus 52

The Pharma Data

SEPTEMBER 28, 2021

By testing only what’s medically relevant and necessary, we enable targeted therapy, avoid misuse of antibiotics and reduce unnecessary intervention.”. the power to consolidate and automate a wider array of testing on one platform helps to extend operational efficiency, maximise laboratory space and minimise capital investment.

Marketing 52

Marketing Virus DNA Disease 52

The Pharma Data

OCTOBER 30, 2020

Veklury is a nucleotide analog RNA polymerase inhibitor and works by stopping replication of SARS-CoV-2. kg to less than 40 kg with suspected or laboratory confirmed COVID-19 for whom use of an intravenous (IV) agent is clinically appropriate. It is widely available in U.S. hospitals.

FDA Approval 52

FDA Approval Hospitals Treatment FDA 52

Codon

JUNE 26, 2023

Since the last Codon Digest, I’ve published: Reasons to Be Grateful for Biotechnology (with Avadhoot Jadhav) AAV Foundations (Part I) An overview of AAV-based gene therapies, how they get made, and where they go wrong. Nevertheless, phage therapy did have a stint in the U.S. I’ll post an update soon. coli in mice.

DNA 52

DNA Therapies Engineering Clinical Trials 52

Codon

JUNE 26, 2023

Since the last Codon Digest, I’ve published: Reasons to Be Grateful for Biotechnology (with Avadhoot Jadhav) AAV Foundations (Part I) An overview of AAV-based gene therapies, how they get made, and where they go wrong. Nevertheless, phage therapy did have a stint in the U.S. I’ll post an update soon. coli in mice.

DNA 52

DNA Therapies Engineering Clinical Trials 52

The Pharma Data

APRIL 26, 2022

The expanded indication for Veklury for the treatment of children is a testament to the safety, tolerability and efficacy profile of this therapy, which has remained the foundational antiviral for COVID-19 treatment,” said Merdad Parsey, MD, PhD, Chief Medical Officer, Gilead Sciences.

Treatment 52

Treatment Hospitals Science Licensing 52

Codon

APRIL 23, 2023

Most people would take the two CRISPR gene-editing components (a Cas9 protein and guide RNA), package them up inside of a virus, and then inject the viruses into the skulls of mice. Read A gene therapy for Duchenne muscular dystrophy, developed by a company called Sarepta, is expected to be approved (or rejected) by the F.D.A.

Vaccine 52

Vaccine DNA Virus Engineering 52

Codon

APRIL 23, 2023

Most people would take the two CRISPR gene-editing components (a Cas9 protein and guide RNA), package them up inside of a virus, and then inject the viruses into the skulls of mice. Read A gene therapy for Duchenne muscular dystrophy, developed by a company called Sarepta, is expected to be approved (or rejected) by the F.D.A.

Vaccine 52

Vaccine DNA Virus Engineering 52

PLOS: DNA Science

APRIL 20, 2023

Alfred Hershey (1908–1997) and Martha Chase (1927–2003), at the Cold Spring Harbor Laboratory on Long Island, finally ruled out protein as the genetic material. In 1909, Russian-American biochemist Phoebus Levene identified the 5-carbon sugar ribose in RNA, and then deoxyribose in DNA.

DNA 52

DNA Virus Science RNA 52

Codon

SEPTEMBER 22, 2024

How Eggs and Sperm Get Made In the laboratory where I work, just down the street from the bustling Fenway Park in Boston, there is an incubator with a 96-well plate inside. Up until now, the only way to achieve meiosis in the laboratory has been to culture germ cells, taken from a mouse, together with fetal ovarian supporting cells.

DNA 116

DNA Laboratories Regulations Therapies 116

Codon

MAY 15, 2023

2/ Monkey Embryos Grow to 25 Days Two Chinese groups published papers in Cell , back-to-back, that show how cultured monkey embryos can be grown in the laboratory, for up to 25 days, in 3-D gels. This paper paves the way for off-the-shelf cell therapies that don’t require immunosuppressive drugs. Gene Therapy. link ) An E.

DNA 52

DNA Bioinformatics Engineering Virus 52

Codon

MAY 15, 2023

2/ Monkey Embryos Grow to 25 Days Two Chinese groups published papers in Cell , back-to-back, that show how cultured monkey embryos can be grown in the laboratory, for up to 25 days, in 3-D gels. This paper paves the way for off-the-shelf cell therapies that don’t require immunosuppressive drugs. Gene Therapy. link ) An E.

DNA 52

DNA Bioinformatics Engineering Virus 52

The Pharma Data

JULY 18, 2021

That’s why we set out to develop a conjugate vaccine that includes pneumococcal serotypes that pose the greatest threat and elicits a strong immune response to each serotype covered,” said Dr. Roy Baynes, senior vice president and head of global clinical development, chief medical officer, Merck Research Laboratories.

Disease 52

Disease Immune Response Vaccine FDA Approval 52

PPD

NOVEMBER 8, 2022

The approval of mRNA-based COVID-19 vaccines was a key step in reifying a mainstream future for novel therapeutic platforms, including other nucleic acid therapeutics and cell and gene therapies. Together, we can capitalize on these opportunities to expertly advance your therapies into the new year.

Clinical Trials 52

Clinical Trials Trials Drug Development Clinical Research 52

The Pharma Data

MARCH 22, 2022

During the pandemic, Pfizer will offer its oral therapy through a tiered pricing approach, pending country authorization or approval, based on the income level of each country to promote equity of access across the globe. High and upper-middle income countries will pay more than lower income countries.

Treatment 52

Treatment Virus Therapies Hospitals 52

Broad Institute

JANUARY 30, 2024

He spent many school breaks at Argonne National Laboratory where his parents both worked as scientists. The findings could one day lead to a clinical test for sepsis or to therapies that manipulate MS1 cells to improve a patient’s prognosis.

Hospitals 137

Hospitals Virus Medical Schools Science 137

Codon

APRIL 16, 2023

Compact gene editors , called enOsCas12f1 and enRhCas12f1, are smaller than many existing Cas proteins and can thus be more easily delivered in a single virus for gene therapies. Read Development of a translatable gene augmentation therapy for CNGB1-Retinitis Pigmentosa. Molecular Therapy. Molecular Therapy. Occelli L.M.

Engineering 52

Engineering DNA RNA Science 52