Drug Target Review

APRIL 18, 2025

Recent years have witnessed remarkable advancements in cellular therapies, particularly in the development of treatments that harness the power of stem cells and the immune system. At the forefront of these innovations is Dr Thomas Ichim, a distinguished expert in biotechnology and stem cell therapy at Immorta Bio.

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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.

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Codon

JUNE 5, 2025

The treatment, now known as Casgevy, became the first CRISPR-based therapy to gain FDA approval, in 2023. The core of the CRISPR immune response is a guide RNA (gRNA) that binds to a CRISPR-associated (Cas) protein. Dozens more clinical trials, based upon similar gene-editing technologies, are now underway.

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DNA RNA Clinical Trials Engineering 83

Codon

NOVEMBER 3, 2024

The experience showed me that new therapies are needed not only to meet the targets laid out by the End TB Strategy but also to prevent drug resistance from negating the effectiveness of current therapeutics. Subscribe to Asimov Press. tuberculosis. A Peace Corps Volunteer administers the BCG vaccine to a six-year-old Korean boy.

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Broad Institute

FEBRUARY 26, 2025

These gene signatures provide a roadmap that the field can use to study myeloid cells and how they impact the way brain tumors respond to therapy, Bernstein said. New findings from researchers at the Broad Institute of MIT and Harvard and the Dana-Farber Cancer Institute (DFCI) could help make immunotherapies for brain cancer more effective.

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Broad Institute

JUNE 25, 2025

The scientists suggest that new therapies that target these genes could directly address fibrosis and potentially be more effective for this complication than existing drugs, which are primarily focused on reducing inflammation. Drugs targeting strictures have failed in clinical trials, suggesting the need for a deeper look.

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Disease Medical Schools RNA Molecular Biology 111

BioPharma Drive: Drug Pricing

JUNE 27, 2025

Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. BCMA and CD19 are the protein targets of the seven approved CAR-T therapies. You can unsubscribe at anytime.

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Therapies FDA Treatment Licensing 178

Conversations in Drug Development Trends

OCTOBER 14, 2024

Fortunately, advances in clinical research are providing hope for better treatments and outcomes. 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.

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Drug Target Review

FEBRUARY 9, 2024

What key findings about stem cell behaviour, differentiation and integration within host tissues impact the development of stem cell therapies? Additionally, ADSCs possess immunomodulatory properties, enabling them to modulate immune responses and promote tissue healing.

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Drug Target Review

NOVEMBER 17, 2023

NK cells are among the front line of protection from infected and abnormal cells as part of the ‘innate immune response’. They recognise ‘cell stress molecules’ on the surface of infected, old, injured and cancerous cells without the need for complex pre-stimulation signals of the adaptive immune system (eg, T cells).

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Drug Target Review

NOVEMBER 17, 2023

NK cells are among the front line of protection from infected and abnormal cells as part of the ‘innate immune response’. They recognise ‘cell stress molecules’ on the surface of infected, old, injured and cancerous cells without the need for complex pre-stimulation signals of the adaptive immune system (eg, T cells).

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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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Therapies FDA Clinical Trials Disease 101

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. (You can download a pdf copy of the blog here.) appeared first on Plenge Gen @rplenge.

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Immune Response Disease Clinical Trials Drugs 52

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

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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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

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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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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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.

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Clinical Trials Vaccine Trials Virus 52

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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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.

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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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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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Conversations in Drug Development Trends

JUNE 21, 2024

The 2024 ASCO Annual Meeting from the American Society of Clinical Oncology is a fantastic platform for clinical researchers to discuss the latest advancements and challenges in oncology research. 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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The Pharma Data

JANUARY 17, 2021

LONDON–( BUSINESS WIRE )– Ixaka Ltd , an integrated cell and gene therapy company focused on the natural power of the body to cure disease, launches today. The new business will continue to develop Ixaka’s proprietary technologies – concentrated multi-cell therapies (MCTs) and targeted nanoparticle (TNP) therapeutics.

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The Pharma Data

JUNE 17, 2022

In a Phase 2/3 clinical trial, the Pfizer-BioNTech COVID-19 Vaccine elicited a strong immune response in this age group Three 3-µg doses had favorable safety profile similar to placebo in young children ages 6 months through 4 years in Phase 2/3 clinical trial Pfizer-BioNTech COVID-19 Vaccine now authorized in the U.S.

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The Pharma Data

JANUARY 5, 2021

5, 2021 — Stem cells derived from a baby’s umbilical cord can help save the lives of the sickest COVID-19 patients, results from a small new clinical trial suggest. The therapy also proved safe, with no infusion-related serious adverse events. ” Results from the new trial were published Jan.

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The Pharma Data

JANUARY 12, 2021

The Israeli-based biotech is dedicated to developing innovative therapies for cancer, based on its proprietary research of newly discovered immune-checkpoints and immune-evasion mechanisms. Ben-Moshe told BioSpace that BND-22 differs from traditional immunotherapy in that it can elicit a more total immune response. . “It

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Immune Response Clinical Trials Licensing Licensing 52

Olympian Clinical Research

MAY 6, 2024

As medical understanding grew, so did the sophistication of the therapies. These targeted therapies revolutionized treatment, offering relief to severe cases by specifically addressing the underlying immune responses causing the disease.

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Treatment Small Molecule Clinical Research Immune Response 52

Olympian Clinical Research

NOVEMBER 30, 2023

Topical Treatments and Light Therapy Initially, treatments focused on topical applications and light therapy. Targeted Therapies Recent developments have introduced targeted therapies that focus on the underlying causes of vitiligo, such as immune system modulation.

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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. Clover intends to initiate a separate pivotal clinical trial of the S-Trimer vaccine candidate in combination with Dynavax’s advanced CpG 1018 adjuvant plus alum in the first half of 2021.

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Drug Target Review

OCTOBER 17, 2024

It is not known whether CD24 on cancer cells has a unique epitope that can be specifically targeted for cancer therapy. The broad regulatory function of this pathway suggests that we do not need to limit our clinical strategy until more information is gathered in the FIH trials. Biomarkers are the holy grail of clinical trials.

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The Pharma Data

JANUARY 6, 2021

Quantitative imaging of CD8 T cells enables quicker identification of drug efficacy, therefore potentially reducing the length of clinical trials, reducing costs and helping new therapies to advance to market faster, which ultimately will improve treatment and care of cancer patients. About ImaginAb. ImaginAb Inc.

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The Pharma Data

JANUARY 3, 2021

Weber is a renowned melanoma specialist and leading immunotherapy translational and clinical scientist. Both will help NexImmune advance current early-stage clinical trials and will guide Company’s translational efforts to develop new immunotherapy products. GAITHERSBURG, Md.,

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The Pharma Data

DECEMBER 10, 2020

The vaccine targeted both the inner nucleocapsid (N) and the outer spike (S) proteins of the virus to maximize the immune response. The study showed this broad immune response led to the complete clearance of the virus in a matter of days after infection of previously-vaccinated primates. link] 1. About ImmunityBio.

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