Drug Target Review

NOVEMBER 29, 2023

Our primary focus is to design and develop RNA therapies for liver diseases. Our team of chemists then uses this information to develop novel therapies. By doing this, we get a superior understanding of which therapies will and will not work in human biology before a clinical trial.

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Therapies Disease RNA Engineering 102

Drug Target Review

NOVEMBER 17, 2023

They do not induce ‘graft versus host’ disease when transplanted for allogeneic therapy and there appears little sign of immune rejection. Peripheral blood NK cells have been used in allogeneic cancer therapies and shown to be safe. NK cells can be readily extracted from umbilical cord blood or peripheral blood of adult donors.

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Therapies Clinical Trials Cell Biology Trials 118

Drug Target Review

JULY 31, 2023

Our inaugural report is a groundbreaking exploration of the remarkable advancements in cell and gene therapy that are revolutionising the field of drug discovery. Expert Insights We are honoured to have collaborated with renowned experts in the field of cell and gene therapy, who have generously shared their invaluable insights.

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Therapies Disease Clinical Development Hospitals 98

FDA Law Blog: Drug Discovery

DECEMBER 7, 2022

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

Therapies 119

Therapies FDA FDA Approval Treatment 119

PLOS: DNA Science

MARCH 21, 2024

The Food and Drug Administration just announced approval of Lenmeldy (atidarsagene autotemcel), a gene therapy to treat the neurological condition metachromatic leukodystrophy (MLD). The history of gene therapy for MLD is compelling – DNA Science covered it for Rare Disease Day in 2021, here. She passed away in 2013.

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Therapies Clinical Trials DNA Trials 98

Drug Target Review

NOVEMBER 17, 2023

They do not induce ‘graft versus host’ disease when transplanted for allogeneic therapy and there appears little sign of immune rejection. Peripheral blood NK cells have been used in allogeneic cancer therapies and shown to be safe. NK cells can be readily extracted from umbilical cord blood or peripheral blood of adult donors.

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Therapies Clinical Trials Immune Response Trials 103

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

thought leadership

JULY 24, 2023

Decentralizing cell & gene therapy generation enables treating physicians to drive the treatment and make informed decisions about options, thus putting the patient first and creating the best possible outcome. Patients have the best outcomes when the treatment is safe, effective and provided at the right time.

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Therapies Treatment 75

Drug Target Review

JUNE 19, 2024

The mission of Lineage Cell Therapeutics is to deliver on some of the early promises of cell therapy. Cell therapy as a concept is a wonderful idea, but many of the early efforts never generated the kind of clinical data that gets people excited and leads to new medicines. Hearing aids also have all sorts of deficits.

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Therapies Small Molecule Pharmaceutical Companies Molecular Biology 84

Drug Target Review

NOVEMBER 23, 2023

This means the neuropeptide network probably uses different strategies to transmit and process information than a more hierarchical wired network does. elegans be applied to the development of targeted therapies for neuropsychiatric conditions like eating disorders, OCD, and PTSD? elegans ?

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Therapies Molecular Biology Laboratories Government 98

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

Conversations in Drug Development Trends

MARCH 12, 2024

Biomarkers provide a rapid way to inform many clinical oncology study development decisions, including study endpoints. They can inform intervention outcomes, serve as study endpoints, and are advantageous across the spectrum of cancer care. What are they, how do they work, and how can they benefit your oncology study?

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Clinical Trials Immune Response Trials Disease 72

FDA Law Blog: Drug Discovery

MARCH 13, 2023

Who better than people living with a condition to inform drug companies, physicians, academics, and the FDA on what it is like to live with their condition, what symptoms most impact their lives, what goes into their decision about whether to participate in a clinical trial, and what kind of treatment effects would be most meaningful to them?

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Therapies Trials FDA Clinical Trials 98

Advarra

FEBRUARY 4, 2025

At a busy research site, a clinical trial is ready to launch but its stuck in limbo over a single paragraph in the informed consent form (ICF). Minor differences in language whether related to costs, state laws or institutional policies can derail timelines, slowing the path to innovative therapies.

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Clinical Trials Trials Compliance Therapies 52

DrugBank

JUNE 13, 2024

  Gene Therapy: Reprogramming the Body's Cellular Code    Gene therapy is an exciting field that treats diseases at their genetic roots.    A key part of gene therapy is efficiently delivering the therapeutic genetic material directly into target cells.

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Therapies Drugs DNA Treatment 74

SCIENMAG: Medicine & Health

JUNE 16, 2023

Breast cancer is a complex disease, and its progression is difficult, yet important, to predict. Credit: UMass Amherst Breast cancer is a complex disease, and its progression is difficult, yet important, to predict.

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Therapies Disease 57

PPD

OCTOBER 12, 2023

Cell and gene therapies (CGTs) are one of the fastest growing areas in human therapeutics. Since chimeric antigen receptor T cell (CAR-T) therapy was first approved in 2017, there has been a marked increase of cell and gene therapy studies resulting in significant changes in the way diseases are treated as well as patient outcomes.

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Laboratories Clinical Trials Protein Expression Pharmacokinetics 52

Drug Target Review

DECEMBER 20, 2024

However, he believes that these fears will dissipate as pharma companies come to realise that AI firms like Unlearn adhere to stringent data control measures, ensuring the ethical use of patient information.

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Clinical Trials Trials Pharmaceuticals Drug Development 128

Chemical Biology and Drug Design

APRIL 1, 2023

The summarized information presented in this review article may help and lead the researchers, scientists/industry persons to use berberine as a promising candidate against cancer. In addition to these actions, Berberine plays a role in, the regulation of reactive oxygen species and inflammatory cytokines in preventing cancer formation.

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Therapies RNA Regulations Research 100

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. Regarding CAR-T cell therapy, could you highlight the promising findings that have been identified in the early stages of drug discovery, as well as potential challenges?

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Therapies Treatment Vaccine FDA Approval 64

ASPET

OCTOBER 8, 2024

Tyrosine kinase inhibitors (TKIs) targeting the BCR-ABL fusion protein, such as imatinib (Gleevec), have revolutionized targeted cancer therapies. However, drug resistance and side effects, particularly those affecting hemostasis, continue to pose significant challenges for TKI therapies.

Small Molecule 100

Small Molecule Therapies Drugs 100

The Premier Consulting Blog

APRIL 18, 2024

Designing an adequate nonclinical program to support the safety of cell or gene therapy products is not always straightforward. The cell or gene therapy should be pharmacologically active in the animal model. In the end, the data generated needs to provide biologically relevant information about how the therapy will behave in humans.

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Therapies Small Molecule Production Clinical Trials 52

Drug Target Review

JANUARY 14, 2025

provide more general information. ChatGPT can also aid in deciding which visualisation options are available and what information each will provide, based on the data and your research question. By posing questions such as, ‘Which database should I use to search for data on the effects of longevity drugs in rodents?’

Drugs 69

Drugs Protein Expression Science Research 69

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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Therapies Clinical Trials Small Molecule Immune Response 52

Advarra

NOVEMBER 14, 2024

The draft guidance highlights several key points pertinent to international trials: Diversity of expertise: KOLs with varied backgrounds and expertise enhances the DMC’s ability to address complex clinical and safety issues, and is crucial for interpreting data accurately and making informed decisions. local standards of care).

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Clinical Trials Trials International Clinical Research 97

thought leadership

JULY 2, 2024

Change in the Submission of the Summary Notification Information Format The Summary Notification Information Format Form As of January 31, 2023, Sponsors are required to submit a Clinical Trial Application (CTA) in the EU through the Clinical Trial Information System (CTIS).

Trials 52

Trials Therapies Clinical Trials Regulations 52

thought leadership

JULY 1, 2024

Although this regulation applies to CTAs for gene therapy studies, it does not cover the additional requirement for a Genetically Modified Organism (GMO) application for such studies.

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Therapies Trials Clinical Trials Regulations 52

Drug Target Review

JULY 11, 2024

To date, there are less than 30 known surface proteins that form the basis of all approved cancer-targeted therapies of all modalities, including antibody-drug conjugates (ADCs), T-cell engagers, CAR T-cells and radiopharmaceuticals. These could be targets for antibody-drug conjugates (ADCs), bi-specific antibodies, or other therapies.

Therapies 56

Therapies RNA Pharma Companies Disease 56

FDA Law Blog: Drug Discovery

NOVEMBER 2, 2023

Lewis, Senior Regulatory Device & Biologics Expert — On October 20, 2023, FDA announced the availability of the final guidance authored by CBER titled “Voluntary Consensus Standards Recognition Program for Regenerative Medicine Therapies.” It finalized a draft guidance published in 2022.

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Therapies FDA Production Regulations 59

PPD

DECEMBER 16, 2024

Internal costs: The complexity of protocol designs a necessity for innovative therapies often requires more diverse patient populations, more extensive data collection and sophisticated trial methodologies, all of which demand higher financial outlays. Nearly 39% of sponsors cite these costs as primarily driven by complex protocols.

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Drug Development Clinical Trials Compliance Trials 52

FDA Law Blog: Drug Discovery

MARCH 26, 2024

Sasinowski — On March 21st, FDA announced the approval of the first nonsteroidal therapy for the treatment of Duchenne Muscular Dystrophy (DMD) (FDA press release available here ). It was also the first trial to be run with a placebo control over 18 months, whereas other DMD therapies have been tested in pivotal trials of 1 year or less.

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FDA Approval Therapies FDA Trials 59

ASPET

MARCH 20, 2024

Research on animal models of hypothyroidism has revealed critical information on how deficiency of thyroid hormones impacts the electrophysiological and molecular functions of the brain, which lead to the well-known cognitive impairment in untreated hypothyroid patients.

Therapies 100

Therapies Research 100

KIF1A

JANUARY 27, 2024

Rare Roundup KRIBB develops new gene therapy candidate for hereditary spastic paraplegia This week we’re flipping the format and starting with our Rare Roundup, after families contacted us with this article about a gene therapy candidate for hereditary spastic paraplegia.

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Therapies Small Molecule DNA Treatment 111

Drug Hunter

MAY 21, 2023

These findings highlight the versatility of DCAF1 for TPD and offer a new avenue for developing therapies for diseases where catalytic inhibition is not an option. Drug Hunter Premium is drug discovery, distilled, so you can quickly catch up and make informed decisions based on industry examples. Get ahead now by requesting a trial.

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Targeted Protein Degradation Trials Therapies Disease 131

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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Therapies Virus DNA Compliance 52

Alta Sciences

APRIL 16, 2024

Inside The Altascientist: The Benefits of PCR for Your Gene Therapy Programs pmjackson Tue, 04/16/2024 - 19:04 The gene therapy landscape continues to accelerate in preclinical and clinical research, with programs constantly in development for targeted, personalized medicines. But how are PCR analysis techniques applied?

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Therapies DNA RNA Laboratories 52

Broad Institute

SEPTEMBER 23, 2024

New approach may yield modified messenger RNAs for treating a wide range of conditions By Ari Navetta September 23, 2024 Breadcrumb Home New approach may yield modified messenger RNAs for treating a wide range of conditions The LEGO framework could help scientists create mRNA therapies that last longer and require smaller doses.

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RNA Vaccine Small Molecule Therapies 134