PLOS: DNA Science

OCTOBER 19, 2023

Last week DNA Science covered a setback in a clinical trial of a gene therapy for Duchenne muscular dystrophy (DMD). Also recently, FDA’s Cellular, Tissue, and Gene Therapies Advisory Committe turned down a stem cell treatment for amyotrophic lateral sclerosis, aka ALS, Lou Gehrig’s disease, or motor neuron disease.

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Conversations in Drug Development Trends

OCTOBER 14, 2024

With the support of global networks like ours at Worldwide Clinical Trials (Worldwide), autoimmune and bone health research is accelerating, providing new solutions to patients around the world. Given the complexity and widespread impact of autoimmune and bone health conditions, developing new therapies is essential.

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Drug Target Review

OCTOBER 2, 2024

Cell and gene therapies (CGTs) have made significant advancements in treating oncological diseases, with therapies like CAR-T cell treatments transforming cancer care. However, cell-based therapies are intended to leverage those healthy cells via transplant to regenerate damaged tissue.

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Drug Target Review

JANUARY 23, 2024

Cell-based therapies, such as tumour-infiltrating lymphocyte (TIL) therapy, have also drawn significant attention following promising results in a melanoma trial. Such approaches apply artificial intelligence (AI) and machine learning (ML) to predict mutations most likely to be good targets for the immune system.

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Drug Target Review

DECEMBER 13, 2024

2 Unmet needs in lung cancer treatment Recent decades have seen significant advancements in lung cancer treatment, especially with the introduction of targeted therapies and immunotherapies, which have notably improved survival rates. These can include anti-tubulin compounds, potentially reducing side effects and improving quality of life.

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Therapies Treatment Clinical Trials Drugs 98

Plenge Gen

APRIL 7, 2020

Repurposing of approved therapies is the fastest way to impact patients today, as these medicines have regulatory approval to enable investigator-initiated trials and have a manufacturing process to ensure drug supply. Pharmacologic intervention has the opportunity to impact disease progression in the SARS-CoV-2 / COVID-19 crisis.

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The Pharma Data

AUGUST 16, 2021

Better activation of innate and adaptive immune responses was achieved with CV2CoV, resulting in faster response onset, higher titers of antibodies, and stronger memory B and T cell activation as compared to the first-generation candidate, CVnCoV. “In Induction of innate immunity was investigated via specific cytokine markers.

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DrugBank

MARCH 12, 2025

  The Expanding Role of mRNA in Cancer Therapy One of the most exciting applications of mRNA therapeutics lies in cancer treatment, where leveraging the immune system to target tumors offers a novel approach. This process triggers a robust immune response, enabling the immune system to recognize and attack cancer cells.

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Drug Target Review

MARCH 1, 2024

How significant are the results in terms of advancing TCR-T cell therapy and AMP immunotherapy for the treatment of solid tumours? We have already applied this approach to several important tumour targets where enhancements in TCR-T therapy may lead to important gains in clinical benefit, including mutated KRAS, NY-ESO, and HPV E7 antigens.

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PLOS: DNA Science

OCTOBER 12, 2023

In the final chapter of my 2012 book The Forever Fix: Gene Therapy and the Boy Who Saved It , I predicted that the technology would soon expand well beyond the rare disease world. Gene therapy clearly hasn’t had a major impact on health care, offering extremely expensive treatments for a few individuals with rare diseases.

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Broad Institute

OCTOBER 11, 2023

His mother had a presentation of the disease that suggested her immune system was already on the job. But immunotherapy was not yet widely used and had not been applied clinically to Merkel cell carcinoma, so she received traditional chemotherapy and radiation therapy, suffering life-threatening complications along the way.

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Drug Target Review

FEBRUARY 28, 2024

Over the past 25 years, T-cell therapies have gained significant ground in the treatment of cancer. Preclinical research on γδ T cells has made great strides since the cells were first identified in the 1980s, with γδ T-cell therapies from several companies, including IN8bio, now in or nearing clinical trials for various cancers.

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Therapies Engineering Molecular Biology Science 105

Alta Sciences

FEBRUARY 19, 2025

These interactions may disrupt protein function, trigger immune responses, or contribute to other toxicological risks. to ligands or linkers) may elicit immune responses. An oligonucleotide is a short strand of nucleotides, the building blocks of DNA or RNA, used in genetic research and therapy.

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Drug Development Clinical Pharmacology Immune Response Drugs 52

Drug Target Review

JANUARY 15, 2024

Onchilles Pharma has translated this breakthrough research into a proprietary set of therapeutics and plans to start first-in-human clinical trials for its first programme, N17350, in 2024 in skin cancers, head and neck cancer, and triple-negative breast cancer. The concept of the ‘abscopal effect’ in the 4T1 tumour model is intriguing.

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Treatment Immune Response Therapies Clinical Trials 105

Drug Target Review

JUNE 6, 2023

We are in an era of immuno-oncology (IO) revolution with many approved therapies now available to treat a broad range of cancers. Generally, IO has been focused on harnessing the anti-tumour activity of certain cancer-fighting T-cells , a key cell type involved in the adaptive immune defense system.

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Engineering Immune Response Biochemical Pharmacology Clinical Trials 98

Drug Target Review

FEBRUARY 21, 2024

Stem cell transplants have saved patients’ lives time and time again, which led us to launch our own Stem Cell Transplant and Cellular Therapy Program. Stem cell transplants primarily help the immune response through the “graft-versus-leukaemia effect,” and we have to manage the “graft versus host effect.”

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The Premier Consulting Blog

APRIL 18, 2024

Development of cell and gene therapies is growing rapidly, given the major advances in genomic technologies and increasing scientific understanding of genetic regulation and immunology. This blog will outline three characteristics of a successful nonclinical program to support entry into clinical trials for a cell or gene therapy product.

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SugarCone Biotech

JUNE 10, 2024

Targeting 4-1BB remained of interest to the immuno-oncology field as cell culture experiments and tumor models in mice suggested that robust anti-tumor immune responses could be triggered by anti-4-1BB antibody therapies. This is the T cell type most closely associated with anti-tumor immune responses.

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Immune Response Production Regulations Clinical Trials 88

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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Vaccine Therapies Clinical Trials Small Molecule 52

Codon

JULY 14, 2024

While this departs from the reductionist, single-component therapies that have dominated transfusion medicine since World War II, emerging data underscores that whole blood transfusions—blood with all its parts—yield better outcomes following severe blood loss than transfusions involving discrete blood components. Always free.

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DrugBank

MAY 6, 2024

Unlike conventional chemical-based medicines, biologics offer targeted therapies derived from living cells, which can result in more effective and personalized treatments. A Wave of Innovations The year 2024 has already proven to be significant for the field of biologics, witnessing a notable increase in FDA approvals.

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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. 9 In addition, host immune responses further add to the complexity of developing cell-specific AAV capsids for clinical applications. Molecular Therapy 20 , 1831-1832 (2012).

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Engineering Virus Therapies Immune Response 105

The Pharma Data

APRIL 15, 2022

NYSE: PFE) and BioNTech SE (Nasdaq: BNTX) today announced positive results from a Phase 2/3 clinical trial evaluating the safety, tolerability and immunogenicity of a 10-µg booster (third) dose of the Pfizer-BioNTech COVID-19 vaccine in healthy children 5 through 11 years of age. About the Phase 1/2/3 Trial in Children. Pfizer Inc.

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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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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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Drug Target Review

NOVEMBER 6, 2023

2 It is this complexity that necessitates powerful, targeted combination therapies. 4, 5 More recently, new combination regimens have emerged that incorporate targeted therapies to treat a variety of blood cancers, including multiple myeloma (MM), chronic lymphocytic leukaemia (CLL) and acute myelogenous leukaemia (AML).

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Small Molecule Therapies Treatment Disease 111

Vial

DECEMBER 11, 2023

In the decades since its identification in 1989, advances in clinical research for new Hepatitis C therapies and the introduction of direct-acting antiviral agents (DAAs) have led to viral eradication in more than 98% of patients, resulting in HCV crossing from manageable to being the first curable , chronic viral infection. Zhao et al.

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The Pharma Data

JUNE 26, 2021

BioNTech SE (NASDAQ: BNTX, “BioNTech” or “the Company”), announced that the first patient has been treated in its BNT111 Phase 2 cancer vaccine trial (2020-002195-12; NCT04526899). The trial is enrolling a total of 120 patients and will evaluate the effects of the combination as well as single agents alone.

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Clinical Trials Trials Immune Response Vaccine 52

The Pharma Data

OCTOBER 14, 2021

Biogen today announced results of a new analysis of immune response to the COVID-19 vaccine among people with multiple sclerosis (MS). Biogen today announced results of a new analysis of immune response to the COVID-19 vaccine among people with multiple sclerosis (MS). Using data from the MS PATHS network in the U.S.,

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Vaccine Therapies Immune Response Disease 52

Broad Institute

OCTOBER 4, 2023

Cancer immunotherapy drugs called PD-1 inhibitors are widely used to stimulate the immune system to fight cancer, but many patients either don’t respond or develop resistance to them. A new small-molecule drug candidate being tested in an early-stage clinical trial aims to improve patient responses to immunotherapy.

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Conversations in Drug Development Trends

JUNE 21, 2024

Worldwide Clinical Trials attends each year to hear from the research community, connect with our sponsors and sites, and explore potential partnerships to drive forward novel treatments. Previously, T-cell therapies had been primarily used on liquid tumors, as the T-cells were unable to penetrate the solid tumor environment.

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Drug Target Review

SEPTEMBER 9, 2024

Additionally, ‘1104 increases the number of activated regulatory T and B cells, which help modulate the immune response and maintain immune tolerance, preventing the immune system from entering overdrive. As such, ‘1104 uniquely affects both the effector and regulatory arms of the immune response.

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Disease Immune Response Clinical Trials Treatment 64

The Pharma Data

MARCH 25, 2021

today announced primary results from the Phase 2/3 RELATIVITY-047 (CA224-047) trial evaluating the fixed-dose combination of relatlimab, an anti-LAG-3 antibody, and Opdivo (nivolumab) versus Opdivo alone in patients with previously untreated metastatic or unresectable melanoma. About RELATIVITY-047 (CA224-047).

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Trials Clinical Trials Immune Response Therapies 52

The Pharma Data

JUNE 25, 2021

To identify strains causing COVID-19 infections within the trial, sequencing of virus variants has so far been performed on 474 COVID-19 cases, of which 124 fulfilled adjudication criteria and were included in the present efficacy analysis. Phase 1 and 2a clinical trials of CVnCoV began in June and September 2020, respectively.

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Vaccine Trials Immune Response Virus 52

The Pharma Data

NOVEMBER 12, 2020

12 November 2020 — The CALAVI Phase II trials for Calquence (acalabrutinib) in patients hospitalised with respiratory symptoms of COVID-19 did not meet the primary efficacy endpoint. No new safety signal for Calquence was observed in the trials. No new safety signal for Calquence was observed in the trials.

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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. The average pass rate of phase I trials went up by 5 percent. Existing preclinical studies were halted based on suggested toxicological concerns the model raised.

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The Pharma Data

JANUARY 31, 2021

Adjuvanted S-Trimer COVID-19 vaccine candidates demonstrated favorable safety and tolerability profiles and strong neutralizing immune responses in a phase 1 trial. The phase 1 clinical trial was a randomized, double-blind and placebo-controlled study in 150 adult and elderly participants. About Dynavax.

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