Drug Discovery World

SEPTEMBER 4, 2024

EVO756 is a highly selective small molecule antagonist of mas-related G-protein coupled receptor X2 (MRGPRX2) and could offer the potential for once-daily oral administration without the side effects observed with other therapies.

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Drug Discovery World

DECEMBER 12, 2023

Exonate, an mRNA therapy company focused on treatments for diabetic complications, has announced that its lead ophthalmology asset, EXN407, has achieved its prespecified endpoints in a Phase Ib/IIa study. During the trial, EXN407 met all safety and pharmacokinetic parameters and displayed encouraging signals of biological activity.

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Drug Discovery World

MAY 30, 2024

A new research paper has validated the mechanism of action of the Atrogi’s first-in-class, small molecule, ATR-127, in combating obesity and metabolic complications. Unlike existing anti-obesity therapies that compromise lean muscle mass, ATR-127 induces significant weight loss while preserving crucial lean body mass.

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Drug Discovery World

JUNE 26, 2024

Spinogenix has initiated a Phase II trial to evaluate SPG302 for the treatment of adult participants with mild-to-moderate Alzheimer’s disease (AD), following approval for the trail in Australia. SPG302 is a once-a-day pill being developed as a regenerative treatment for neurodegenerative and neuropsychiatric diseases.

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Covalent Modifiers

FEBRUARY 6, 2024

Fibroblast growth factor receptor (FGFR) kinase inhibitors have been shown to be effective in the treatment of intrahepatic cholangiocarcinoma and other advanced solid tumors harboring FGFR2 alterations, but the toxicity of these drugs frequently leads to dose reduction or interruption of treatment such that maximum efficacy cannot be achieved.

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Drug Discovery World

DECEMBER 8, 2023

Andrew Busey , Co-Founder, Form Bio discusses why vertical AI is set to reshape the cell and gene therapy sector. The cell and gene therapy industry is no exception to this digital transformation. It is imperative for the industry to communicate the precise composition and attributes of these therapies to regulatory bodies.

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Drug Target Review

OCTOBER 11, 2023

Most of these conditions are genetic in origin and the majority have no effective treatment. 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.

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

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Therapies Small Molecule Pharmaceutical Companies Molecular Biology 84

Drug Discovery World

FEBRUARY 12, 2024

Cameron Ross, SVP, Generative AI, Elsevier “AI has the potential to dramatically shorten one of the most costly areas of early drug development – small molecule discovery. We’re already seeing biotechs making breakthroughs with AI in this area, with more than 150 small molecule drugs discovered and 15 already in clinical trials.

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Drug Discovery World

AUGUST 16, 2024

Misetionamide is a tumour cell selective small molecule drug that is broadly active in multiple cancer models. New published data have demonstrated positive monotherapy results for misetionamide as well as synergistic activity with PARP inhibitors (PARPis) and bevacizumab antineoplastic in ovarian cancer.

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Drug Discovery World

OCTOBER 21, 2022

While advances in treatment over the last few years have resulted in good overall survival rates for patients with non-metastatic disease (90% five-year survival rate in the US 1 ), outcomes are still poor in certain cancers that do not respond to standard therapies. Advances in understanding treatment resistant cancer cells (part 1).

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Drug Discovery World

FEBRUARY 19, 2024

However, despite these challenges, there are some positive developments; for instance, potential signs of improvement in the financial situation were flagged in late 2023 with AstraZeneca’s acquisition of cell therapy company Gracell for $1.2bn. antibody-drug conjugate [ADC] pipelines in some oncology settings).

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Drug Discovery World

MARCH 12, 2024

Advancements in antibody-drug conjugates are also expected, enhancing targeted delivery for cancer treatments.” Carole Nicco, CSO, BioSenic “mAb immunotherapy is the most widely used approach and has achieved tremendous success. Other ACTs such as TIL, TCR, NK cell, Treg, and MDSC therapies are now emerging.

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Molecular Biology Small Molecule Therapies FDA Approval 205

Olympian Clinical Research

MAY 6, 2024

The journey toward effective treatments has been long and evolving, with recent breakthroughs offering new hope to those living with this challenging condition. From Coal Tar to Biologics: A Historical Perspective The history of psoriasis treatment is as old as the condition itself, with records dating back to ancient times.

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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,

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Small Molecule Disease Immune Response Licensing 52

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. On the strength of those findings, nusinersen was approved by the FDA at the end of 2016 for the treatment of SMA , marketed under the commercial brand Spinraza. We will replace it.”

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Drug Discovery World

MARCH 15, 2024

Following the recent AD/PD 2024 conference, which took place on 5-9 March in Lisbon, Portugal, there have been a number of key announcements regarding investigational therapies for neurological disorders, particularly Alzheimer’s disease and Parkinson’s disease.

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The Pharma Data

JANUARY 19, 2021

Precision medicine developer Kura Oncology is positioned to make a meaningful difference in the treatment of cancer in the next few years with its two, wholly-owned, small molecule compounds. As with any innovative therapy, “It takes multiple iterations and time to get things right,” he pointed out. “We dependent HNSCC.

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Drug Discovery World

JULY 18, 2024

DSP-5336 is an investigational small molecule inhibitor of the menin and mixed-lineage leukaemia (MLL) protein interaction, which plays key roles in gene expression and protein interactions involved in many biological pathways, including cell growth, cell cycle, genomic stability, and haematopoiesis.

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Drug Discovery World

MAY 24, 2023

million to develop the next generation of small molecule drugs against disease areas of high clinical need. The aim is to develop therapies with fewer side effects to treat diseases with a high clinical need. PharmEnable has closed a pre-series A investment round of $7.5 The post $7.5M

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Small Molecule Disease Therapies Treatment 130

Broad Institute

NOVEMBER 2, 2023

Bayer and Broad Institute extend cancer therapy research collaboration By Corie Lok November 2, 2023 Breadcrumb Home Bayer and Broad Institute extend cancer therapy research collaboration Long-standing industry-academia collaboration has already resulted in three clinical oncology candidates.

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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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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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Drug Discovery World

APRIL 5, 2024

The platform has a track record of identifying highly selective, brain penetrant small molecule drugs, a class that has the potential to revolutionise the treatment of neurological disorders. “We

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Drug Discovery World

JULY 19, 2024

LT-HSCs are vital for bone marrow transplants as they can replenish the blood cells after treatment for blood cancers. Currently used animal models are poor predictors of drug outcomes, and many of the blood disease treatments on offer – such as mRNA drugs and human-specific small molecules – don’t test well in animal models.

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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. 3D rendering of Antibody Drug Conjugate Molecules.

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Drug Discovery World

SEPTEMBER 6, 2022

DDW Editor Reece Armstrong looks at the cell and gene therapy landscape, examining the challenges facing developers and the trends we can expect to see throughout the year. . There’s no doubt that cell and gene therapies present some of the most exciting opportunities for emerging drugs. million price point per dose.

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Drug Discovery World

AUGUST 18, 2023

My chosen news stories this week all discuss pre-clinical research studies that could have a huge impact on our therapeutic approach to several hard-to-treat diseases, helping to overcome drug-resistance and make treatments more targeted in the future.

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Drug Discovery World

AUGUST 16, 2023

Over the next two decades, it is quite possible that most new drugs approved could be based on some form of RNA, whether it be through its delivery or targeting the molecule itself. Second, this approach is safer than other currently available treatment options because dark genome lncRNA targets are highly specific to tissues and cell states.

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PPD

JUNE 27, 2024

In recent years, AA has followed in the footsteps of other dermatology indications — including psoriasis and atopic dermatitis — with growing research and clinical studies focusing on small molecules with broad cellular effects (JAK and PDE4 inhibitors) and cytokine-specific molecules antagonists (IL-23, Th2 and IL17) to treat the condition.

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Drug Discovery World

MARCH 10, 2023

Perhaps most interestingly this week, after previous caution over in vivo gene therapy trials, the FDA has given the go ahead to Intellia Therapeutics to study NTLA-2002 in hereditary angioedema (HAE).

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Drug Discovery World

JANUARY 17, 2023

“The peptides that we have discovered can be used as targeting ligands directly conjugated to silencing RNAs, small molecules for therapeutics or as imaging probes,” Herrera-Barrera added. . Limitations of AAV gene therapy . Sahay and Ryals have received a $3.2

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Drug Discovery World

NOVEMBER 27, 2023

. “The NMPA approval of vebreltinib is an important milestone toward providing a new treatment option for patients with MET exon 14 skipping NSCLC in China. Vebreltinib is a potent, small molecule, orally bioavailable and highly selective c-Met inhibitor.

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Drug Discovery World

JULY 27, 2022

Biotechnology company Recursion has been granted an Orphan Drug Designation for its potential treatment of the rare disease familial adenomatous polyposis (FAP). . As it stands there are no approved therapies for the disease. . Now, the European Commission has granted REC-4881 Orphan Drug Designation. . Official comments .

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Drug Discovery World

OCTOBER 4, 2022

Using its proprietary compound screening platform, Oligomerix discovers and develops differentiated, oral, small molecule inhibitors of tau self-association that are easy to administer and cost-effective. . With its recent award of $3.35 With its recent award of $3.35 Focused on opportunity .

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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. Adoptive T Cell therapies, therapeutic antibodies, and immunomodulatory proteins represent just some of the potentially beneficial treatment strategies for successful mRNA cancer trials.

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Drug Discovery World

MAY 1, 2024

Many of the best-selling drugs in 2023 were monoclonal antibody (mAB) therapies, including Keytruda (pembrolizumab), Humira (adalimumab), and Dupixent (dupilumab) 1. These enable researchers to better manipulate biological molecules or identify the best candidates to progress to the next stage of development.

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