The Pharma Data

NOVEMBER 15, 2020

R&D collaboration building on Lead Pharma’s expertise in the discovery, design and optimization of small molecule treatments. ” About Immune Mediated Diseases. .” ” About Immune Mediated Diseases. A wide range of human diseases is driven by deregulated immune function.

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Drug Target Review

JULY 23, 2024

I studied Natural Sciences at the University of Cambridge, where I was exposed to a range of disciplines from chemistry to physiology, specialising in my last year in biochemistry. Shortly after finishing my studies, I landed my first job in industry working on cell biology research for several disease indications.

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Drug Target Review

JULY 6, 2023

TPD is a rapidly evolving therapeutic modality to degrade a disease-causing proteins, thereby eliminating their function specifically. 1 TPD is expected to challenge undruggable proteins, which are highly difficult to target by conventional small molecules.

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Drug Target Review

JULY 7, 2023

Approximately three percent of the global population — 240 million people — experience autoantibody diseases, which occur when one’s own body attacks critical organs and tissues. This can create an abnormal immune response that attacks the cells of our bodies and contributes to the development of autoantibody diseases.

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DrugBank

MAY 15, 2024

This method was more about serendipity than science. Today, we're able to identify and target specific molecules involved in disease processes—a method that's much more like using a sniper rifle than throwing darts blindfolded. One approach is to look beyond the traditional drug molecule.

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Covalent Modifiers

JANUARY 29, 2024

3c01835 Building on recent advances in peptide science, medicinal chemists have developed a hybrid class of bioconjugates, called peptide–drug conjugates, that demonstrate improved efficacy compared to peptides and small molecules independently. Moore Journal of Medicinal Chemistry 2024 DOI: 10.1021/acs.jmedchem.3c01835

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LifeSciVC

JANUARY 16, 2024

recent announcements from Merck and Sanofi for obesity and broader metabolic disease). Small molecule GLP1s? Kidney disease: outcomes in CKD are likely to benefit from weight loss as well, with the semaglutide Phase 3 FLOW trial stopped early last October after an interim efficacy read (full data expected this year).

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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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Drug Target Review

NOVEMBER 6, 2023

Advances in disease understanding and combination strategies in haematology We now know that blood cancers are characterised by molecular and phenotypic heterogeneity. These regimens frequently include one or two small molecule inhibitors and immuno-oncology (IO) therapies, such as monoclonal antibodies and T-cell engagers.

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Drug Target Review

OCTOBER 4, 2023

Antibody-drug conjugates (ADCs) have been a groundbreaking approach to cancer treatment with their ability to deliver cytotoxic drugs directly to diseased cells while sparing healthy tissues. Groundbreaking strategies like proteolysis-targeting chimeric molecules (PROTACs) are also being explored. Science 261 , 212–215 (1993).

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Broad Institute

OCTOBER 17, 2023

That interest in science led her to a weekend science course and a field trip to a plant research laboratory, where she saw scientists hybridizing roses and genetically modifying tomatoes. A lot of what we do at the Metabolomics Platform is look for early indicators of disease and use metabolomics to monitor disease progression.

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Drug Target Review

SEPTEMBER 25, 2024

Applying our technology has enabled us to develop a new small molecule, TT125-802, and to assign a new role to the epigenetic regulator CBP/p300 as a novel master regulator of non-oncogene resistance. This orally available small molecule binds to the bromodomain of CBP/p300 in a highly specific manner.

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The Pharma Data

NOVEMBER 19, 2020

The Company is also aiming to leverage the highly conserved structure of the SARS-CoV-2 M pro protease as a basis for the design of novel oral small molecules against predicted future variants of SARS-CoV-2 and other related human viruses. Zhang, et al, Science (2020) DOI: 10.1126/science.abb3405. References. Hilgenfeld, Febs J.

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KIF1A

JULY 22, 2023

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. A suppressor mutant means the disease model worm shows movement defects, however the suppressor shows better movement. Endpoints are measurements of a disease.

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KIF1A

JULY 1, 2023

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. In science, aligning mathematical models with experimental observations improves our understanding of the underlying biology. What’s a pre-print? . What’s a pre-print?

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NIH Director's Blog: Drug Development

OCTOBER 30, 2018

Now comes word of another absolutely incredible use of cryo-EM: determining with great ease and exquisite precision the structure of the smaller organic chemical compounds, or “small molecules,” that play such key roles in biological exploration and drug development. Also analyzed were eight less-familiar small molecules.

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The Pharma Data

OCTOBER 27, 2021

Small molecule candidate Anle138b targets disease modification for multiple system atrophy and other neurological disorders. Anle138b targets pathological alpha-synuclein oligomers and is being evaluated in patients with neurodegenerative diseases for potential disease modification. Multiple System Atrophy.

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Drug Target Review

DECEMBER 4, 2024

G protein-coupled receptors (GPCRs) represent a cornerstone of modern drug discovery due to their crucial role in regulating human physiology and their involvement in numerous diseases. Many of these receptors are key targets for addressing obesity and metabolic disorders.

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The Pharma Data

MAY 31, 2022

BIIB122 is an inhibitor of LRRK2, a potential novel target intended to impact the underlying biology and slow the progression of Parkinson’s disease Phase 2b LUMA to enroll approximately 640 participants with early-stage Parkinson’s disease; most advanced clinical study of a LRRK2 inhibitor. Denali Therapeutics Inc.

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DrugBank

OCTOBER 17, 2024

Data science has emerged as an innovative tool in the biopharmaceutical industry, leveraging the power of machine learning and artificial intelligence to drive innovation and efficiency across the entire drug development lifecycle. These complex molecules require precise engineering to ensure optimal efficacy and safety.

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The Pharma Data

DECEMBER 15, 2020

A healthy immune system defends the body against disease and other conditions. Autoimmune disease impacts different parts of the body, weakening functionality. Researchers are aware of more than 80 diseases that occur when the immune system attacks the body’s own organs, tissues and cells. It may be life-threatening.

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The Pharma Data

JANUARY 5, 2021

Durham, North Carolina-based Ribometrix announced a strategic collaboration deal with Genentech , a Roche company, to identify and advance novel RNA-targeted small molecule therapeutics. This is because many proteins do not have small-molecule binding sites. This is not the first deal for RNA therapeutics for Genentech.

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Drug Target Review

MAY 6, 2024

Luckily, my love for science remained. Melanoma was an ideal disease for studying this phenomenon because cutaneous tumour biopsies are readily available. What if our science can directly impact patients’ lives?” It’s essential for the performance and competitiveness of our businesses and the progress of science in general.

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DrugBank

MAY 22, 2024

These tough nuts to crack in medical science—biological targets known to play roles in diseases but resistant to traditional drug design—are now seeing new strategies that shift the paradigm from "undruggable" to "druggable." " The Challenge of Selectivity  Why are some targets so elusive?

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NIH Director's Blog: Drug Discovery

APRIL 11, 2017

Credit: National Center for Advancing Translational Sciences, NIH When you think of the causes of infectious diseases, what first comes to mind are probably viruses and bacteria. Missing was the usual pocket-shaped active site, where a traditional small molecule can readily bind and block function. ” [1] Or was it?

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PerkinElmer

MAY 28, 2020

Time is of the essence when it comes to the development of a vaccine for the novel infectious disease in our current pandemic. The importance of pseudoviruses Because of their versatility and safety, tools such as pseudoviruses can be used by virologists as an alternative method to develop a vaccine for the novel viral disease.

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KIF1A

JANUARY 27, 2024

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. This would allow a single gene therapy to replace an area of KIF1A that includes multiple disease-causing mutations, and treat multiple mutations at once.

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Sygnature Discovery

APRIL 18, 2023

Sygnature Discovery is pleased to have entered into a research collaboration agreement with the global healthcare group Daewoong Pharmaceutical to accelerate the discovery of a novel small molecule to target autoimmune disease.

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Broad Institute

SEPTEMBER 26, 2024

Yes, it changed me as a researcher: I gained the skills to overcome obstacles in science, plan and execute my experiments, and dive deep into the background of a project to build confidence in my research area. Additionally, astrocyte inflammation has been observed in patients with many neurodegenerative diseases.

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KIF1A

MAY 27, 2023

Dylan Verden of KIF1A.ORG summarize newly published KIF1A-related research and highlight progress in rare disease research and therapeutic development. Goodbye with Gratitude: Dr. Dominique Lessard It is with heavy hearts that this week we say goodbye to Dr. Dominique Lessard, Chief Science Officer of KIF1A.ORG. What’s a pre-print?

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Dark Matter Blog

JUNE 21, 2023

Since our company’s founding in 2015, we have taken the long view and been singularly focused on building an extremely flexible and broadly applicable platform that can develop a host of RNA‑targeted small molecules (rSMs) to deliver precision medicines for dozens of targets that have been out of reach for conventional approaches.

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The Pharma Data

JUNE 7, 2022

AL01811 is a preclinical selective GBA2 inhibitor with first-in-class potential as an oral disease modifying treatment for Parkinson’s Disease Alectos to receive a $15 million upfront payment and is eligible to receive potential future development and commercial milestone payments. Biogen Inc. President and CEO at Alectos Therapeutics.

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The Pharma Data

NOVEMBER 29, 2020

(Headquarters: Cambridge, UK, “Wren”) today announced that the companies have entered into an exclusive research collaboration agreement aiming to advance the discovery of novel small molecules that target ?-synuclein synuclein for the potential treatment of synucleinopathies including Parkinson’s disease and dementia with Lewy bodies.

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LifeSciVC

JULY 6, 2023

Although these results cannot prove that there is no possibility of finding a high-affinity small molecule binder of NBD1, they are discouraging…” It turns out the small molecules they were looking for are not only possible – they were ready for a dedicated team to push them ahead.

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The Pharma Data

JULY 15, 2021

This extension will further help connect life-science innovators in Victoria to accelerate their healthcare solutions across the globe,” said Stacy Feld, Regional Head, Johnson & Johnson Innovation LLC, West North America, Australia & New Zealand.

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BMG Labtech

MAY 22, 2024

Options for cell-based protein degrader assays Interest is growing in the use of targeted-protein degradation to induce the breakdown of disease-causing proteins after treatment with small molecule drugs.

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Dark Matter Blog

JANUARY 16, 2023

Our terra firma is small-molecule medicines. As we know, small molecules have been the mainstay of our pharmacopeia for several centuries and have, almost without exception, targeted proteins. This is why Arrakis set out in 2015 on an expedition to figure out how to develop small molecules that can target RNA.

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