DrugBank

JULY 24, 2024

These multifunctional small molecules are like tiny spies, hijacking the body’s natural protein degradation system to remove unwanted proteins. For example, PROTACs targeting STAT3, a protein crucial for tumor growth, have shown promise where conventional therapies have failed.

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The Pharma Data

NOVEMBER 17, 2020

Lead Pharma has entered into a collaboration and license agreement with Roche , hoping to lead the way in the development of oral small molecules for immune-mediated diseases like rheumatoid arthritis, psoriasis and inflammatory bowel diseases. . This includes therapies against the nuclear receptor ROR?t, Source link.

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Broad Institute

OCTOBER 4, 2023

By Allessandra DiCorato October 4, 2023 Credit: AbbVie The new small molecule inhibitor (green) sits inside the PTPN2 protein, where acidic sites are marked in red and basic sites are marked in blue. 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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Drug Target Review

JANUARY 4, 2024

What distinguishes Alder Therapeutics’ approach to regenerative cell therapy development from traditional methods, and how does it aim to reduce risks in the preclinical phase? Traditional approaches to regenerative cell therapy development are defined by several challenges. So, what makes these therapies unique?

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Dark Matter Blog

AUGUST 27, 2020

A few years ago, at Arrakis Therapeutics, we set out to conquer a strange new territory, drugging RNA structures with small molecules. The public reports of this drug’s clinical benefits were thrilling to everyone – patients, physicians, and researchers. But we have not been the only people on this mission. We will replace it.”

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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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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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DrugBank

MAY 15, 2024

Researchers would sift through natural extracts , hoping to stumble upon a compound that exhibited beneficial effects. In fact, it's one of the most exciting areas in drug research today. With billions in funding and innovative research, we're starting to see strategies that can tackle these tough targets.

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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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Drug Target Review

OCTOBER 6, 2023

This interview explores how CoRegen’s innovative techniques are not only transforming cancer treatment but also hold promise for a wide range of medical applications, all while minimising the side effects often associated with traditional therapies. What are the key benefits of your approach?

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Drug Target Review

JUNE 15, 2023

In this article Drug Target Review’s Izzy Wood spoke to Sam Hasson, Director of Target Biology at Rgenta Therapeutics, a biotech firm in Massachusetts, US, that aims to develop small molecule therapeutics to target RNA processing. It is somewhat like how PROTACs have changed the perspective on what is a tractable therapeutic target.

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Drug Target Review

SEPTEMBER 25, 2024

Most targeted cancer therapies used today operate by inhibiting targets along well-known oncogenic signalling cascades. The reactivation of oncogenic signalling upstream or downstream of the driving oncogene is a well-studied source of resistance to targeted cancer therapies.

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Drug Target Review

OCTOBER 4, 2023

The research community pressed on, turning to novel compounds that were substantially more potent than those used in the first generation. Groundbreaking strategies like proteolysis-targeting chimeric molecules (PROTACs) are also being explored. Dual cytotoxic payload ADCs could address the inherent heterogeneity in tumours.

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Drug Target Review

OCTOBER 11, 2023

1,2 Many, however, do have dysfunctional proteins at their core, and for these diseases, a new option is being explored: autophagy-boosting therapies. Gene therapy, of course, also holds great promise. Autophagy boosters, in contrast, are conventional small molecule drugs.

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Drug Target Review

JULY 23, 2024

Shortly after finishing my studies, I landed my first job in industry working on cell biology research for several disease indications. At the time, research and leadership roles in biotech/pharma industry were predominately held by men and there were not as many opportunities for career development and mentorship from women leaders.

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Drug Target Review

NOVEMBER 6, 2023

Advances in research and development and the utilisation of combination treatment strategies are revolutionising the haematology care landscape, but more must be done to address patients’ individual needs. 2 It is this complexity that necessitates powerful, targeted combination therapies.

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KIF1A

JANUARY 27, 2024

Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. The study found improved spasticity and movement in mice treated with ARL61P1 gene therapy; it also restored abnormalities observed in the brains of ARL61P1-deficient mice.

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LifeSciVC

JANUARY 16, 2024

These are incredible forecasts and CAGRs, which if we assume directionally correct, rely not only on steady growth for approved therapies but also a substantial success rate of, and continued investment in, the development pipeline. Small molecule GLP1s?

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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.

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DrugBank

OCTOBER 17, 2024

By harnessing the vast amounts of data generated throughout the development pipeline, pharmaceutical companies can accelerate the discovery of novel therapies, optimize clinical trial design, enhance drug safety monitoring, and deliver personalized medicine, ultimately improving patient outcomes and transforming the future of healthcare.

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PerkinElmer

NOVEMBER 9, 2022

Immunotherapy has revolutionized cancer treatment and proved to be pivotal in the field of oncological research. While certain patients respond to these therapies, antibody drugs have some natural drawbacks such as poor oral bioavailability, poor permeability of tumor tissues, immune-related adverse events, and high medical costs.

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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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Conversations in Drug Development Trends

MAY 30, 2024

These advancements have fostered increased focus from politicians, policymakers, and the investment communities backing academic and industry-sponsored research projects. Researchers established efficacy of edaravone in a 24 week, randomized, placebo-controlled double-blind study in 137 Japanese patients.

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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. While mRNA usage has played several roles in clinical research , oncology researchers in particular are eager to explore the possibilities of mRNA-based cancer vaccines.

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Drug Target Review

MARCH 1, 2024

Can you elaborate on the key findings of the preclinical data published in Cancer Immunology Research? How significant are the results in terms of advancing TCR-T cell therapy and AMP immunotherapy for the treatment of solid tumours?

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The Pharma Data

JANUARY 19, 2021

A naturally occurring system for tuning CRISPR-Cas9 expressing in bacteria, identified in a study published in Cell , could have implications for gene editing therapies as well. In particular, Editas is known to be exploring tunable CRISPR-Cas9 systems for therapies has developed self-inactivating Cas9 adeno-associated virus vectors.

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The Pharma Data

APRIL 6, 2021

Bayer will present new research across its oncology portfolio at the virtual American Association for Cancer Research (AACR) Annual Meeting 2021 , taking place over two weeks on April 10-15 and May 17-21, 2021. The compound has also received a breakthrough therapy designation in the U.S.

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The Pharma Data

NOVEMBER 30, 2020

Biohaven acquires exclusive global rights to a portfolio of novel, small-molecule CGRP antagonists. We look forward to working on this new collaboration with Biohaven, a world leader in the clinical development of CGRP-targeted therapies.” TOKYO and CAMBRIDGE, England , Dec. Vlad Coric , M.D.,

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DrugBank

JANUARY 9, 2025

Targeted Therapies vs. Traditional Treatments Traditional cancer treatments, such as chemotherapy and radiotherapy, are inherently cytotoxic, targeting both cancerous and healthy cells. The development of imatinib to treat chronic myeloid leukemia (CML) serves as a hallmark for targeted therapies.

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Drug Target Review

MAY 6, 2024

Could you share an example of a specific project or research that you have worked on and the impact it has had in your field? The clinical efficacy of BRAF inhibitors, like most other targeted cancer therapies, is inevitably limited by resistance development. Luckily, my love for science remained.

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DrugBank

AUGUST 15, 2024

To effectively navigate this ecosystem and expedite the development of new therapies, collaboration between the pharmaceutical industry and academia is proving increasingly vital. An example is the collaboration between Novartis and the University of Oxford to develop a gene therapy for spinal muscular atrophy, a rare genetic disease.

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Broad Institute

JUNE 30, 2023

Common genetic variants associated with cardiometabolic disease can produce phenotype changes of such small effect that they can be difficult to characterize. The researchers also looked at the tool’s ability to detect changes in cellular phenotypes after exposure to drugs and small molecule compounds.

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Drug Target Review

SEPTEMBER 11, 2024

How does morADC technology combine the properties of small molecules and monoclonal antibodies to enhance anti-aggregation effects for CNS applications? The morADC are able to cross the blood-brain barrier more efficiently and offer higher potency than individual parent molecules.

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NIH Director's Blog: Drug Development

OCTOBER 11, 2022

Through the years, NIH has supported a total of 169 researchers who have received or shared 101 Nobel Prizes. This form of chemistry has made it possible for researchers to snap together, like LEGO pieces, molecular building blocks to form hybrid biomolecules, often with easy-to-track imaging agents attached.

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The Pharma Data

AUGUST 5, 2021

The acquisition strengthens BioNTech’s cell therapy pipeline by accelerating the individualized solid tumor Neoantigen TCR cell therapy research and development program. It also expands the Company’s cell therapy capabilities and manufacturing footprint in North America, building on its acquisition of Neon Therapeutics in 2020.

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New Drug Approvals

APRIL 2, 2025

1] [2] It is a non- opioid , small-molecule analgesic that works as a selective inhibitor of Na v 1.8 acting director of the FDA’s Center for Drug Evaluation and Research. The application received Breakthrough Therapy , Fast Track and Priority Review designations by the FDA. under nitrogen. ESI-MS m/z calc.475.0922,

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KIF1A

MAY 9, 2023

KIF1A.ORG’s 23rd Research Roundtable meeting, “Structure to Patient Perspective: Vision Loss in KAND” was presented by KIF1A.ORG’s Dr. Dylan Verden. Dr. Verden is the Research Engagement Director for KIF1A.ORG. He has brought his expertise in neurodevelopment and neurodegeneration to KIF1A.ORG’s science team.

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