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. Only three of the CAR-NK studies were for the treatment of solid tumours.

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Therapies Clinical Trials Cell Biology Trials 118

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. In 2025, I think well see breakthroughs that allow us to make significant advances in rare diseases, where the data is small.

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Clinical Trials Trials Pharmaceuticals Drug Development 128

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). million price tag for the one-time infusion, and for the older siblings who contributed to developing the gene treatment, but were too sick to receive it.

Therapies 98

Therapies Clinical Trials DNA Trials 98

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.

Therapies 102

Therapies Disease RNA Engineering 102

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.

Therapies 98

Therapies Disease Clinical Development Hospitals 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. 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?

Therapies 65

Therapies Treatment Vaccine FDA Approval 65

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. Only three of the CAR-NK studies were for the treatment of solid tumours. About the authors

Therapies 103

Therapies Clinical Trials Immune Response Trials 103

thought leadership

JULY 24, 2023

Patients have the best outcomes when the treatment is safe, effective and provided at the right time. 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.

Therapies 75

Therapies Treatment 75

Conversations in Drug Development Trends

AUGUST 14, 2024

By: Simran Padam, Medical Director, Medical Affairs Personalized treatment approaches have emerged as pivotal in improving outcomes for hematological cancers. Hematological cancer cells can also develop resistance to therapies over time, reducing treatment effectiveness.

Treatment 78

Treatment Therapies Disease Protein Expression 78

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. Could you give us an overview of the Lineage platform?

Therapies 84

Therapies Small Molecule Pharmaceutical Companies Molecular Biology 84

Drug Target Review

JULY 12, 2023

Molecular-level biochemical assays like transcriptomics, genomics and proteomics have emerged as valuable tools for identifying potential targets in cancer treatment through deep cyclic inhibition (DCI). Importantly, transcriptomics information shows that certain aspects of a disease change the gene expression profile.

Biochemical Assays 98

Biochemical Assays Treatment Bioinformatics Disease 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.

Therapies 105

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?

Clinical Trials 72

Clinical Trials Immune Response Trials Disease 72

Broad Institute

NOVEMBER 7, 2023

While RA therapies targeted to specific inflammatory pathways have emerged, only some patients’ symptoms improve with treatment, emphasizing the need for multiple treatment approaches tailored to different disease subtypes. They also found that patients’ CTAPs were dynamic and could change over time in response to treatment.

Treatment 96

Treatment RNA Hospitals Disease 96

Drug Target Review

OCTOBER 25, 2023

Their findings may result in new treatments for reproductive conditions. He stated that this could inform new treatments for reproductive conditions like recurrent pregnancy loss and preeclampsia. Our findings could potentially lead to a cell therapy approach to treat those types of conditions.”

Treatment 106

Treatment Virus Research Science 106

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 ?

Therapies 98

Therapies Molecular Biology Laboratories Government 98

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

Codon

NOVEMBER 3, 2024

In the 1924 novel, The Magic Mountain , Thomas Mann describes a sanatorium patient named Anton Ferge as he undergoes a painful tuberculosis (TB) treatment. These challenges have led to strategies such as directly observed therapy (DOT), in which nurses or physicians monitor patients to ensure they take their medicine every day.

Vaccine 92

Vaccine Trials Therapies Clinical Trials 92

DrugBank

JUNE 13, 2024

These innovative technologies are changing how medications reach their targets within the body, leading to more effective treatments with fewer side effects.   Take cancer treatment, for example. Implantable insulin pumps equipped with continuous glucose monitoring (CGM) sensors can update old diabetes treatment options.

Therapies 74

Therapies Drugs DNA Treatment 74

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.

RNA 134

RNA Vaccine Small Molecule Therapies 134

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). While the trial waits for approval, patients who are eager for a new treatment face delay. Language relating to the potential cost of participation.

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Clinical Trials Trials Compliance Therapies 52

Fierce BioTech

JULY 25, 2023

How combining datasets from multiple sources can inform and improve commercial strategies Hundreds of rare disease treatments have entered the market over the past decade—thanks largely to a combination of government incentives, strong urging from patient advocacy groups and advances in cell and gene therapies.

Pharma Companies 52

Pharma Companies Government Therapies Disease 52

Drug Target Review

JULY 4, 2023

These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule drug alternatives. ADCs have the potential to redress the poor balance between safety and efficacy seen with traditional cancer treatment options.

Small Molecule 145

Small Molecule Drugs Clinical Pharmacology Trials 145

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

DrugBank

JULY 8, 2024

Revolutionizing Treatment Through the Skin with Transdermal Drug Delivery Systems The human skin, often perceived as a passive barrier to the external environment, holds potential as a route for drug administration. These fluctuations can lead to unwanted side effects or compromise the effectiveness of the treatment.

Treatment 78

Treatment Drugs Clinical Trials Engineering 78

PPD

OCTOBER 12, 2023

Cell and gene therapies (CGTs) are one of the fastest growing areas in human therapeutics. These treatments use a vector to introduce the desired nucleic acid code to replace or modify protein expression or use cells to alter/restore a specific cell type.

Laboratories 52

Laboratories Clinical Trials Protein Expression Pharmacokinetics 52

Drug Target Review

JANUARY 12, 2024

How does the integration of Real-World Data (RWD) with genomic biomarker data contribute to a more comprehensive understanding of disease progression and treatment response? This one size fits all approach to drug prescribing still pervades in mostly all therapy areas except oncology. Most drugs do not work in all people.

Doctors 111

Doctors Clinical Trials Treatment Therapies 111

Broad Institute

NOVEMBER 25, 2024

Ladders to Cures (L2C) Accelerator By Maria Nemchuk November 25, 2024 Breadcrumb Home Ladders to Cures (L2C) Accelerator The Ladders to Cures (L2C) Accelerator aims to catalyze progress across the research ecosystem and accelerates advances leading to treatments and cures for patients with rare genetic diseases.

Clinical Trials 52

Clinical Trials Disease Trials Therapies 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 ). By Charles G. Raver & James E. Valentine & Frank J. Duvyzat (givinostat), a histone deacetylase (HDAC) inhibitor developed by Italfarmaco, S.p.A.,

FDA Approval 59

FDA Approval Therapies FDA Trials 59

ASPET

JULY 15, 2024

XELOX therapy, which comprises capecitabine and oxaliplatin, is the standard first-line chemotherapeutic regimen for colorectal cancer. This study aimed to develop a translational PK-TD model using rat data to inform dosing strategies and TD implications in humans.

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Pharmacokinetics Therapies Drugs Treatment 100

Drug Target Review

AUGUST 21, 2023

To unravel the mysteries behind the treatment response, researchers from the Department of Otolaryngology/Head and Neck Surgery at UNC School of Medicine collaborated with experts from Yale Cancer Center, the Yale Head and Neck Cancer Specialised Program of Research Excellence (SPORE), and the ECOG-ACRIN Cancer Research Group.

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Therapies Treatment Clinical Trials Laboratories 98

Chemical Biology and Drug Design

SEPTEMBER 13, 2023

This review aims to provide information on cutting-edge Nano particulate-based technologies and Nano therapies for colorectal cancer. Abstract The advancement of colorectal cancer (CRC) prevention, detection, and treatment is essential to ensure that survivors live longer and higher-quality lives.

Drugs 100

Drugs Therapies Science Pharmaceuticals 100

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.

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Therapies RNA Pharma Companies Disease 56

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.

Drug Development 52

Drug Development Clinical Trials Compliance Trials 52

Fierce BioTech

AUGUST 7, 2024

A Focus on ADC and Radioisotope Therapy pesurya Wed, 08/07/2024 - 11:14 Wed, 10/16/2024 - 14:00 Resource Type Webinar Promotion Start Fri, 08/16/2024 - 11:17 Promotion End Sun, 02/16/2025 - 11:00 Duration 60 Minutes Medical imaging is critical in clinical trials, serving as a key component in assessing treatment efficacy and safety.

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Therapies Clinical Trials Trials Treatment 52

Advarra

NOVEMBER 29, 2022

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.

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Therapies Clinical Trials FDA Trials 52

Advarra

JUNE 13, 2023

Research in gene therapies and genetically engineered drugs and vaccines are growing exponentially, and will only continue to become more popular. The accelerating gene therapy market is expected to grow globally by 16.6% Some of these treatments potentially make permanent changes to the humans they are introduced into.

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Therapies Research Clinical Trials Trials 52