Drug Discovery World

SEPTEMBER 6, 2022

DDW Editor Reece Armstrong looks at the cell and gene therapy landscape, examining the challenges facing developers and the trends we can expect to see throughout the year. . There’s no doubt that cell and gene therapies present some of the most exciting opportunities for emerging drugs. million price point per dose.

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Drug Discovery World

OCTOBER 24, 2022

New research suggests messenger RNA can be effectively used as a ‘universal’ therapy against different coronaviruses. . Rather than messenger RNA as a vaccine, this shows that mRNA can be used as a universal therapy against different coronaviruses,” said Gaurav Sahay, OSU and the Texas Biomedical Research Institute.

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Drug Discovery World

MAY 22, 2023

Cell therapy, RNA technology, antibody drug conjugates and AI/machine learning are among the key areas of investment for pharma, according to a new report. Pharma is also keen to access next generation RNA platforms, such as self-amplifying RNAs derived from the genomes of positive-strand RNA viruses.

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Drug Discovery World

JUNE 22, 2023

The technology enables active loading of adeno-associated virus (AAV) into exosomes followed by their release when the AAV-loaded exosomes are delivered into recipient cells. This is part of our long-term business strategy of leveraging exosome-mediated delivery to create and enable genetic medicines such as gene therapy and genome editors”.

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Drug Discovery World

JANUARY 17, 2023

Oregon State University College of Pharmacy scientists have shown that lipid nanoparticles and messenger RNA could treat blindness associated with a rare genetic condition. . Limitations of AAV gene therapy . Sahay and Ryals have received a $3.2

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Drug Discovery World

NOVEMBER 1, 2022

If further studies verify the results, this could be a new method for targeting specific cell types for a variety of disorders that could be treated with gene therapies. Therapies that don’t precisely target diseased cells can have unintended effects in other healthy cells.” . Splicing-linked expression design.

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Drug Discovery World

MAY 13, 2024

MA: Regarding vaccine development specifically, and the role my team plays in the development of cryogenic electron microscopy (cryo-EM) technology, we see the study and dissemination of virus structures, and the proteins that comprise them, as vital to vaccine design. RA: How reliable are vaccines now for respiratory viruses such as flu?

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The Pharma Data

MARCH 23, 2022

To treat Ebola virus infections, researchers are taking a close look at a key piece of the virus: polymerase. Polymerase is a viral protein that directs how Ebola virus replicates its genome as it infects new hosts. Drugs that target polymerase could potentially treat Ebola virus infections and save lives.

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Drug Discovery World

MAY 23, 2024

Reece Armstrong speaks to Samuel Deutsch , Chief Scientific Officer of Nutcracker Therapeutics about the RNA landscape and how it can benefit the development of immunotherapies. RA: What role does RNA play in the development of effective immunotherapies? SD: This is definitely a challenge.

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Alta Sciences

MARCH 1, 2024

Norbert Makori and Pierre Jolicoeur: The Benefits of Combining Preclinical and Bioanalytical for Your Gene Therapy Studies pmjackson Fri, 03/01/2024 - 22:20 Recent advances in gene therapy have allowed for diseases to be approached differently. q: How can Altasciences support sponsors’ Gene Therapy Programs?

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The Pharma Data

MARCH 9, 2022

Novartis today announced a license option agreement with Voyager Therapeutics, a gene therapy company focused on next-generation adeno-associated virus (AAV) technologies, for three capsids to use in potential gene therapies for neurological diseases, with options to access capsids for two other targets.

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Drug Discovery World

JULY 8, 2024

Beard Distinguished Professor of Experimental Surgery and the Founding Director of the Translational Research Institute at Duke University, explores the rise of RNA therapeutics. However, the RNA landscape has drastically changed in recent years and we now know that the molecule is more than just a transient, linear carrier of information.

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Drug Discovery World

APRIL 30, 2024

Reece Armstrong explores the potential and future opportunities for mRNA-based therapies. Messenger RNA (mRNA) has come into focus within the drug discovery and development as an exciting tool to deliver genetic information. For instance, consider how at the end of 2018, the mRNA therapies market was valued at $3.43

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Drug Discovery World

SEPTEMBER 14, 2023

Pascal Soriot, Chief Executive Officer of AstraZeneca, said: “Each of our non-Covid-19 therapy areas saw double-digit revenue growth, with eight medicines delivering more than $1bn of revenue in the first half, demonstrating the strength of our business.

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The Pharma Data

DECEMBER 5, 2020

5, 2020 — A pair of new gene therapies promise a potentially lasting cure for sickle cell disease by subtly altering the genetic information in patients’ bone marrow cells, researchers report. Both of the new gene therapy studies were published online Dec. SATURDAY, Dec. 5 in the New England Journal of Medicine.

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Drug Discovery World

OCTOBER 4, 2022

This year’s event will cover some of the most exciting advancements in areas spanning screening, automation, high content imaging, disease models, cell and gene therapies and how innovation is being driven through partnerships and collaborations. Cell and gene therapies. In 2021, levels of investment hit an all-time high at $22.7

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The Pharma Data

MARCH 8, 2022

The study was published online in advance in the journal Molecular Therapy – Nucleic Acids and is now available in the final version. The virus carries the information to produce a series of proteins, capable of inhibiting antiviral recognition systems of the infected cell. Camouflage protects virus from immune system.

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Drug Discovery World

AUGUST 10, 2022

But decades of research led to improvements in cancer therapy, centred around the idea that one’s immune system could aid in treatment. Next, the advent of antibody treatments in 1997 created the first targeted cancer therapy 2. Since 2017, five CAR T cell therapies have been approved and have shown incredible success in the clinic.

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Drug Target Review

SEPTEMBER 4, 2023

Infectious agent includes bacteria, virus, fungi, and parasites, and they are capable of causing disease in a living organism. I had earlier proposed the use of Palmatine and Silver Nitrate in the efficacy against the RNA structure of the COVID-19 virus. We discovered it is highly effective and environmentally friendly.

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Advarra

MARCH 31, 2023

The use of engineered genetic materials in clinical trials is rapidly expanding, with potential applications for genetic vaccines, gene-modified cellular therapies, and gene therapies. Gene Delivery Systems Genetic material, in the form of DNA or RNA, does not easily enter cells without the aid of a delivery system.

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The Pharma Data

AUGUST 16, 2021

During challenge with the original SARS-CoV-2 virus, animals vaccinated with CV2CoV were found to be better protected based on highly effective clearance of the virus in the lungs and nasal passages. Clearance of the virus in the lungs and nasal passages of the animals was tested following challenge infection with the original virus.

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The Pharma Data

JANUARY 31, 2021

Similar to other enveloped RNA viruses such as HIV, RSV and Influenza, SARS-CoV-2 is also an RNA virus that has a trimeric spike (S) protein on its viral envelope. FDA approval for prevention of infection caused by all known subtypes of hepatitis B virus in adults age 18 years and older. About Dynavax.

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The Pharma Data

OCTOBER 21, 2020

In the midst of the global pandemic, the analysis found no antiretroviral therapy interruptions across the entirety of the ongoing clinical development programme for long-acting cabotegravir and rilpivirine. Of those participants who transitioned back to injectables, the median duration of oral therapy was 51 days.

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The Pharma Data

DECEMBER 3, 2020

Similar to other enveloped RNA viruses such as HIV, RSV and Influenza, SARS-CoV-2 is also an RNA virus that has a trimeric spike (S) protein on its viral envelope. Additionally, Clover is leveraging its in-house GMP biomanufacturing capabilities to support large-scale production of its biologic therapies.

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Drug Discovery World

AUGUST 1, 2023

1 August was first chosen as World RNA Day in 2018 as a play on AUG (adenine, uracil and guanine), a triple sequence of RNA (called a codon) that initiates protein synthesis by the cell. Since then, it has been observed to publicise the importance of this molecule in the generation of proteins in the body.

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The Pharma Data

NOVEMBER 5, 2020

5 of its ongoing Phase 2 extension study, Study 211, examining vebicorvir (VBR, or ABI-H0731) in patients with hepatitis B virus infection. The company’s other two Phase 2 trials, Study 201 and 202, showed that the addition of VBR to NrtI therapy achieved a quicker, deeper level of viral suppression than with NrtI alone.

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The Pharma Data

JANUARY 31, 2021

” Dr. Richard Hatchett , Chief Executive Officer of CEPI, commented: “To end the acute phase of this pandemic, and to control the virus in the longer term, the world needs multiple safe and effective vaccines which can be deployed in a range of populations and countries. About CpG 1018.

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Drug Discovery World

JULY 29, 2024

There are a number of cell and gene therapies (CGTs) currently being developed to address these increasingly prevalent conditions. Read more: US and UK fast track designations for Parkinson’s gene therapy A natural killer cell therapy NKGen Biotech is one company investigating cell therapies for Alzheimer’s disease.

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The Pharma Data

FEBRUARY 25, 2022

. “There remains an urgent need for SARS-CoV-2 therapeutic agents that target parts of the virus other than the spike protein that are not as likely to evolve.” Pfizer’s COVID-19 therapy Paxlovid, for example, targets Mpro. ” The findings published today (Feb. 25) in the journal Communications Biology. .

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The Pharma Data

JANUARY 21, 2021

The once-a-month regimen was preferred by nine out of 10 patients over the daily oral therapy. While much more remains to be done to make HIV history, today’s milestone reminds us how far medical innovation has come since the first reported cases of the virus almost 40 years ago.”. clinical practices. Most Read Today. Source link.

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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.

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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.

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The Pharma Data

DECEMBER 10, 2020

In the study, immunization with the hAd5-COVID-19 vaccine inhibited SARS-CoV-2 virus replication in 100% (10 of 10) of Rhesus macaques, with a drop in viral replication starting on the first day of vaccine administration, and undetectable viral levels as early as three to five days post-challenge in most of the animals.

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The Pharma Data

MARCH 9, 2021

The study showed the antiviral activity of GSK’254, establishing a relationship between dose and antiviral response, with the 140 mg and 200 mg doses showing the greatest reduction in plasma HIV-1 RNA. These findings were presented today at the (virtual) 2021 Conference on Retroviruses and Opportunistic Infections (CROI). log 10 and -2.0

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The Pharma Data

OCTOBER 19, 2020

. “Innovation in cancer treatment is realized through collaboration, and the physicians and researchers at Emory’s Winship Cancer Institute have consistently shown their commitment to developing novel cancer therapies using this collaborative approach,” said Chadi Nabhan , M.D., Ramalingam , M.D.,

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Broad Institute

MAY 16, 2024

By Allessandra DiCorato May 16, 2024 Credit: Deverman lab Brain vasculature (in blue) surrounded by RNA (in orange) transcribed from the gene delivered to the brain in humanized mice using an engineered AAV targeting the human transferrin receptor.

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Drug Target Review

MARCH 21, 2024

There are a few approved therapies for DMD including four exon-skipping drugs and one AAV-microdystrophin drug, which uses a shortened version of dystrophin. Because mRNA is not a virus, it doesn’t have to meet biosafety requirements for manufacturing and administration that an AAV approach would need.

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