Drug Target Review

OCTOBER 26, 2023

Genome engineering and gene therapies that manipulate DNA sequences in cells have driven a biotechnological revolution over the past decade. 1 Adeno-associated virus (AAV) vectors are the leading platform for in vivo gene delivery for the treatment of various human diseases. Molecular Therapy 20 , 1831-1832 (2012).

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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. Molecular Therapy-Nucleic Acids.

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Drug Target Review

DECEMBER 5, 2024

Viral vectors have been crucial in transforming the gene therapy landscape due to their natural ability to infect cells. 1 In 2017, the US Food and Drug Administration (FDA) approved the first AAV-based gene replacement therapy (Luxturna), for Leber congenital amaurosis type 2.

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Drug Target Review

APRIL 17, 2024

HLA are the critical molecules that our immune systems use to present peptides to T cells and facilitate recognition and killing in immune responses to pathogens. HLA-II presents peptides to CD4 T cells thought to be important for indirectly helping immune responses and facilitating antibody production.

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PLOS: DNA Science

FEBRUARY 8, 2024

The molecular tools of CRISPR were borrowed and developed from the natural immune response of bacteria to viruses – bacteriophages – that infect them. CRISPR may make sense for replacing a single mutant gene, like the one behind sickle cell disease, for which a CRISPR-based gene therapy was recently approved.

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Drug Target Review

SEPTEMBER 11, 2023

This sugar-coated mucin binding to checkpoint receptors signals that the cancer cell poses no threat, consequently blocking the immune response. Tender elaborated that these results in immune cells ignoring the cancer rather than eliminating it, as they normally would.

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Codon

SEPTEMBER 29, 2024

It suggested that, in the future, certain classes of drugs, specifically genetic therapies, could be screened entirely virtually via models of life. Models trained on nucleotide data were ideal for understanding how genetic therapies altered cellular dynamics, so they powered the genetic editing revolution.

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Alta Sciences

JANUARY 11, 2024

It is estimated that there are over 6,000 monogenic diseases, affecting over 350 million people worldwide; for these diseases, cell and gene therapy may provide hope for a cure. However, there are significant challenges associated with the successful development of these complex, leading-edge therapies. what is the altascientist?

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The Pharma Data

JANUARY 18, 2021

Use of AAVnerGene capsid library provided to Neurophth for ophthalmic gene therapy. Next Generation AAVs enhance gene therapies by increasing transduction efficiency and specificity while reducing immune responses and cost. gene therapy company (hereafter Neurophth), and AAVnerGene Inc. ,

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Drug Target Review

JULY 7, 2023

Antibodies protect us by attaching to molecules on those invaders (known as antigens) and triggering our body’s natural immune response to destroy them. This can create an abnormal immune response that attacks the cells of our bodies and contributes to the development of autoantibody diseases.

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Advarra

JANUARY 11, 2024

The applications of mRNA-based therapies in cancer research represent one of the next groundbreaking steps toward improved cancer treatments. Adoptive T Cell therapies, therapeutic antibodies, and immunomodulatory proteins represent just some of the potentially beneficial treatment strategies for successful mRNA cancer trials.

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The Pharma Data

MARCH 3, 2022

If approved by the FDA, this maternal immunization has the potential to be the first vaccine candidate to help protect infants in their vulnerable first months of life from disease caused by this highly-contagious virus. Burden of RSV RSV is a contagious virus and a common cause of respiratory illness. Pfizer Inc.

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Drug Target Review

JULY 17, 2024

How does GigaGen’s single-cell discovery and development platform differ from traditional methods of producing polyclonal antibody therapies? Current methods for producing pAb therapies rely on regular plasma donations for their development.

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The Pharma Data

SEPTEMBER 28, 2020

Australian biotech Ena Respiratory has revealed new data demonstrating that its novel nasal spray almost halted replication of the SARS-CoV-2 in animals when used as a prophylactic, showing potential to improve immune response when used alongside COVID-19 vaccines. . We’ve been amazed with just how effective our treatment has been.

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Drug Target Review

JANUARY 15, 2024

Each T cell expresses a TCR specific for an HLA‑presented peptide signal, and when that signal is detected a T cell expressing the relevant TCR can launch a complex array of immune effector responses that can suppress or destroy tumours or virus-infected cells. Engineering soluble T-cell receptors for therapy.

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Vial

DECEMBER 11, 2023

Hepatitis C virus (HCV) infection is a global health problem with complications that place a significant economic burden on national health systems. DAAs and other HCV therapies in the primary stage of infection can lead to good patient outcomes, provided infection is diagnosed early.

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The Pharma Data

AUGUST 7, 2020

CoV2373 has shown that the therapy generated “robust antibody responses” when used with or without the company’s Matrix?M Furthermore, many participants also generated wild-type virus neutralising antibodies, with both these kinds of responses stacking up favourably against responses seen in patients hospitalised with COVID-19.

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The Pharma Data

JANUARY 31, 2021

(Nasdaq: VIRI), a biotechnology company focused on advancing novel antiviral therapies to treat diseases associated with a viral triggered abnormal immune response, announced today that it is entering a collaboration with Dr. Michael Camilleri of the Mayo Clinic to explore the role of antiviral therapy in managing Irritable Bowel Syndrome (IBS).

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The Pharma Data

MARCH 8, 2022

SARS-CoV-2 viruses can hide from recognition by the immune system. However, the antiviral immune receptor RIG-I can be stimulated, which improves protection against lethal SARS-CoV-2 infections. The study was published online in advance in the journal Molecular Therapy – Nucleic Acids and is now available in the final version.

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The Pharma Data

DECEMBER 3, 2020

Strong Th1 cell-mediated immune responses were also observed for the vaccine candidates with either adjuvant. We are encouraged by the high level of neutralizing antibodies in combination with the strong Th1 response which we believe could play an important role in controlling infection.

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The Pharma Data

NOVEMBER 20, 2020

NASDAQ: REGN) today announced that the antibody cocktail casirivimab and imdevimab administered together (formerly known as REGN-COV2 or REGEN-COV2), a therapy currently being investigated for use in COVID-19 , has received Emergency Use Authorization (EUA) from the U.S. TARRYTOWN, N.Y., November 21, 2020 – Regeneron Pharmaceuticals, Inc.

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Codon

JULY 27, 2023

Their cone cells, which are responsible for color vision, don’t work like normal. But what happens if you restore these cone cells, using gene therapy? approved a gene therapy for hemophilia A for the first time. The therapy works by replacing beta-cells with “fresh” cells taken from a, err…dead person.

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Codon

JULY 27, 2023

Their cone cells, which are responsible for color vision, don’t work like normal. But what happens if you restore these cone cells, using gene therapy? approved a gene therapy for hemophilia A for the first time. The therapy works by replacing beta-cells with “fresh” cells taken from a, err…dead person.

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Codon

APRIL 9, 2023

” — Harold Morowitz 🔥 Ten Amazing Things (that happened this week…) A CAR-T therapy was tested in 27 children with neuroblastomas. Read Rational design of a highly immunogenic prefusion-stabilized F glycoprotein antigen for a respiratory syncytial virus vaccine. Molecular Therapy. Gene Therapy.

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The Pharma Data

SEPTEMBER 30, 2021

That’s how easy it’s for SARS-CoV-2, the virus that causes COVID-19, to enter your nose. Anderson Professor of Chemical and Biomolecular Engineering, and his colleagues, are reporting in iScience the event of an intranasal subunit vaccine that gives durable local immunity against inhaled pathogens. Breathe in, exhale.

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The Pharma Data

DECEMBER 20, 2020

BT-001 is a best-in-class oncolytic Vaccinia virus. platform and its patented large-capacity VV cop TK-RR- oncolytic virus, which has been engineered to encode both a Treg-depleting human recombinant anti-CTLA4 antibody generated by BioInvent’s proprietary n-CoDeR ® /F.I.R.S.T LUND, Sweden and STRASBOURG, France , Dec.

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The Pharma Data

JUNE 25, 2021

The outcome confirms that only one single case was attributable to the original SARS-CoV-2 virus. “In addition, the variant-rich environment underlines the importance of developing next-generation vaccines as new virus variants continue to emerge.” More than half of the cases (57%) were caused by Variants of Concern.

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The Pharma Data

JANUARY 11, 2021

a San Diego-based biotechnology company with an array of technology platforms for antibody discovery and optimization, and novel NK and T cell engager generation, today announced licensing of a panel of its anti-SARS-CoV-2 antibody clones to IGM Biosciences for COVID-19 therapy development.

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NIH Director's Blog: Drug Development

FEBRUARY 1, 2022

The NIH continues to support the development of some very innovative therapies to control SARS-CoV-2, the coronavirus that causes COVID-19. Though more study is needed, the researchers say the decoy therapy could potentially be delivered directly to the lungs through an inhaler and used alone or in combination with other COVID-19 treatments.

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NIH Director's Blog: Drug Development

MAY 21, 2020

B38 blocks SARS-CoV-2 from binding to the ACE2 receptor (light pink) of a human cell, ACE2 is what the virus uses to infect cells. Just one infusion of this combination antibody therapy lowered the amount of viral genetic material in the animals’ lungs by as much as 30 percent compared to the amount in untreated animals.

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The Pharma Data

OCTOBER 28, 2020

“We continue to see the strongest effects in patients who are most at risk for poor outcomes due to high viral load, ineffective antibody immune response at baseline, or pre-existing risk factors. In the overall patient group with detectable virus at baseline, the average daily reduction in viral load through day 7 was a 0.36

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The Pharma Data

JANUARY 11, 2021

Testing Therapies, Antivirals and Vaccines. Pfizer and BioNTech reported that their COVID-19 vaccine appears to be effective against a key mutation in new variants of the SARS-CoV-2 virus that causes COVID-19. SARS-CoV-2, the virus that causes COVID-19, has mutated into several strains. A study published by the U.S.

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The Pharma Data

SEPTEMBER 17, 2020

The new Elecsys Anti-SARS-CoV-2 S test can quantitatively measure the level of antibodies against SARS-CoV-2 in patients who have been exposed to the virus. This is the focus of vaccines in development and convalescent plasma therapy. The test targets antibodies against the spike protein. said Thomas Schinecker, CEO Roche Diagnostics.

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PLOS: DNA Science

JANUARY 4, 2024

The end-of-year FDA approval of the first CRISPR-based therapy , for sickle cell disease, came a mere dozen years after Jennifer Doudna and Emmanuelle Charpentier introduced the technology. The microbes deploy them to dismantle the genetic material of infecting viruses, a little like an immune response.

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The Pharma Data

DECEMBER 29, 2020

As in earlier outpatient trial, immune status when patients entered the trial was a strong predictor of viral load and clinical outcomes. First antibody therapy to demonstrate anti-viral effect in patients hospitalized with COVID-19. TARRYTOWN, N.Y. , Regeneron Pharmaceuticals, Inc. futility analysis). . In the U.S.

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The Pharma Data

MARCH 16, 2021

The cobas SARS-CoV-2 Variant Set 1 Test is designed to detect key spike mutations in virus variants associated with increased human-to-human transmission. Among them, mutations E484K, N501Y and del 69-70 are located in the spike protein, the region that enables the virus to attach to and enter the human cell. 2,3 Another variant, B.1.351,

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NIH Director's Blog: Drug Development

FEBRUARY 16, 2021

This improvement stems from several factors, including the FDA’s emergency use authorization (EUA) of a number of therapies found to be safe and effective for COVID-19. ACTIV was founded last April to accelerate drug research that typically requires more than a decade of clinical ups and downs to develop a safe, effective therapy.

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